Podcasts about Gene therapy

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into some fascinating updates that are shaping the future of medicine and healthcare.Let's start with a groundbreaking development in cancer treatment. Researchers have announced significant progress in a novel therapy targeting a specific mutation often found in non-small cell lung cancer. This mutation, known as EGFR exon 20 insertion, has historically been resistant to standard treatments. The new therapy employs a targeted approach that precisely inhibits the mutant protein while sparing normal cells. Early-phase clinical trials have shown promising results, with substantial tumor shrinkage observed in participants. This could potentially redefine treatment protocols for patients who previously had limited options and improve their overall survival rates. As the study progresses into later phases, the industry is watching closely to see if these initial successes translate into long-term benefits.In another significant development, we're seeing advancements in gene therapy for inherited retinal diseases. A recent study has highlighted a novel gene-editing technique that promises to restore vision in patients with certain genetic forms of blindness. By utilizing CRISPR-Cas9 technology, scientists have been able to directly correct mutations in retinal cells. The preclinical models have shown restored function and improved visual responses, paving the way for human trials. This breakthrough is not just a beacon of hope for those affected by genetic blindness but also underscores the transformative potential of gene-editing technologies in treating complex diseases.Moving on to regulatory news, there's an update on new drug approvals that could have widespread implications for public health. The FDA has recently approved a first-in-class drug for the treatment of severe migraines. This medication represents a novel mechanism of action by targeting the calcitonin gene-related peptide (CGRP) pathway, which plays a crucial role in migraine pathophysiology. Clinical trials indicated that it significantly reduces the frequency and severity of migraine attacks compared to existing treatments. For millions of sufferers worldwide, this approval offers a new avenue for relief and highlights the importance of continued innovation in chronic pain management.Shifting gears to vaccine development, there's exciting progress in the fight against infectious diseases. A new vaccine candidate for malaria has shown an unprecedented level of efficacy in trial settings. This vaccine utilizes a protein-based approach that targets multiple stages of the parasite's lifecycle, thereby enhancing its protective effects. Given malaria's devastating impact globally, particularly in sub-Saharan Africa, this development is being hailed as a potential game-changer in global health efforts. As further studies and real-world evaluations unfold, this vaccine could become a cornerstone tool in reducing malaria's burden.Now turning our attention to industry trends, there's growing momentum around personalized medicine and its integration into mainstream healthcare systems. Personalized medicine tailors treatment strategies to individual patient profiles based on genetic, environmental, and lifestyle factors. Recent advances in genomics and data analytics have accelerated this shift, allowing for more precise and effective interventions. For healthcare providers and pharmaceutical companies alike, this trend necessitates rethinking traditional drug development models and embracing collaborative approaches to harness big data effectively.Finally, let's look at an intriguing development in neurodegenerative disease research. Scientists are exploring a new class of drugs designed to target protein misfolding—an underlying cause of conditions Support the show

In this visionary episode of Beauty Bytes, I am saying goodbye to the "filler fatigue" of 2025 and unveiling my Top 10 Aesthetic Predictions for 2026. This isn't just about trends; I'm mapping out a paradigm shift toward hyper-personalization and regenerative medicine that will change how we treat aging forever. I explain why the era of overfilling is dead and how I am pivoting to "undetectable" results using micro-tox, strategic lifting, and collagen biostimulators. I also explore the cutting-edge future of AI diagnostics, where digital avatars will allow me to simulate your aging trajectory and show you treatment outcomes years in advance . We dive deep into the science of skin senescence, targeting "zombie cells" to restore resilience rather than just treating wrinkles . From turning on your body's mTOR cycles for cellular repair to the rise of collagen banking for Gen Z, I'm sharing exactly where the world of aesthetics is heading next year.

Shunji Tomatsu, MD, PhD, Professor and Head, Nemours Children's Health, Delaware, USA; Alessandra d'Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children's Research Hospital, Tennessee, USA; Merve Emecen Sanli, MD, Associate Professor, Department of Pediatrics, University of Texas Southwestern Medical Center, Texas, USA; and Ryan Colburn, patient with Pompe disease and president of Odimm Inc, discuss new and emerging gene therapies for lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, please visit https://checkrare.com/learning/p-grids2025-session6-current-issues-in-gene-therapies-for-lysosomal-disorders/  Learning ObjectivesDescribe current and emerging gene therapy data in lysosomal disorders and its clinical relevanceDescribe role of patients in gene therapy developmentFacultyShunji Tomatsu, MD, PhD, Professor and Head, Nemours Children's HealthAlessandra d'Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children's Research HospitalMerve Emecen Sanli, MD, Associate Professor, Department of Pediatrics, University of Texas Southwestern Medical CenterRyan Colburn. Odimm, Inc.DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Shunji Tomatsu, MD, PhD Dr. Tomatsu has received the following grants: Morquio Foundations and families: Scarlett Grifith, Bennett, A Cure for Roberts, and Morquio Conference; MPS Societies: Japanese, National, and Austrian; NIH grants: 1-R01-HD102545, NIH, NICHD, Tomatsu (PI), 1R01HD104814-01A1, NIH, NICHD, Langan, T.J. (PI), Role: Site-PI, R43HD114328-01, NIH, ACOSTA, WALTER (PI), Role: site PI, 1R43AR084638-01, NIH, MOUNZIH, KHALID (PI); Foundation of NIH: FNIH RFP NUMBER: 2022-BGTC-005 Tomatsu (PI). Alessandra d'Azzo, PhDDr. D'Azzo has no relevant financial relationships to disclose.Merve Emecen Sanli, MDDr. Sanli has no relevant financial relationships to disclose.Ryan ColburnMr. Colburn has an advisory, consulting and/or project based relationship or stock holding with: Abeona Therapeutics, Amicus Therapeutics, Astellas Gene Therapies, Avidity Biosciences, Bayer, Catalyst Pharmaceuticals, Denali Therapeutics, M6P Therapeutics, Sangamo Therapeutics, Sanofi, Solid Biosciences.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.comSend customer support requests to cds_support+ldrtc@affinityced.com

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a series of fascinating stories that underscore the dynamic nature of our industry, where scientific innovation meets real-world application.Starting with a significant advancement in gene therapy, researchers have made remarkable progress in a novel approach to treating rare genetic disorders. This new methodology involves the use of CRISPR technology to edit genes directly within the human body, paving the way for potential cures once thought impossible. By targeting specific DNA sequences, scientists can now correct genetic mutations at their source. This breakthrough not only promises to transform the treatment landscape for rare diseases but also enhances our understanding of genetic disorders at a molecular level. The implications here extend beyond rare conditions, potentially offering new avenues for tackling more common genetic diseases in the future.Moving forward, let's discuss recent regulatory developments that have caught the industry's attention. The FDA has granted accelerated approval to a new oncology drug that shows promise in treating advanced forms of breast cancer. This decision was based on compelling clinical trial results demonstrating significant improvements in patient survival rates compared to existing therapies. The drug targets specific proteins involved in tumor growth, offering a more precise treatment option with potentially fewer side effects. This approval exemplifies how regulatory bodies are adapting to expedite access to life-saving treatments while ensuring rigorous safety and efficacy standards.In another noteworthy development, a Phase III clinical trial has yielded positive results for a novel Alzheimer's drug. The trial demonstrated that this drug significantly slows cognitive decline in patients with early-stage Alzheimer's disease. By targeting amyloid plaques in the brain, which are believed to contribute to neurodegeneration, this therapeutic approach represents a potential shift in how we treat this debilitating condition. These findings provide hope for millions affected by Alzheimer's and underscore the importance of continued investment in neuroscience research.Turning our attention to infectious diseases, there's exciting news from a biotech company focusing on vaccine development. They've announced encouraging preliminary data from trials of their new mRNA-based vaccine for respiratory syncytial virus (RSV). RSV is known for causing severe respiratory illness, particularly in infants and the elderly. The vaccine demonstrated robust immune responses and a favorable safety profile, suggesting it could become an important tool in preventing RSV infections globally. This development highlights the versatility of mRNA technology, which has already revolutionized COVID-19 vaccine design and holds promise for addressing various infectious diseases.In terms of industry trends, one cannot overlook the growing emphasis on personalized medicine. Recent advancements in biomarker research are enabling more tailored therapeutic approaches across multiple disease areas. By identifying specific genetic or molecular markers associated with diseases, healthcare providers can better predict patient responses to certain treatments. This shift towards precision medicine not only improves patient outcomes but also enhances healthcare efficiency by reducing trial-and-error prescribing.Lastly, we delve into an intriguing area of metabolic disorders where innovative therapeutic strategies are emerging. A biotech firm has developed a first-in-class oral medication for treating non-alcoholic steatohepatitis (NASH), a serious liver condition linked to obesity and metabolic syndrome. The drug works by modulating key metabolic pathways involved in liver inflammation and fibrSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world.Today, we dive into the latest breakthroughs and innovations shaping our industry. We start with a remarkable advancement in Alzheimer's disease treatment. A recent clinical trial has demonstrated significant progress in slowing cognitive decline among patients suffering from this challenging condition. The study, which involved a novel monoclonal antibody, showed promise by targeting amyloid plaques, a hallmark of Alzheimer's pathology. This approach not only opens new avenues for treatment but also provides hope for millions of patients and their families. The scientific community is closely monitoring these developments as they could redefine therapeutic strategies for neurodegenerative diseases.Moving on to regulatory news, the FDA has recently granted approval to a groundbreaking gene therapy for hemophilia B. This therapy represents a significant milestone as it offers a potential one-time treatment to correct the genetic defect underlying the disorder. By delivering a functional copy of the gene necessary for clotting factor production, patients may experience reduced bleeding episodes and improved quality of life. This approval underscores the FDA's commitment to advancing personalized medicine and highlights the transformative potential of gene therapies in addressing rare genetic conditions.In oncology, we've seen exciting progress with a novel targeted therapy showing efficacy in treating advanced stages of lung cancer. The drug specifically inhibits a mutation found in non-small cell lung cancer, which is often resistant to conventional treatments. Clinical trials have reported improved survival rates and better tolerability compared to existing therapies, marking a significant step forward in precision oncology. As researchers continue to unravel the complexities of cancer genomics, targeted therapies like this one offer new hope for patients battling aggressive forms of cancer.The biotech industry is also witnessing a surge in collaborations aimed at expediting vaccine development. In light of recent global health challenges, several companies have entered strategic partnerships to leverage their combined expertise in mRNA technology. These collaborations aim to accelerate the production and distribution of vaccines for infectious diseases beyond COVID-19. By pooling resources and sharing technological advancements, these alliances have the potential to enhance our preparedness for future pandemics and improve global public health outcomes.Shifting focus to autoimmune diseases, a novel small molecule inhibitor has shown potential in managing rheumatoid arthritis symptoms more effectively than traditional treatments. This new drug targets specific pathways involved in inflammation without compromising immune function. Early clinical data suggests it could offer patients relief with fewer side effects, representing a promising addition to the therapeutic arsenal against chronic inflammatory conditions.In an intriguing development within regenerative medicine, researchers have made strides in bioengineered organs. A recent breakthrough involves creating functional liver tissue from stem cells, paving the way for future organ transplantation solutions. These lab-grown tissues have demonstrated essential liver functions in preclinical models, bringing us closer to addressing organ shortages and enhancing transplant success rates. The implications of such advancements extend far beyond liver disease, offering transformative possibilities for regenerative therapies across various medical fields.Turning our attention to antimicrobial resistance, an ever-pressing concern within global health, scientists have discovered a new class of antibiotics capable of combating multidrug-resistant bacteria. This discovery comes atSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into some fascinating breakthroughs and regulatory updates that are shaping the future of healthcare.Starting with a pivotal advancement in Alzheimer's research, a new drug has shown promising results in slowing cognitive decline in patients with early-stage Alzheimer's disease. This innovative therapy targets amyloid plaques in the brain, which are believed to play a central role in the progression of Alzheimer's. Recent clinical trials have demonstrated that patients receiving this treatment experienced a significant reduction in the rate of cognitive deterioration compared to those on a placebo. The implications of this development are profound, offering hope for millions affected by this debilitating condition. As researchers continue to unravel the mysteries of Alzheimer's, this breakthrough marks a crucial step forward in understanding and potentially halting disease progression.Moving on to regulatory news, the FDA has recently granted accelerated approval to a novel gene therapy for a rare genetic disorder known as spinal muscular atrophy (SMA). This condition, which affects motor neurons and leads to muscle wasting and weakness, primarily impacts infants and young children. The newly approved therapy works by delivering a functional copy of the defective gene directly into the patient's cells. Early trials have shown remarkable improvements in motor function and survival rates among treated infants. This approval represents not only a lifeline for affected families but also underscores the growing potential of gene therapies to address previously untreatable genetic diseases.In another significant development, researchers have unveiled a groundbreaking study on an experimental cancer vaccine that has demonstrated efficacy in preventing tumor growth in preclinical models. Unlike traditional vaccines aimed at infectious diseases, this cancer vaccine is designed to harness the body's immune system to specifically target and destroy cancer cells. The study's results indicate that the vaccine was successful in generating a robust immune response, which significantly inhibited tumor progression. If these findings can be replicated in human trials, it could open new avenues for cancer prevention and treatment, particularly for cancers with limited therapeutic options.Shifting our focus to trends within the industry, there's an increasing emphasis on personalized medicine, reflecting a broader shift towards treatments tailored to individual patients' genetic profiles. This approach aims to optimize therapeutic efficacy while minimizing adverse effects by considering each patient's unique genetic makeup. Advances in genomic sequencing technologies and bioinformatics are driving this transformation, enabling more precise diagnostics and targeted therapies. As personalized medicine continues to evolve, it holds the promise of revolutionizing how diseases are treated and managed in clinical practice.On the topic of drug approvals, a new oral medication for type 2 diabetes has received regulatory clearance after demonstrating superior glycemic control compared to existing treatments. This drug belongs to a novel class of medications that enhance insulin sensitivity and reduce glucose production by targeting specific metabolic pathways. Clinical trials indicated significant improvements in blood sugar levels and overall metabolic health among participants. Given the global prevalence of type 2 diabetes and its associated health complications, such advancements are crucial for improving patient outcomes and reducing healthcare burdens.Lastly, focusing on collaborations within the industry, several prominent pharmaceutical companies have announced partnerships aimed at accelerating research in infectious diseases.Support the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we are diving into a series of significant breakthroughs and updates that are shaping the industry landscape.Starting with a remarkable scientific advancement, researchers have made headway in the development of a new class of antibiotics that shows promise against drug-resistant bacteria. This comes as a beacon of hope in the ongoing battle against superbugs, a problem that has been escalating over the past few decades. The new antibiotics work by targeting bacterial cell walls in a novel way, which may bypass the resistance mechanisms that have rendered many traditional antibiotics ineffective. This innovation could potentially extend the lifespan of existing drugs and provide new treatment options for infections that are currently difficult to manage. It's crucial to monitor how these developments will proceed through clinical trials and regulatory scrutiny, as successful outcomes could revolutionize our approach to bacterial infections.Shifting focus to regulatory news, the FDA has recently approved a groundbreaking gene therapy for a rare genetic disorder affecting children. The therapy is designed to target and correct specific genetic mutations, offering hope for families affected by this debilitating condition. This approval not only marks a milestone for personalized medicine but also sets a precedent for future gene therapies targeting other rare diseases. The implications of such advancements are vast, as they open doors to tailored treatments that address the root causes of genetic disorders rather than just managing symptoms. As we continue to explore the potential of gene editing technologies like CRISPR, it's important to consider both the ethical and logistical challenges that accompany these scientific leaps.In clinical trial news, a late-stage study has shown promising results for a new cancer immunotherapy targeting non-small cell lung cancer. This therapy leverages the body's immune system to identify and destroy cancer cells more effectively than traditional treatments. The trial demonstrated significant improvements in patient survival rates and quality of life, underscoring the potential of immunotherapies to transform oncology care. These findings add to a growing body of evidence supporting immunotherapy as a cornerstone of future cancer treatment regimens. However, it is essential to continue researching how these therapies can be optimally combined with existing treatments to enhance outcomes and minimize side effects.Turning our attention to industry trends, there is an increasing emphasis on digital health solutions in drug development processes. Pharmaceutical companies are integrating artificial intelligence and machine learning technologies to streamline clinical trials and accelerate drug discovery. These digital tools enable more efficient data analysis, patient monitoring, and predictive modeling, which can significantly reduce development timelines and costs. As this trend gains momentum, it will be important to assess how these technologies can be best utilized without compromising data integrity or patient safety.Lastly, let's discuss an interesting development in sustainable biomanufacturing practices. Companies are investing in greener production methods that reduce environmental impact while maintaining high-quality standards for pharmaceuticals. This includes optimizing energy use, minimizing waste, and incorporating renewable resources into manufacturing processes. As regulatory bodies increasingly prioritize sustainability, we can expect these practices to become more widespread across the industry.These stories highlight how innovation continues to drive progress within pharmaceuticals and biotechnology, offering new possibilities for treatment and care. As always, it's eSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the significant events of 2025, a year marked by pivotal scientific breakthroughs, regulatory changes, and industry trends that have reshaped drug development and patient care.One of the standout advancements was Novo Nordisk gaining FDA approval for an oral version of Wegovy, a glucagon-like peptide-1 (GLP-1) receptor agonist for obesity management. This marks a notable shift in treatment accessibility, as it provides an easier alternative to injectables for those managing weight and cardiovascular risks. This development could significantly enhance patient adherence and broaden access to this critical therapy.However, not all news was positive. Pfizer faced a challenging situation when a patient death occurred in the extension of their Hympavzi hemophilia study. Such incidents highlight the intrinsic risks of clinical trials, especially within gene therapy realms where safety monitoring is paramount. These events remind us of the delicate balance between innovation and patient safety in advanced biologic therapies.In legal news, Johnson & Johnson was ordered by a Baltimore jury to pay $1.56 billion in a talc-related cancer case. This ruling underscores heightened scrutiny on product safety and consumer protection within the pharmaceutical industry, potentially influencing future litigation and regulatory measures.Clinical trial outcomes also presented mixed results. Neurocrine Biosciences' Ingrezza did not meet efficacy endpoints in its phase 3 trial for cerebral palsy-related dyskinesia. Although it is approved for other movement disorders, this setback reflects the complexities involved in expanding drug indications. Such challenges highlight ongoing hurdles in translating preclinical successes into clinical realities.Despite geopolitical tensions, particularly between China and the U.S., Chinese biotech firms thrived, maintaining robust deal activity. China's continued growth as an innovation hub is driven by strategic investments and collaborations that bolster global drug development efforts, underscoring its increasing influence in life sciences.Regulatory landscapes also shifted with proposals from the Center for Medicare & Medicaid Innovation to align U.S. drug prices with international rates under Medicare Parts B and D. These proposed models could significantly impact pricing strategies and market dynamics within the U.S., requiring pharmaceutical companies to adapt while ensuring equitable access to medications.Ethical challenges surfaced as six individuals were charged with insider trading involving biotech stocks. Such incidents highlight the necessity for stringent ethical standards and regulatory oversight to maintain investor confidence and market integrity.Meanwhile, AstraZeneca's extended partnership with Niowave for actinium-225 supply reflects an interest in radiopharmaceuticals as targeted cancer therapies. This collaboration highlights the potential of radiopharmaceuticals in oncology, opening promising avenues for precision medicine approaches.As 2025 closes, it's clear that this year has been one of both triumphs and trials for the pharmaceutical and biotech industries. Scientific innovations like Novo Nordisk's oral GLP-1 receptor agonist offer new hope for patients, yet challenges such as clinical trial setbacks and legal battles indicate ongoing hurdles in drug development and commercialization. These developments will likely influence industry strategies and regulatory policies as we advance into 2026.The sustained momentum of China's biotech industry amid global trade tensions remains notable. This trend reflects China's strategic investments in biotech capabilities and its growing role in global markets despite geopolitical frictions.In clinical research, Hope BioscienceSupport the show

Julie A. Parsons, MD Haberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USAHow have programs adapted to the experiences from clinical trials? I'm just looking at SMA because we've had SMA. We've had onasemnogene around for the longest period of time. We want to always confirm a diagnosis and know that the patient is right. We do antibody testing for these disorders prior to delivering the AAV therapies. We have to know that the product that is incredibly expensive is handled appropriately by the institution. Dealing with the pharmacy, making certain that you handle the agent properly, patients need to be pretreated at this point with prednisone, and that really has to happen so that you know that they're ready for treatment, that they don't have any infections prior to treatment.Then we need to monitor and provide medication and follow-up afterwards. As I said, I think this is really, really important to make sure that you're connected well with the patient. If you live in an area as we do, that has a huge catchment area with patients that come from hundreds of miles away, sometimes they need to stay with us for a period of time, so that we can ensure the safety and follow-up of these patients after we deliver gene therapies.Again, a recurring theme is the patients that you're treating who are not in a clinical trial are not the homogeneous, well-selected patients. It's really all actors. The population that you're treating commercially is very different. We're now moving into treating patients with larger body masses and older ages. We don't always know, because those patients haven't really been included in the clinical trials. We don't really know what some of the effects are going to be with that group of patients as well.I am a neurologist. I am not an immunologist. I have had to learn a lot of immunology at this point, but it's still not sufficient. I think that we also need to reach out to our subspecialist colleagues who really do have more experience than we do to try to help us with some of these issues, because as we look at these viral vector capsids and the transgenes, we have to say, is there something that we can do to mitigate the immune response that we're seeing when we're giving massive doses of these agents and really taxing the immune system in our patients?Looking at possibilities, we give steroids, and that's really what we've done. That was what was done in the early clinical trials with MENDEL. It's like, okay, prednisone, that's all we have to do is we give steroids and everybody will be fine. That really isn't maybe the answer. As we have more information, we know that we're going to start with steroids, but we're really going to look at, is there a way to block both the B-cell response, the T-cell response? Is there something that we can do so that we don't have to sit on the edge of our seats and not sleep for months after we treat these patients?At least in a trial, was done looking at patients who were treated just with corticosteroids. Those patients had rapid increases in IgM and IgG. There's complement activation. Both the adaptive and the acute immune responses are triggered. That's really what we're doing as standard practice right now, but in the trial looking at treating patients and pretreating patients with rituximab blocking B cells and sirolimus and corticosteroids, then no significant change in IgM, IgG.Is that something that we should be doing? I think that some of the clinical trials that are being set up are looking at instituting some of these immune-modulating features to see whether or not their outcomes are improved. Can we do anything proactively to prevent our patients from having some of these very severe events or fatalities? I think that's really what we need to be looking at now. I think we are looking at that as a community, and to me, is a story that is still unfolding in terms of how we keep our patients safe.In the next part, Doctors Beggs and Parsons will discuss key issues on gene therapy development.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. In the ever-evolving landscape of pharmaceuticals and biotechnology, a series of strategic transactions and scientific advancements are reshaping the industry.BioMarin's acquisition of Amicus Therapeutics for $4.8 billion is a significant highlight, marking the company's largest transaction to date. This move signifies a strategic pivot towards enhancing its capabilities in the rare disease sector, leveraging Amicus's expertise and robust pipeline to potentially improve patient outcomes in this highly specialized area. This acquisition is expected to enrich BioMarin's portfolio significantly with promising assets from Amicus, reflecting a strategic shift under new leadership towards rare disease treatments.Regulatory affairs have seen considerable activity as well, with the FDA raising concerns over manufacturing practices at Catalent's gene therapy facility. These issues, documented in a Form 483 following inspections, particularly pertain to the production of Elevidys. Such regulatory scrutiny emphasizes the critical importance of maintaining compliance with manufacturing standards in gene therapy—a burgeoning field within biotech that holds immense promise for treating genetically-driven conditions.The FDA's oversight extends beyond manufacturing practices to advertising, as evidenced by an untitled letter issued to Bristol Myers Squibb regarding their Cobenfy TV ad. This action is part of the FDA's broader initiative to ensure that direct-to-consumer marketing materials accurately portray drug benefits and risks, thereby protecting public health.In another strategic move, Alvotech and Teva are gearing up for the 2026 U.S. launch of an Eylea biosimilar following a settlement with Regeneron. This development highlights the competitive dynamics within the biosimilar market—a segment poised for growth as patents on major biologics expire, offering more cost-effective alternatives and expanding treatment access.Meanwhile, Clovis Oncology has achieved a milestone with Rubraca, which transitioned from accelerated approval to full FDA endorsement for prostate cancer treatment after five years. This progression underscores Rubraca's demonstrated efficacy and safety profile in addressing advanced prostate cancer—a notable achievement amid an increasingly competitive oncology market.Policy changes proposed by Health and Human Services Secretary Robert F. Kennedy Jr. could have profound implications by disrupting funding streams for hospitals providing gender-affirming care to minors. The potential impact on healthcare providers and patients who rely on these services is significant.Turning to clinical trials, Daiichi Sankyo has seen success with Enhertu receiving FDA approval for first-line HER2-positive breast cancer treatment. Nonetheless, challenges persist as a separate phase 3 trial for another antibody-drug conjugate was paused due to unexpected patient deaths. Meanwhile, Takeda plans to seek FDA approval for its TYK2 inhibitor following successful phase 3 trials in psoriasis—indicating promising potential in autoimmune disease therapies.Strategic shifts are evident across organizations as well, highlighted by Kathy Fernando's departure from Pfizer to join Replicate Bioscience as Chief Business Officer. Her new role focuses on advancing Replicate's self-replicating RNA technology platform—an area gaining traction due to its implications for vaccine development and therapeutic applications.On the clinical trials front, Altimmune reported encouraging results from a 48-week study on metabolic dysfunction-associated steatohepatitis (MASH). Their GLP-1/glucagon dual receptor agonist demonstrated sustained weight loss and improvements in non-invasive liver fibrosis measures—offering new hope for MASH patients who face limited treSupport the show

Alan Beggs, PhDDirector of the Manton Center for Orphan Disease ResearchSir Edwin and Lady Manton Professor of Pediatrics, Boston Children's HospitalHarvard Medical School, Boston, MA, USA Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USAThe ASPIRO Clinical Trial is on clinical hold since September 2021. In this part, Doctors Beggs and Parsons will discuss key issues on gene therapy development.Question: Is there a standardized immunomodulation regimen being considered for gene therapy?Julie A. Parsons, MDAs I mentioned, right now, I think there are a number of different concepts that are being utilized. We don't really have a recommended standard regimen at this point. There are a number of different trials that are ongoing looking at trying to answer this question. In some of the clinical trials, there is an immune modulating regimen that is being put in place but being looked at. There isn't anything that we have as a standard at this moment for all gene transfer therapies, but I'm hopeful that we will come up with something that really makes sense in each patient population as we go forward with specific gene transfer therapies.Question: What are the long-term implications, safety and efficacy of a one-time gene therapy in pediatric patients with neuromuscular diseases?Alan Beggs, PhDOne question is the efficacy. For example, Donovan Decker's story, he had an experimental treatment of one muscle. It was a phase one safety trial, and he knew that nothing was going to come of it in terms of direct benefit to him. As a result, though, 25, 30 years later, he still has a tighter against AAV vectors. He's not a candidate for gene therapy under current protocols, although there's a lot of work going on to redosing. But for now, it's a one-time treatment. What you get is what you get, and there's not a chance to go back and do it again.The other question is durability. We really don't know about the long-term durability for these treatments. I should say that, for example, in the studies that we did, David Mack, who's here in the audience, managed a dog colony for a dog model of excellent tubular myopathy. Those animals lived 10 years in a... We never used the C-word, but they were cured. They were healthy, happy, normal dogs who would have had to be put down at 6 months of age otherwise. And then, as we heard, I'll let you talk about the concern for unanticipated SAEs as time goes on, but I think there's other aspects we need to think about.Julie A. Parsons, MDYeah. I think that this is really the key question that all of us are going to need to help answer over the next several years. Efficacy, we're looking at outcomes, and outcomes come in a variety of flavors. I think we do a decent job with motor outcomes. We don't do a decent job with some other outcomes. I think we need to look more broadly in terms of what we mean in terms of beneficial outcomes and really take some of those cues from the patients themselves about if these are efficacious treatments, because, again, the risk is high as we deliver these agents, and we need to know that it's worth it to the patients and families.In terms of safety, we're working on it. There are all sorts of things that are coming forward as issues with these patients. I think that collectively as a community, that our responsibility is to follow patients for the long term. There are lots of registries and outcome studies. We're not very good as a community about reporting adverse events to central groups. We're not great about broadcasting that to each other in real-time. I think those are things that we really need to work on as a community in terms of helping with the safety issues so that we all have a communal better understanding of what some of those issues are.

Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USANow, with our collective experience, we can at least put together the information that we have in terms of what can we expect and what's the timeline that we expect in terms of our patients having reactions. I will tell you, and I've said this multiple times, when I deliver a gene transfer therapy, I hold my breath for 2 months. Now, maybe it's going to have to be extended to a year, but it's typically at least for 2-3 months. It's like, okay, what's going to happen? You sit on the edge of your seat on pins and needles, going, "Is this kid going to be okay or not?" I think that's the appropriate response to have in terms of the light of things that have happened over time. We have to be really careful.We have a little bit of a framework now to say, when do we need to be really excited? We know that our patients, most all of them, are going to develop a transaminitis, and that ends up happening early on, but we get a couple of peaks. We get really excited that the 4-8 week time point with transaminitis looking for liver failure.The cholestatic liver disease that happened in the patients with X-linked MTM happened a little bit later, so Week 2, all the way out to six months afterwards. The acute cardiomyopathy a little bit earlier, so we're looking a little bit earlier for that effect. TMA, usually the end of the first week to about 2 weeks is when we would expect that to come in. Then the transgene-related myositis and immune-mediated myocarditis, weeks, maybe 2 to a couple of months.How do we adapt our gene transfer programs to the clinical trial experience? I think that there are a couple of points that are important. One is that the outline that I showed you, there are some disease-agnostic issues that come up with transaminitis, with TMA. I think there are some final common pathways related to the immune responses that we see with these patients. Then there are going to be some disease-specific disorders that are going to come up with each of these therapies and agents.We need to have good communication, honestly, in real-time. I still don't know that we have a good mechanism for that as a community, but to share these adverse events that come up so that we can all learn as a collective about what to expect, what to anticipate, and how to best take care of our patients. We know now how we need to monitor patients closely from a laboratory standpoint, from a clinical exam standpoint, and we really need to work on how are we going to mitigate some of these risk issues that we have with these patients.I think the collaborative aspect, particularly at meetings like this, is important. Last year, for the people that were at MDA, you remember that we really spent a lot of time looking at gene transfer delivery. Many of us got together as providers and actually met together to say, "Is there something that we can think about in terms of best practice or consensus in terms of how we would want to manage patients or how we'd want to share information?"Now, actually, on the MDA website, we really do have some guidelines, and there will be a publication coming out shortly that we'll have this available to everybody again. It's not necessarily the right answer, but it's at least from a collective experience, what's the best way that we can go forward? Some of the suggestions were that the adverse events right now, we can put them into some a predictable timeline, but we don't really know all the risks at the time of dosing.We know that gene transfer therapy can be safe for the right patient at the right time for the right disorder. That's really what we want to do. There's a Neurotherapeutic window between efficacy and toxicity. How are we adjusting that? What are we working on to make sure that we're getting that right? The preclinical data is helpful, but it's never the full story. Any time we go from a homogeneous population that we see in a clinical trial to a heterogeneous population, as we throw this out to the world, we're going to have new issues that arise, and we need to be aware and ready for those.We want to be able to predict what happens, but we can't always do that. Then follow-up is so important. The post-marketing study, sharing adverse events, sharing experiences, I think, is really important as well. Clinicians really should be familiar with this entire field before ever delivering gene transfer therapy. I don't think that every site should be delivering gene transfer. I think that from an institutional standpoint, you need to be ready. You need to have a team who knows what they're doing and knows how to handle the issues and the problems, or you need to have lifelines set up in advance if you're going to deliver these treatments.

Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USAThe gene transfer trials for musculoskeletal disorders, if we look at musculoskeletal and neurologic disorders, we really do have the highest success rate in terms of treatment, but we also carry the highest incidence of treatment-emergent severe adverse events. And why is that true? Yesterday, when we were hearing about Donovan as well, we looked and said, When the first gene transfer therapies were started, he had a single muscle that was injected.When we look at Luxturna, we injected the retina. Now, what is happening with these disorders is that we're giving these huge, massive doses of viral vector to patients. There haven't been a lot of gene transfer therapies that have reached the market. But you saw yesterday, so many gene transfer therapies being worked on, but there are very few that have actually come to market. There are a couple of reasons for that.One is with the indications that we have, we know that the musculoskeletal disorders are most likely to achieve benefit, but there are the high risk of severe adverse events. Route of Administration, IV, for most of our disorders is the way we're going. We may end up having some Intrathecal therapies as well that are coming on board, but right now it's IV, and that means, a huge dose of this viral vector and antigenic risk that is being administered.In the vector design now, we actually have more specific vectors as well as promoters that are being utilized to really target specific tissues, so that we're able to focus in a little bit more on the tissues that we want to have affected. And then the dose has gone from these little tiny local injections to really systemic, much broader. And now our patients, are larger. So we're giving a viral genome per kilo dose that is just massive as we look at that.Then there really are challenges in terms of the translation of clinical trials to commercial treatment with these agents. And we don't always know, we're not always great when we do tests in clinical trials in small populations, about when that's broadened to the commercial availability and we hit larger heterogeneous populations.There are safety issues arising from these therapies, and I think that we have some experience now, certainly with the three diseases that I mentioned at the beginning, in terms of collecting some data and information to have a little bit more of an idea what to expect. Although to me, the recurring esteem is always, expect the unexpected. Because we still are learning about this. Hepatotoxicity. We know that transaminitis is something that we see in almost every gene transfer therapy that has been delivered, and we have to watch really, really closely and follow our patients closely for this. We also have to select patients that we don't think have risk for additional liver injury or underlying liver pathology, because as we found out in the XLMTM boys, we missed that. Thrombotic Microangiopathy. We look at this disorder. We've had deaths in SMA from TMA. We have Duchenne patients that have had TMA.This is scary because as many of us as clinicians who have treated patients, you know that we end up getting thrombocytopenia. So is that it this time, or are they going to be fine, or the platelet is going to go back to normal? This is another one that we have to watch really, really closely for. Cardiac Toxicity. We have had cardio myositis. We've had deaths from cardiac toxicity.Something really, really important for us to think about. In little kids, vomiting could be a sign of cardiac myositis. And for most of us who've treated patients with gene transfer therapy, what's one of the first issues that you get?You get nausea of vomiting, they don't feel good. So is that myocarditis or is it just a standard side effect that we're seeing with treatment? Importantly, as we discovered, there actually can be an immune response to the transgene. It's not just the viral vector capsid, it's actually the transgene as well. That was discovered in patients who were treated for Duchenne. So that's a really important thing in terms of looking now at what's our patient's selection and how do we pick the right patients.Next part, Dr. Parsons will discuss understanding and preparing risk factors associated with AAV gene therapies.

Julie A. Parsons, MD Haberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USAAs we talk about the gene transfer therapies and the modalities that we have to use, it's really interesting. Yesterday, with our keynote speaker, you could see this logarithmic growth of the use of gene transfer therapies for these disorders. If you look at the Venn diagram, you can see that really 27% almost of gene transfer therapies that are used are in musculoskeletal and neurology. For many of us as neurologists, we also take care of metabolic disorders.We really own right now this landscape, and of course, our two approved modalities are Onasemnogene and Delandistrogene. We're going to look at three different disorders, monogenic disorders, monogenic diseases, to typify what we look at in terms of some of the risks and benefits of these treatments. SMA, Duchenne, and X-linked myotubular myopathy are all rare disorders. They're all diseases that have a high unmet medical need and a significant disease burden.I think they're all good in terms of typifying where we are clinically with these disorders. The first question is, is it worth it? Are these effective treatments? We know from looking at the information about SMA that just looking early on, we know that if we treat kids early, that we do see a marked improvement in motor scores for kids that are treated early with Onasemnogene.In Duchenne, we have information that there is at least some improvement in the 4-5-year-olds in terms of motor skills treated with Delandistrogene. In terms of X-linked MTM, which was a very dramatic improvement, you could see that for boys who were basically traked, vented, and had no mobility, the bottom line, the blue line, is actually looking at ventilator dependence. Are they effective? Yeah, they're effective, but then we have to say, okay, what's the downside?The downside is that there's tremendous risk associated with treatment with these agents. If we really look at the sobering facts, we know that with SMA, there have been deaths, there have been fatalities related to thrombotic microangiopathy to patients who have liver failure, a couple of patients have died. With Onasemnogene, this is 4,000 plus doses that have so far been given. With Duchenne, unfortunately, many of us got the letter yesterday talking about an additional death in a patient treated with commercial Delandistrogene.We also know with some of the other agents, like fordadistrogene, patient died of heart failure, cardiac arrest, another patient who had acute respiratory syndrome with pulmonary edema. Again, we look at this and say this is significant. With X-linked MTM, as Alan said, there were some unanticipated deaths, four deaths from patients who ended up having cholestatic liver diseases that really wasn't anticipated prior to the patients being treated with the animal models and all that we had. Then many of you have heard about the patient with Rett syndrome who had a systemic hyperinflammatory syndrome. Again, these are rare disorders. They have a high disease burden, but the risk of treatment is significant.In the next part, Dr. Parsons discuss factors impacting safety and efficacy of AAV-mediated gene therapies.

Alan Beggs, PhDDirector of the Manton Center for Orphan Disease ResearchSir Edwin and Lady Manton Professor of Pediatrics, Boston Children's HospitalHarvard Medical School, Boston, MA, USAThe challenges that you've heard about are real. Some of them I think we could have foreseen others. There was no way to know until we actually started treating patients in clinic. But we now know that there are immune responses and also responses just to the viral load. As Julie mentioned, we're giving massive doses to these patients on the order of one times ten to the 14 viral genomes per kilogram.Think about the fact that when these capsids are manufactured, there's a certain percentage of empty capsid. The amount of protein that's being delivered to these patients can be massive. One of the approaches to mitigate some of the risk would be to lower the dose. While early studies demonstrated that in order to get adequate delivery to skeletal muscle, you need to give these very large doses. But what if we could engineer a viral capsid that would be potent at lower doses?There has been quite a bit of research in this area that's ongoing, and some new next generation vectors that are just starting to enter the clinic. In particular, there are a class of Myotropic viral vectors or capsids so-called RGD vectors. RGD refers to arginine, glycine, and aspartic acid, which are three residues which, when present at a particular point in the viral capsid proteins interact with integrin receptors that are specific for skeletal muscle. These viral capsids home to skeletal muscle and can deliver their genetic payload at much lower doses. There was one group of these developed in Germany by Theo Grimm's lab.These were the so-called AAV Myos, and simultaneously in Boston at the Broad Institute, a group of capsids was developed that were called Myo AAV. These were both based off of an AAV nine backbone. It's basically an AAV nine legacy vector with these three amino acids changed. Now Solid Biosciences also has their own independently derived vector that I believe is also an RGD vector. These vectors give us the potential then for more efficient and specific delivery to muscle cells.They may or may not target the liver depending on the particular virus. Some of them the risk to the liver is mitigated by delivering a lower dose. You can also develop these vectors in a way that will be liver targeted, that specifically less of it gets delivered to the vectors. These would be really, in my mind potentially third generation vectors.Strategies, there are a number of strategies. You heard about the immunomodulation regimens. I just talked about optimizing vector design. Also, Doctor Parsons mentioned earlier the fact that where you deliver so zolgensma is delivered Intrathecally. We get it to the place we need it, and we're less likely to have off target effects through other tissues.Then improved manufacturing is very important. I mentioned the fact that every viral preparation contains empty capsids. There are ways to minimize the production of empty capsids, and also effective ways to filter out and remove those empty capsids. This is actually a very important aspect that is being developed further by the CMO community. Then in summary, I think it's important to take a holistic approach when we're thinking about the development of AAV based gene therapies for neuromuscular disease.It starts from the fact that for any given disease we're interested in, we need to define the genetic etiology. Since these are gene directed therapies. We need to pay careful attention to the preclinical animal models. How accurately do they really reflect the human condition? Or are there potentially responses in our human patients that we haven't experienced in the animals? It's important to understand the natural history and the patient population.Recognize that there's extensive heterogeneity, not just in age and severity, but also potentially in underlying susceptibilities in our patients. We have a group of toxicities that we know about and can anticipate. But as Julie was saying, you need to be really careful and think about any potential unexpected SAEs. And then finally I mentioned the manufacturing aspect, the development of newer vectors and quality control aspects that go into making a safe and effective therapeutic.In the next part. Doctor Parsons will discuss clinical safety and efficacy observed in AAV mediated gene therapy programs in DMD, SMA, and XLMTM.

Alan Beggs, PhDDirector of the Manton Center for Orphan Disease ResearchSir Edwin and Lady Manton Professor of Pediatrics, Boston Children's HospitalHarvard Medical School, Boston, MA, USA Julie A. Parsons, MDHaberfield Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyUniversity of Colorado School of Medicine, Children's Hospital ColoradoAurora, CO, USADoctors Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight part podcast series. This is derived from the symposium that was presented at the MDA 2025 conference in Dallas, Texas, in March 2025 and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established. In contents of this podcast, shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The ASPIRO clinical trial is on clinical hold since September 2021.In this part, Doctor Beggs will provide an explanation of AAV-mediated gene therapies.Alan Beggs, PhDAAV vectors, which I'm going to be talking about more today, or Adeno associated viral vectors are small viruses. Their DNA gets delivered into the cell and remains extrachromosomal. There are very rare occasional integrations, but the risk of oncogenesis as a result is significantly lower as a consequence of remaining extrachromosomal, though, we do have to think about what happens as the cells divide and potentially the durability of treatment is more limited.There have been a lot of movement and development over the years, starting back in the 1980s when the first AAV genomes were isolated and sequenced. This led to a development of methods to produce recombinant AAVs that would lack the genes necessary for viral replication, but contain a therapeutic gene you wish to deliver. Through this, the structure of AAVs have been developed. There have been isolation of a number of naturally occurring variants. You've heard of AAV8, AAV9, also RH 74, derived from a rhesus monkey for the RH. These have all been used in clinical trials. Then at the end I'll talk a little bit about directed evolution methods to actually engineer capsids with particular properties that are beneficial.Throughout this we've identified some of the issues that arise in this. It was initially thought that AAV vectors were non-immunogenic, but in fact there are immune responses not just to the viral payload to the therapeutic protein, but also to the viral vectors, and you're going to hear about that from Doctor Parsons. Over time, as we've come to understand these challenges, we've also been developing approaches to mitigate them. In terms of clinical trials and treatments, the very first studies were done back in the 1970s.By the early 2000, the very first clinical therapeutic was approved in China. It was actually an oncolytic virus carrying a p53 gene to treat head and neck cancers. By now there are over 40 approved treatments for various types of AAV delivered gene therapies. Of course, the ones we know a lot about are Zolgensma, which was approved in 2019, and Elevidys, which was approved last year. A number of challenges and then also a number of approaches to overcome those challenges. First of all, the preclinical data are not always sufficient to predict the response of a human patient.For example, in X-linked myotubular myopathy we had mouse and dog models that exhibited a myopathy but nothing else, and yet when we treated human patients, we discovered that patients with X-linked myotubular myopathy actually had a previously only poorly recognized hepatopathology that led to potential liver consequences following gene therapy. The animal models don't always predict the clinical outcome in humans.Also, we have small disease populations. These are rare diseases. It's important to understand the natural history of these diseases, understand the heterogeneity among the clinical population. It's very important to engage with families and with patients and communities, understand who might be at increased risk to treatment with one of these. This feeds into safety considerations. We need to think also about some of the immune responses. I think we're starting to learn, for example, with the gene therapies for Duchenne, and we know this from SMA that some patients get into trouble and others don't. We need to understand why that may be, and we don't know about the long term effects. This has been very recent.

In this episode of BioTalk Unzipped, hosts Gregory Austin and Dr. Chad Briscoe sit down with Glafabra CEO: Dr. Chris Hopkins, geneticist, biochemist, and biotech entrepreneur, to explore the science and strategy behind next generation cell-based gene therapies for rare diseases.With more than 25 years of experience spanning gene augmentation, rare disease biology, CRISPR licensing, and biotech formation, Dr. Hopkins shares how autologous, ex vivo engineered cell therapies may overcome key limitations of current enzyme replacement and viral gene therapies, particularly for Fabry disease.The conversation dives deep into: • How lentiviral gene augmentation in patient derived cells enables sustained enzyme production • Why redosing matters and where one time AAV therapies fall short • The scientific rationale for early intervention, including potential newborn treatment • Differences between autologous and emerging allogeneic approaches • Regulatory pathways for rare disease therapies and recent FDA developments • The role of non animal models in translational research • Montana's early access therapy law and its broader implications • Building biotech platforms amid a challenging funding environmentTopics include cell based gene therapy, Fabry disease, lentiviral vectors, stem cell engineering, rare disease drug development, regulatory science, and translational medicine. Subscribe to BioTalk Unzipped for in depth conversations with the scientists and leaders shaping the future of biomedical innovation.00:00 - Intro00:53 – Welcome to BioTalk Unzipped, Guest intro: Dr. Chris Hopkins02:10 – Guest charity: Environmental Defense Fund03:12 – His journey into rare-disease therapeutics and Glafabra05:58 – Discovering a new enzyme-deficiency therapy 06:39 – Current standard of care 07:42 – How the new autologous cell therapy works09:40 – Treating patients earlier (even newborns)10:33 – Emerging therapies - AAV gene therapy vs. cell-based therapy12:16 – Long-term results & repeat dosing14:30 – Future plans: T-cells & allogeneic approaches18:08 – New News: FDA resubmission for rare disease20:00 – Navigating FDA pathways22:06 – Non-animal testing & alternative models25:50 – Montana's early-access therapy law & medical tourism29:03 – Could other states follow?31:31 – Biotech's current funding challenges33:46 – New News: Gene therapy trial saves 4-year-old37:09 – Long-term vision for expanding therapies39:53 – Personal segment: outdoor life & skiing44:43 – Guest question on international trade Dr. Christopher Hopkinshttps://www.linkedin.com/in/christopherehopkins/ Glafabra - https://www.glafabra.com/ Environmental Defense Fund - https://www.edf.org/ Dr. Chad Briscoe

Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfesor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this 8-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th-7th 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Roberts will discuss immune responses and other safety concerns related to gene therapies.Mark Roberts, MDUndoubtedly, the immune system is a major issue in these patients. It would be fantastic if we could immunotolerize our patients and indeed prevent the rejection of the therapy. We've talked about the fact that these are viral vectors and of course there may be high seroprevalence of antibodies to these viral vectors, and it's very important in the pre-screening of patients who might be eligible to understand that at the beginning. These of course can have developed over the years and of course can be part of immunological memory and therefore extremely difficult and probably impractical to actually shift.On giving the treatment though as I think we're all aware there is this problem of the innate immunity and potential therefore for acute toxicities and then a learned or adaptive response with cytotoxic T cells and antibodies which may of course become high tighter neutralizing antibodies and potentially antibodies not only against the viral vector, even the functional protein, even the transgene are all theoretical possibilities with time. The capsid, the transgene, and even the protein product can all potentially induce an immunological event. Of course, all of these would lead to both potential patient changes and then a lack of efficacy of the treatment.Indeed, there have been some serious and indeed fatal problems in the gene therapy development program as I think we're all aware. Though many of these are thankfully been overcome. Spinal muscular atrophy has a gene therapy which is licensed, but there were early patients who actually had significant problems. A patient of just 6 months of age who developed kidney failure, two other patients who actually developed liver failure.In Duchenne muscular dystrophy, a very common condition, again there were significant issues and crucially in these patients who all have cardiomyopathy, it was heart failure and cardiac arrest that were big concerns and pulmonary edema and this was seen even with a CRISPR-based technology and is perhaps is best known but has been addressed the excellent myotubular myopathy patients, four patients died and crucially quite a long time after the gene therapy emphasizing the need to monitor these patients extremely carefully and these patients died of cholestatic liver failure albeit that they had a degree of liver dysfunction.That's changed our screening of course of patients, we're now all looking in myotubular patients for liver involvement and Rett syndrome as well. Now these immunoprophylaxis treatment regimes to hopefully try and reduce the immunological reaction against the gene are certainly evolving.This is just a summary of some of the other immunosuppressive regimes used in other disorders, for example, spinal muscular atrophy, but Pompe and MPS as examples of LSDs. Certainly these regimes will continue to evolve and are going to be very important in seeking to make sure that these treatments are effective. It reminds me somewhat of what's happened with enzyme replacement therapy that the use of these immunological strategies in infants has revolutionized the utility of those treatments in early patients.In the next part, Dr. Roberts will discuss lessons learned from gene therapy trials.

Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs.Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lessons learned from gene therapy trials.Mark Roberts, MDWhen we think about the challenges of actually doing clinical trials with these gene therapies, there's a huge development stage in terms of picking the right viral vector with the right surface receptor. That's a major piece of work. That can often take years. The preclinical work is obviously very important as indeed is understanding the natural history because it's really not practical to do placebo-controlled trials of gene therapies.In contrast to other studies, when we turn to phase 1 and phase 2, you'll notice that the patient numbers are often quite small. One is having to think carefully about surrogate measurements of response. Especially when in phase 3 studies, we may be thinking about withdrawing the existing, for example, enzyme replacement therapy because we believe the gene therapy will then be effective.That's just a few snapshots of where we've come and there's a lot more work to be done.In the next part, Dr. Longo will discuss the current treatment landscape and limitations in lysosomal disorders.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss ongoing gene therapies in lysosomal disorders.Nicola Longo MD, PhDI'm going to present to discuss some example of ongoing gene therapy for lysosomal disorder. There are gene therapy in development for both Fabry disease and some of this involve ex vivo gene therapy, many others involve systemic administration with an AAV, Gaucher disease type 1 that affect the periphery, and Gaucher disease type 2, where the replacement should occur within the central nervous system because this condition affects the brain. There is already one approved gene therapy for lysosomal disorder, which is for the early onset metachromatic leukodystrophy. This has been approved both in Europe and now even in the United States, which consists of ex vivo gene therapy with the administration of an extra gene that restore the function of the defective enzyme. Now there are many others that are ongoing for the same indication. There are gene therapy programs for GM1 and GM2 gangliosidosis, and at least one for Krabbe disease. It is important to know that some of these condition are actually included in the recommended uniform screening panel. Basically, we would have access to patients in a timely manner for some of these conditions. Then there are several gene therapy under development for the mucopolysaccharidoses, including MPS-IH, MPS-II, MPS-IIIA and MPS-IV.There are different type of lysosomal disorders, the one caused by mutation, integral membrane protein, not enzyme within the lysosome, but protein that are present on the membrane of the lysosome. This gene therapy that have been tested, it is for cystinosis, that it is caused by a defective lysosomal and for Danon disease, which is caused by a deficiency of an integral membrane part. Finally, one lysosomal disorder, which obviously seems a metabolic condition, but it is really not, is glycogen storage disease type 2 or Pompe disease, in which there is the intralysosomal accumulation of glycogen. There are several ongoing clinical trials to try to correct the problem in this condition.Now, I'm going to discuss some of the most advanced program in the lysosomal storage disorder. This include one for Fabry, which is on an accelerated approval pathway with phase 1 and 2 data, one for Gaucher disease type 1. Obviously, I'm going to discuss the one that has been already approved for metachromatic leukodystrophy. There is one for Hunter syndrome, and the difference of the one for Hunter syndrome, it is an example of the direct administration of gene therapy within the central nervous system.Finally, there is one ongoing for glycogen storage disease type 2 or Pompe disease in adult patients. In gene therapy for metachromatic leukodystrophy, it was the first gene therapy approved for lysosomal disorder in human, and this requires harvesting the CD34 cell from affected patient and then introducing the [inaudible 00:04:32] gene back in this cell, and then placing them back inside the patient again. This has been very effective in patients who were treated early, and obviously, the treatment needs to occur before there is irreversible brain damage in this patient.In the next part, Dr. Roberts and Longo will discuss treatment with gene therapies.

Nicola Longo MD, PhD, and Mark Roberts, MDDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Doctors Roberts and Longo will discuss treatment with gene therapies.Question: Can one administer AAV-mediated gene therapy repeatedly?Mark Roberts, MDI think the traditional view would have been no. One can think of gene therapy as a silver bullet. Hopefully, it will reach its target. But if it's not effective, that bullet has been shot, the immunological response has occurred, and it means redosing, at least with that particular vector, may become difficult. But this situation is changing and evolving as we have better understanding of immunological modulation for repeat testing. We were discussing this yesterday evening, weren't we, Professor Longo?Nicola Longo MD, PhDCorrect. Basically, the current AAV-based gene therapy cannot be readministered. It is either effective, or it doesn't work. The other thing is that even though in theory, one could utilize a different AAV vector with different immunogenicity, there is many times cross-reactivity among the different adenovirus, adeno-associated viruses. Now, there are approaches in animal models in which you give a strong immune suppression to prevent the creation of the immune response against the adeno-associated virus, and at least in the animal model, it has been possible to give some of the gene therapy repeatedly.The second approach that is being tested is with gene correction therapy, in which by using an RNA guide and the CRISPR/Cas9 system delivered by lipid nanoparticles, you basically correct some of the effective genetic information. Obviously, since this is done by lipid nanoparticles and not by an AAV, the immunity that you create is really not there. You can give this one repeatedly, and in theory, it can be given more than one time. But again, you are absolutely correct. The current gene therapy cannot be given twice, and either it works or it doesn't work.Question:vWill gene-therapy-treated patients be able to go back to the standard of care or enzyme replacement therapy?Mark Roberts, MDI think when we're talking to patients about the potential benefits of gene therapy and the amelioration of the requirement to have these infusions on a regular basis of ERT, the hope is that will work, but they need to be reassured that we can potentially go back to the ERT. Gene therapy is an important treatment, but we don't know the destination of the patient at the beginning, and we have to make it available to them to go back to ERT.One of the crucial questions, of course, though, is the basis of the immunological reaction that perhaps prevented the gene therapy being effective. If it's against the viral vector, well, okay. If it's against the transgene, not great. If it's against the functional protein, that becomes more difficult. It is somewhat, I think at this time, to be fair to say to patients, think of gene therapy as a trial treatment. It is somewhat a leap of faith and an important observation, of course, for the patient community, but just be aware there may be downsides.Nicola Longo MD, PhDThey totally agree with Dr. Roberts. In general, they should be able to go back to enzyme replacement therapy if the gene therapy is not effective. However, what we are starting to appreciate is that we need to understand the immune response, not just to the enzyme replacement therapy, but also to gene therapy. What this field is doing is forcing geneticists to deal with the immune response. I feel that historically has not been dealt together. The two things need to be integrated. The advantage of the gene therapy is that the protein is produced endogenously. There should be the development of some degree of tolerance with time in the body towards the endogenous continuous production of a protein.Now, will that happen all the time? I still do not know. Again, we need to understand much better what is the integration of the immune system with the response to gene therapy in the ongoing clinical trials.

In this episode, we sit down with Dr. Jean Bennett, Professor Emeritus of Ophthalmology at Penn Medicine and a true pioneer in gene therapy research. Dr. Bennett led the team that developed the first FDA-approved gene therapy to treat inherited blindness, transforming the lives of patients with retinitis pigmentosa and other retinal diseases. Her groundbreaking work spans over three decades and has opened doors for gene therapy applications far beyond ophthalmology. Dr. Bennett shares her remarkable journey from a curious child in New Haven, Connecticut—who chose seeds over candy at age four—to becoming a world-renowned scientist. She opens up about the challenges she faced as a woman in science, including being advised to leave her field entirely when seeking tenure at Penn. With candor and humor, she discusses how mentorship from previous Girl Power Gurus guest, Dr. Virginia Lee, helped her persevere, the emotional eureka moment when blind dogs first regained their sight, and what it felt like to witness blind children see for the first time. Dr. Bennett also offers invaluable advice for young women pursuing STEM careers, emphasizing the importance of finding work you love, building collaborative teams, and never giving up on your dreams despite the obstacles. This is a must-listen for anyone interested in science, medicine, or the power of determination.

In this episode, we discuss promising results of gene therapy, administered suprachoroidally, for the treatment of nonproliferation diabetic retinopathy without macular edema with Charles Wykoff, MD, Ph.D., Retina Consultants of Houston.

The company has also fully enrolled the Phase 2/3 VISTA clinical trial for its XLRP gene therapy.

In the final episode of 2025, the GEN team came together to reflect on some of the stories over the last year that they each found impactful. Some of the stories were covered in GEN's December issue, but our editors took the time to discuss some topics in a bit more depth during the podcast. The discussion began with an update on the progress of AI use in drug discovery. Cell and gene therapy was a big topic with the positive outcome for Baby KJ, while Sarepta dealt with a rollercoaster of a year with their DMD treatment, Elevidys. Research and biotech in Africa are growing and the discussion ranged from challenges to promise in research and development on the continent. This year's NIH budget cuts and impacted institutions rounded out the conversation.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, Phd, Kevin Davies, Phd, John Sterling, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base: GEN Editors Reflect on Six of the Biggest Stories of the YearBy Alex Philippidis, Savannah Wiegel, Fay Lin, PhD, Kevin Davies, PhD, Uduak Thomas, and Julianna LeMieux, PhD, GEN, December 4, 2025StockWatch: Sarepta Says Deaths of Two Elevidys Patients Posted on FDA Database Unrelated to Treatment By Alex Philippidis, GEN Edge, August 10 StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT Stocks By Alex Philippidis, GEN Edge, August 3 FDA Probes Death of Brazilian Boy Linked to Sarepta's Elevidys GEN Edge, July 27 Second DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, June 15 DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, March 18From The Editor in ChiefJohn Sterling, December Issue of GEN Gene Therapy in AfricaThe State of CRISPR & Genome Editing Summit, June 11, 2025TOP 50 NIH By Alex Philippidis, GEN, December 4, 2025 FDA Cell & Gene Therapy roundtable: Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions” By Alex Philippidis, and Kevin Davies, PhD, GEN Edge, June 5, 2025Scientists in NYC Rally to Defend and Stand Up for ScienceBy Corinna Singleman, GEN, March 10, 2025Touching Base Podcast Hosted by Corinna Singleman, PhD Behind the Breakthroughs Hosted by Jonathan D. Grinstein, PhD Hosted on Acast. See acast.com/privacy for more information.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established, and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lysosomal disorders and the potential for gene therapies.Mark Roberts, MDI'm going to give an overview of what is gene therapy, emphasizing the current challenges and the development issues and needs that there will be as we try and enable gene therapy for our patients, particularly those with lysosomal storage disorders.I'm going to try and make a case for why lysosomal storage disorders are an extremely good group of conditions for the potential benefits of gene modifying therapies. Firstly, whilst we all recognize that these conditions are inherently individually rare, they're certainly severe. Collectively, with over 70 LSD disorders, 1 in 5,000 may be afflicted by these conditions ultimately in their life and can be detected, for example, by newborn screening programs.Secondly, there's certainly a significant clinical burden with these patients with the current standard of care, so a large unmet need exists. Existing enzyme replacement therapies have undoubtedly changed the natural history of many of these conditions, but there are limitations and often initial benefits and later deteriorations.Unfortunately, for most lysosomal storage disorders, it's only symptomatic treatments and indeed, care that is available for these patients with no specific treatment. Thirdly, these conditions are extremely well-characterized, monogenic singleton and problems of inborn errors of metabolism. We know the functional protein that is deficient in these conditions. Because of that, and knowing that these are critical for lysosomal function, and using preclinical models, we can model the potential benefits of gene therapies very well in a number of systems, including, of course, soon, muscle chip experiments as well.Finally, with these conditions, they may potentially be really useful targets whilst not perhaps curing the condition, at least ameliorating the phenotype, and enabling the addition of other treatments as well, potentially. I've noted, some of these therapies can be directly delivered to certain tissues, so muscle tissue, which is my main interest, but also, crucially, the central nervous system, which is very important when we consider ameliorated phenotypes, for example, treated by enzyme replacement therapy, but where the children who become the adults have significant learning disability as a major component to their problems.In the next part, Dr. Roberts will discuss vectors, different strategies, modes of administration, and targets in gene replacement therapies.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the exciting, yet challenging, landscape of gene therapies and their potential to revolutionize healthcare. Recent scientific advancements have pushed the boundaries of what's possible, offering potential lifetime cures for diseases once considered incurable. However, this breakthrough comes with a significant economic caveat: the staggering cost of these therapies, often ranging between $3 to $4 million per patient. This price tag presents a formidable challenge to current healthcare infrastructures.The disconnect between these innovative treatments and existing payment systems is evident in what industry experts term the "$4 million payment problem." Therapies like Lenmeldy and Hemgenix highlight this issue. Lenmeldy, for instance, can prevent metachromatic leukodystrophy with a single infusion priced at $4.25 million, while Hemgenix offers a cure for hemophilia B at $3.5 million. These therapies effectively convert lifelong treatment costs into a singular, substantial payment, challenging traditional insurance models that are built to spread costs over time.The primary obstacle is not the efficacy of these treatments but rather the financial and logistical infrastructures needed to support them. The current insurance model is ill-equipped to handle such large, one-time payments. Employers who often provide health insurance face a dilemma: investing millions in curing an employee who might leave the company shortly after receiving treatment could result in significant financial risk and disincentivizes employers from covering such therapies.Enter Aradigm Health, which has emerged as a potential solution to this conundrum. Aradigm aims to create an "infrastructure layer" specifically for these high-cost cures. With $20 million in funding backing their initiative, Aradigm seeks to pool financial risk across multiple employers, thus mitigating the impact of substantial individual claims. Their model involves employers contributing a fixed monthly fee into a shared fund that covers these expensive treatments when needed. This approach distributes financial volatility across a broader base rather than placing it on individual employers.Aradigm's strategy is not only about financial solutions but also about streamlining logistical complexities associated with delivering gene therapies. Their patient journey management includes coordinating with biotech companies for manufacturing schedules, arranging travel and accommodation for patients and families, and ensuring seamless insurance paperwork handling. This comprehensive support system reduces barriers that often delay or disrupt treatment delivery.Operating as a public benefit corporation with a "cost-plus" model, Aradigm ensures that any surplus from lower-than-expected claims is returned to employers rather than kept as profit. This aligns incentives towards patient care rather than profit maximization. Their approach highlights a critical need within the biotech and pharmaceutical industries: developing adaptable infrastructures that align with rapid scientific advancements.Meanwhile, Amgen has secured significant ground in 2023 with its second FDA approval for Uplizna in treating generalized myasthenia gravis—a chronic autoimmune neuromuscular disorder characterized by varying degrees of skeletal muscle weakness. Uplizna's mechanism involves targeting CD19 on B cells implicated in autoimmune diseases' pathogenesis. This expansion marks an advancement in therapeutic options for patients and underscores Amgen's growing footprint in treating complex autoimmune conditions.GlaxoSmithKline has also made headlines with Blujepa, marking it as the first new class of antibiotics for gonorrhea in over three decades while receiving approval for treating uncomplicSupport the show

The Smart 7 is an award winning daily podcast, in association with METRO that gives you everything you need to know in 7 minutes, at 7am, 7 days a week...With over 19 million downloads and consistently charting, including as No. 1 News Podcast on Spotify, we're a trusted source for people every day and the Sunday 7 won a Gold Award as “Best Conversation Starter” in the International Signal Podcast Awards If you're enjoying it, please follow, share, or even post a review, it all helps...Today's episode includes the following guests:Anthony Albanese - Australian Prime Minister Julie Inman Grant - Australia's ESafety Commissioner Dr Rachel Murrihy - Clinical Psychologist and Director of the KIdman Centre at Sydney's University of Technology Melanie Dawes - Chief Executive of Content Regulator Ofcom Lisa Nandy - UK's Culture Secretary Will Guyatt - The Smart 7's Tech Guru Reid Wiseman - NASA astronaut and member of Artemis crew Dr David Burtt - Postdoctoral Fellow at NASA's Goddard Space Flight CentreDr Mike Thorpe - Assistant Research Scientist at the University of Maryland, and the Planetary Environments Lab Division at NASADouglas Gonzaga de Sousa - Co-ordinator of the Centre for Specialty Coffees of Espirito SantoEdmond Rhys Jones - Co-lead at Boston Consulting Groups Centre for Climate and Sustainability Policy & RegulationBill Gates - Co-founder of Microsoft and high profile philanthropist Alyssa Tapley - Took part in the gene therapy trialProfessor Waseem Qasim - Consultant in Paediatric Immunology, and Professor of Cell and Gene Therapy at UCLDr Robert Chiesa - Consultant in Bone Marrow Transplantation at Great Ormond Street HospitalDr Roberto Biaggi - Co-author of the study from California's Loma Linda University.Celso Aguilar - Superintendent of the Toro Toro Park in BoliviaContact us over @TheSmart7pod or visit www.thesmart7.com or find out more at www.metro.co.uk Presented by Ciara Revins, written by Liam Thompson, researched by Lucie Lewis and produced by Daft Doris. Hosted on Acast. See acast.com/privacy for more information.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a myriad of transformative advancements and strategic shifts within these industries, illustrating the profound impact of innovation and regulatory changes on healthcare.Eli Lilly's recent strides in obesity treatment highlight a significant scientific breakthrough with their novel drug, retatrutide. Currently in phase 3 trials, this triple agonist targets GLP-1, GIP, and glucagon receptors, achieving an extraordinary 28.7% weight loss in participants. Additionally, it demonstrated a 75.8% reduction in knee osteoarthritis pain. Yet, the journey to this milestone wasn't without challenges. The trials saw a higher discontinuation rate than earlier studies, reminding us of the delicate balance between efficacy and patient tolerability—a consistent theme in obesity pharmacotherapy as developers strive to maximize benefits while minimizing adverse effects.In a testament to the global nature of drug development, Zealand Pharma has embarked on a $2.5 billion collaboration with a burgeoning Chinese biotech firm. This partnership aims to advance oral cardiometabolic therapies, underscoring the crucial role of strategic alliances in accessing innovative scientific platforms and expanding market reach.Rezolute faced a significant setback with a disappointing phase 3 trial for its hypoglycemia treatment, resulting in an 87% drop in stock value. This starkly illustrates biotech's inherent volatility and the critical importance of robust clinical trial design to mitigate financial risks associated with unsuccessful outcomes.Meanwhile, Moderna is leveraging Nanexa's expertise in long-acting formulations to enhance injectable therapy delivery systems. This collaboration is indicative of a broader industry trend focused on optimizing drug delivery technologies to boost efficacy and patient compliance.Operational restructuring is also evident as Pfizer implements cost-cutting measures, including layoffs in Switzerland, as part of broader strategic initiatives to optimize operations amid rising R&D costs and pricing pressures. Simultaneously, Chris Boulton's transition from Amgen to Prolynx underscores the fluid movement of talent within the industry—a reflection of ongoing strategic realignments.Regulatory landscapes are shifting as well. The FDA's approval of the first non-drug at-home treatment for depression marks an expansion into alternative therapeutic modalities beyond traditional pharmaceuticals. This wearable device offers adults with major depressive disorder a novel treatment option, integrating technology into mental health care—a promising addition to holistic treatment strategies.In another significant regulatory update, Teva Pharmaceuticals agreed to delist numerous patents from the FDA's Orange Book following an FTC ruling. This move is anticipated to foster increased generic competition and potentially lower medication costs for conditions like asthma and diabetes—a critical shift towards greater market accessibility.The FDA has also finalized guidance on promotional materials for biologics and biosimilars, standardizing advertising practices to ensure accurate representation of these products' efficacy and safety profiles amidst an expanding biologics market.Moreover, the FDA has launched its Commissioner's National Priority Voucher Pilot Review Program to expedite critical drug approvals. The first beneficiary under this program was USAntibiotics with Augmentin XR, signaling a potential shift towards more rapid access to essential medications.On the international policy front, recent developments between the UK and US have led to reductions in medicine rebate rates within the UK. This adjustment could lead to increased spending on new medicines, indicating more favorable conditions for pharmaceutical Support the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. In the ever-dynamic landscape of these industries, recent advancements have underscored both the scientific ingenuity and strategic foresight shaping patient care today.Pfizer has unveiled promising clinical trial data for Tukysa, indicating its potential as a first-line maintenance therapy in HER2-positive breast cancer. This development suggests that Tukysa could delay disease progression, offering patients extended survival prospects and an improved quality of life. Additionally, Pfizer's recent licensing agreement with Yaopharma for YP05002—a small molecule GLP-1 agonist currently in Phase 1 trials aimed at obesity treatment—highlights their strategic push into the rapidly evolving obesity treatment market.Meanwhile, Fondazione Telethon, an Italian nonprofit organization, has achieved a significant milestone with FDA approval for Waskyra—the first gene therapy for Wiskott-Aldrich syndrome. This ex vivo gene therapy directly targets the genetic roots of this rare disease, shifting treatment from symptomatic management to addressing underlying causes. This approval is transformative not only for patients suffering from this condition but also for the broader field of gene therapies, heralding a new era in treating rare genetic disorders.On the strategic front, Eli Lilly's decision to establish a $6 billion active pharmaceutical ingredient manufacturing facility in Huntsville, Alabama, marks a pivotal investment in U.S. manufacturing capabilities. This site will be critical in producing APIs for small molecule and peptide medicines, a testament to Lilly's commitment to meeting growing therapeutic demands while bolstering domestic production resilience—a trend gaining momentum across the industry. In oncology, Eli Lilly's Jaypirca demonstrated an impressive reduction in disease progression during Phase 3 trials for chronic lymphocytic leukemia.Biocon's acquisition of Viatris' stake in their biosimilar subsidiary exemplifies the shifting dynamics within the biosimilars market. This move allows Biocon to consolidate its market position as biosimilars gain traction as cost-effective alternatives to branded biologics. Such strategic realignments are indicative of competitive maneuvering aimed at capturing greater market share and driving down healthcare costs.Roche has made strides with compelling results from its Phase 3 trial of giredestrant, an oral selective estrogen receptor degrader showing a 30% reduction in risk for invasive breast cancer recurrence or death. The significance of this development lies in offering an oral alternative to injectable treatments, potentially improving patient adherence and reshaping standard care protocols for hormone receptor-positive breast cancer. Furthermore, Roche has achieved another regulatory milestone with its monoclonal antibody Gazyvaro gaining EU approval for treating lupus nephritis following successful Phase 3 trials.Innovation continues unabated as Formation Bio forms a new subsidiary through a $605 million deal with Lynk Pharmaceuticals. By securing rights to a next-generation immunology asset, Formation Bio positions itself at the forefront of immunological research developments. Concurrently, BioNTech and Bristol Myers Squibb have reported positive results from Phase 2 trials of Pumitamig for triple-negative breast cancer—validating bispecific antibodies' efficacy within oncology.Collaborative efforts are also reshaping industry landscapes. Bora and Corealis have partnered to create an end-to-end contract development and manufacturing organization for oral solid dose drug development. This collaboration aims to streamline processes and provide scalable solutions through a single contracting source, reflecting a shift towards integrated service models that enhance efficiencySupport the show

Guest: Victoria Coleman-Cowger, PhD Gene therapy offers transformative potential for individuals with sickle cell disease, but the psychosocial challenges that accompany such a major decision are often underestimated. Hear from Dr. Victoria Coleman-Cowger as she highlights recommendations for supporting emotional wellbeing during and after gene therapy and explains the importance of recognizing both the psychological and social impacts of curative treatment. Dr. Coleman-Cowger is a licensed clinical psychologist and the Associate Vice President of Neurology and Cell and Gene Therapy at Emmes, a full-service clinical research foundation.

In Part 2 of the 2026 pipeline series, host Carolyn Liptak welcomes Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient, to explore the advanced therapies pipeline: cell therapies, gene therapies, tissue-engineered products, and combination advanced therapy products. The discussion explores major pipeline trends, six leading products to watch, and the growing innovation expected to shape clinical practice in 2026.   Guest speakers:  Amanda Frick, PharmD, BCPS Senior Clinical Manager, Strategic Clinical Intelligence Spend Management Vizient Host: Carolyn Liptak, MBA, BS Pharm Pharmacy Executive Director Center for Pharmacy Practice Excellence (CPPE) Vizient    Show Notes: [00:05] — Introduction Announcer opens the episode. Host Carolyn Liptak introduces the focus on advanced therapies: cell & gene therapies, tissue-engineered products, and combination products. Guest: Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient. [01:07] — Defining Advanced Therapies FDA groups cell and gene therapies within advanced therapies. Total FDA-approved advanced therapies: 46. Amanda monitors 29 drug-like therapies within that group. [02:01] — Pipeline Size and Approval Activity S. pipeline: 264 agents in development. About 10 agents approach FDA decision annually. Actual approvals: 5–7 per year on average. [02:56] — Big-Picture Trends in Cell & Gene Therapy Oncology dominates 40–50% of all CGTs in development. Expanding into autoimmune, neurology, and earlier-phase therapies for diabetes, angina, osteoarthritis. Movement toward allogeneic ("off-the-shelf") therapies Designed to overcome limits of autologous cell manufacturing. Reduces wait time and manufacturing failures. Resurgence of therapeutic vaccines Currently 3 approved (Sipuleucel-T, Talimogene, Papzimeos ). 20+ vaccines in the pipeline, largely targeted to cancer. CE program coming Jan 29. [06:13] — Therapy #1:  Tabelecleucel or Tab-cel (Allogeneic EBV-Specific T-Cell Therapy) First allogeneic T-cell therapy expected in the U.S. For EBV-positive post-transplant lymphoproliferative disorder (PTLD). “Off-the-shelf” and donor-derived. [07:07] — Clinical Need & Outcomes Currentstandard of care: rituximab. After relapse, survival

In this podcast, we spoke with Troy Ostreng, Senior Product Manager and David Burdge, Director of Cell and Gene Therapy at CPC about the development of the MicroCNX® aseptic micro-connectors and how they're helping biopharma teams streamline closed-system operations for cell and gene therapies. What unfolded was a detailed and forward-looking conversation that touched on CPC's 47-year legacy, the technical demands of advanced therapies, and the company's plans to drive the future of automation and sterility in manufacturing. A Legacy That Positioned CPC for Today's Advanced Therapy Boom When asked how CPC's long history in biologics and hospital environments prepared the company for today's cell and gene therapy landscape, David took us back to CPC's roots. “CPC was founded in 1978, so that's 47 years of innovation within connection technologies,” he said. “The first biologic was released in 1982, synthetic insulin, and we were there supporting the industry with open-format connectors on single-use bags.” From the early development of biologics through the shift to single-use and the rise of stainless-steel/single-use hybrid systems, CPC continuously evolved its connection technologies. They launched steam-through connectors as bioprocessing grew more complex, released their first aseptic connector in 2009, and introduced their first connector specifically targeted for the cell and gene therapy market in 2017. David explained how that history matters today: “Biologics has about a 35-year head start on advanced therapies. So the question becomes, what lessons can we transfer from biologics to cell and gene therapy as that industry grows at three to four times the rate biologics did in its first decade?” That perspective, combining biological manufacturing experience with the needs of new therapy modalities, forms the foundation for CPC's MicroCNX platform. MicroCNX: The First Aseptic Connector Built for Small-Format Tubing As cell and gene therapy developers began scaling up manufacturing, they quickly discovered a problem: the connectors used for biologics were not designed for small-volume, patient-specific therapies. Troy described it plainly: “Several years ago, we started hearing rumblings that current connectors weren't meeting what cell and gene therapy required.” CPC responded with a deep Voice of Customer (VOC) initiative, interviewing process engineers, operators, manufacturing leaders, and platform developers. Over and over, the same needs emerged. Operators wanted something simple. “Ease of use was the number one requirement,” Troy said. “Operators needed a product that was easy to use so they could make sterile connections in a short amount of time.” Processes demanded robustness. “Customers needed a connection they could trust—no contamination, no failures, no weak spots in the connection process,” he added. Small-volume precise applications required connectors actually designed for them. With autologous therapies, he noted, “We aren't talking about 1,000 liters; we're talking about 250 milliliters. And if there's a mishap, that could mean the difference between life and death for a patient.” All of this laid the groundwork for MicroCNX, which became the first aseptic connector engineered for small-format tubing. The “Pinch-Click-Pull” Process: Sterility Meets Speed One of the standout features of MicroCNX is its elegantly simple pinch-click-pull operation. Troy explained how simplicity came directly from user feedback. “As operators walked us through their pain points, what they needed was clear: a connector they could learn immediately. So MicroCNX has a three-step process—pinch, click, pull. You can literally do it as fast as I say it.” He continued,“Once someone does it one time, they're basically an expert. That ease of use dramatically reduces operator error.” For an industry where operator variability remains one of the biggest sources of risk and batch loss, eliminating complexity is critical. Cryogenic Challenges Call for Cryo-Rated Solutions As the conversation shifted to cryopreservation, a critical component of cell therapy manufacturing,Troy introduced the MicroCNX® ULT and MicroCNX® Nano variants. “These were really developed because therapies were being frozen to –150°C, even –190°C. You need a connector that can be frozen to those temperatures, thawed, and still be as robust as it was before.” The ULT and Nano were engineered with: Low-profile geometries to fit inside freezing cassettes Specialized materials to withstand thermal stress Chemical compatibility with DMSO and other cryoprotectants Enhanced durability to survive impacts while frozen Troy emphasized how critical it was to get the materials right: “We searched extensively for a material that could handle those harsh chemicals and temperatures. What we landed on was PPSU—polyphenylsulfone. It's chemically sound, and it's incredibly impact-resistant at very low temperatures.” CPC built these connectors because customers repeatedly told them: existing solutions were cracking, leaking, or becoming brittle. MicroCNX was engineered to overcome all of that. True Closed Systems vs. Functionally Closed Systems: Why the Difference Matters A substantial part of the conversation focused on the differences between closed, functionally closed, and open systems—distinctions that are often overlooked but critically important. Troy broke down the differences clearly: “An open system is exposed at some point. A functionally closed system is inherently open but gets closed temporarily to let fluid transfer. In comparison, a closed system is never open at any point.” Examples of functionally closed systems include: Biosafety cabinets (BSCs) Luer-based connections Closed system transfer devices These approaches require: Sanitization Careful environmental controls Operator expertise And, as Troy noted, “a mishap in one of these can mean losing a very valuable therapy.” CPC's sterile connectors—including MicroCNX minimize these risks: “Our connectors allow the system to remain closed 100% of the time. That greatly reduces contamination risk.” This distinction isn't merely academic—it has direct regulatory implications as well. David added,“In Annex 1, they refer to intrinsically sterile connection devices—like sterile connectors and tube welders—that allow operations normally requiring Grade A or B to occur in a Grade C or D environment.” That ability to operate safely in lower-grade spaces is increasingly critical as the industry tries to overcome facility and labor bottlenecks. Why Teams Are Moving Away from Tube Welding Tube welding has been part of bioprocessing for decades, but David explained why its era may be ending for CGT. “Tube welding was born out of the blood banking industry when no other solution existed. But sterile connectors don't require capital investment. They're faster. They eliminate issues like tubing alignment or pinhole leaks. They're simply more reliable.” As biologics manufacturers have already done, CGT teams are now transitioning toward connectors like MicroCNX® that provide sterile, consistent, low-burden operations. The MicroCNX® Luer Variant: Supporting Transitional Workflows Not all workflows are ready to move away from luer-based devices. That's where the MicroCNX Luer variant fits in. Troy described how it works.“You connect a syringe or bag with a luer inside the BSC, but then because the MicroCNX® connector itself is sterile, you can take it outside the hood and make a sterile connection elsewhere.” This capability bridges legacy workflows and fully closed systems—critical during process development, technology transfer, or when working with specific devices. Co-Development: The Heart of CPC's Innovation Process As the conversation returned to CPC's broader philosophy, David highlighted how important customer collaboration is. “It's all about the customer for CPC,” he said. “We start with Voice of Customer. Our business and applications managers are out in the field understanding real applications and guiding them to the right products.” This feedback fuels CPC's two major development tracks: Catalog product development (platforms like MicroCNX) Custom-engineered solutions for unique applications David added: “We maintain a full new product introduction roadmap. Some products will be released broadly. Others will be developed specifically for one customer. But both are driven by real application requirements.” This process ensures CPC's products evolve in lockstep with the needs of advanced therapy teams. Looking Ahead: Designing Connectors for Robotics and Automation Toward the end of the conversation, David turned to one of CPC's biggest focus areas: the future of automation. “The ultimate customer in this industry is the patient,” he said. “And right now we face barriers—capacity, speed, accessibility, cost. Process automation can significantly reduce those barriers.” Automation requires connectors designed not just for human hands but for robotics: Predictable geometries Features optimized for machine vision Forces and actuation steps compatible with robotic grippers Designs intended for automated loading and unloading David summarized CPC's future direction: “We're taking a fresh look at our connectors, reimagining them as something designed for robotic manipulation. It's a high priority for us.” Troy echoed the sentiment: “Our connectors are awesomely designed for humans. But automation is coming, and we're focused on the features robots need.” A Future Built on Innovation and Patient Impact The interview closed with both guests reflecting on CPC's mission. “We're incredibly passionate about innovation and meeting the needs of our customers through thoughtful product development,” Troy said.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a series of compelling advancements and challenges that are shaping this dynamic industry.Pfizer has recently unveiled phase 3 results for its hemophilia drug Hympavzi, positioning it as a formidable competitor against Sanofi's Qfitlia and Novo Nordisk's Alhemo. The data suggests Hympavzi offers robust efficacy, potentially revolutionizing hemophilia treatment and enhancing patient outcomes significantly. This development is not just about competition; it represents a critical stride forward in patient care for those affected by this debilitating condition.UCB plans to seek regulatory approval for Fintepla to treat an additional epileptic disorder following positive phase 3 trial results in patients with CDKL5 deficiency disorder. This decision reflects promising results and could offer new hope to patients with limited treatment options, further cementing Fintepla's position in epilepsy management.Gene therapy continues to shine with CSL's Hemgenix demonstrating sustained long-term benefits. After five years of follow-up, a single dose has reduced annualized bleeding rates by an impressive 90% in hemophilia B patients within the Hope-B study. Such long-term efficacy highlights gene therapy's transformative potential, offering lasting improvements in quality of life for patients with genetic disorders.Regulatory frameworks are also evolving, as evidenced by the CDC's Advisory Committee on Immunization Practices (ACIP) voting to modify hepatitis B vaccine guidance for newborns. This decision advocates an individualized approach, sparking debate over vaccination strategies, which reflects the complexities and delicate balance required in public health policies today.Regeneron is making strides to simplify treatment regimens for T-cell engagers Lynozyfic and Ordspono. By reducing regimen complexity, they aim to improve patient compliance and expand access, thus enhancing the potential impact on cancer care—a crucial step toward broader therapeutic accessibility.In an effort to address cost barriers and stimulate domestic production of generics, Mark Cuban has proposed lowering FDA fees. This proposal highlights ongoing discussions around regulatory reforms needed to boost generics manufacturing in the United States. Such initiatives align with broader industry goals of increasing access to affordable medications.Despite these advancements, industry insiders have expressed concerns about "unprecedented turmoil" within the FDA. These challenges underscore the critical role of stable leadership in maintaining public trust and ensuring effective regulation amidst rapid scientific progress.Obesity treatments are gaining significant attention as companies like Wave Life Sciences and Structure Therapeutics report promising data, capturing increased investor interest. This trend underscores a growing focus on innovative pharmacological approaches to address obesity—a complex, multifactorial condition that affects millions globally.The strategic landscape of cancer diagnostics is also evolving, as evidenced by Natera's acquisition of Foresight Diagnostics. This deal underscores ongoing industry consolidation efforts aimed at enhancing technological capabilities and expanding market presence—a testament to the critical role diagnostics play in comprehensive cancer care strategies.As we observe these developments, it's clear that scientific innovation coupled with strategic regulatory maneuvers is shaping the future of healthcare. These advancements offer profound implications for patient care, providing new hope through advanced therapies while highlighting the importance of effective regulation and strategic partnerships.Precision medicine continues to extend beyond oncology into fields like cardiometabolic and neSupport the show

Send us a textOn the latest WTR Small-Cap Spotlight podcast episode, we welcomed Jan Eryk Umiastowski, CFO of GenSight Biologics (Euronext Paris: SIGHT), a late clinical-stage biopharma company based in Paris, France, primarily focused on developing and commercializing novel disease-modifying gene therapies aimed at reversing vision loss from severe neurodegenerative retinal diseases. He discusses GenSight's development pipeline, what's unique about  the company's approach to gene therapy and upcoming target milestones.

Novo Nordisk's highly anticipated data for semaglutide in Alzheimer's dashed hopes that the GLP-1 therapy could become a game changer in the disease. On the latest BioCentury This Week podcast, Executive Editor Selina Koch discusses the Phase III readout including what it says about the mechanism's use in the neurodegenerative disease, Novo's decision to skip Phase II and enroll a large patient group in a later stage trial, and what other datasets for GLP-1s in the indication have shown.Washington Editor Steve Usdin explains why FDA's new vaccine policies, driven by CBER Director Vinay Prasad, could have impacts more far-reaching than expected, including making it more difficult to develop or modify vaccines. And Lauren Martz, Executive Director of Biopharma Intelligence, analyzes the bleak investment outlook for cell and gene therapy companies in the U.S. and why China's biotech ecosystem offers a glimmer of hope for sponsors of these assets.View full story: https://www.biocentury.com/article/657721#Semaglutide #GLP1Therapies #VaccinePolicy #CellAndGeneTherapy #AlzheimersResearch00:00 - Introduction01:53 - Novo's Alzheimer's Miss13:19 - FDA's New Vaccine Policy19:53 - Funding C> BiotechsTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we'll explore the latest advances and strategic moves shaping the industry, providing you with insights into how these developments might influence drug development and patient care.The pharmaceutical and biotech sectors are currently experiencing a wave of transformative changes. A significant development is the trade agreement between the United States and the United Kingdom, which excludes medicines from import tariffs. This strategic move, orchestrated by the Trump administration, is set to reduce costs and bolster investments in pharma sectors across both nations. By enhancing market accessibility, it aims to stimulate cross-border investment in pharmaceutical research and production.On the clinical front, Eli Lilly is making headlines by joining Novo Nordisk in reducing self-pay prices for its GLP-1 receptor agonist, Zepbound. This reflects a broader industry trend towards patient-centric pricing models aimed at improving affordability. With healthcare costs on the rise, these measures could ease financial burdens for patients requiring long-term medication regimens.Regulatory updates are also making waves, with the FDA planning stricter vaccine regulations under Dr. Vinay Prasad's leadership at the Center for Biologics Evaluation and Research (CBER). These updates come amid concerns over vaccine safety during COVID-19-related incidents, underscoring a commitment to maintaining public trust in vaccines. Regulatory scrutiny continues as Prasad focuses on COVID-19 vaccine safety in children amid reports linking 10 child deaths to vaccines via VAERS—emphasizing challenges interpreting safety data while highlighting the need for robust methodologies ensuring reliable causality conclusions.In corporate restructuring news, Valneva is streamlining its operations by closing a site and eliminating 30 roles. This move highlights an industry focus on optimizing resources to bolster vaccine development pipelines. Meanwhile, Microsize and Schedio's acquisition of Lonza's Swiss micronization plant underscores ongoing investments in advanced manufacturing technologies critical for high-quality pharmaceuticals.In ophthalmology, Belite Bio is advancing with promising Phase 3 results for tinlarebant in treating Stargardt disease—a rare genetic eye disorder. This success positions Belite to file for FDA approval, potentially expanding treatment options for this underserved patient population. Such advancements in targeted therapies emphasize the need for ongoing research in genetic disorders.Regeneron is betting $150 million on Tessera's gene writing technology targeting alpha-1 antitrypsin deficiency (AATD). This collaboration highlights the industry's growing interest in gene therapy as a frontier for treating rare diseases, marking a shift towards precision medicine where tailored genetic interventions offer hope for previously untreatable conditions. Regeneron's strategic move investing $275 million partnering with Tessera exploring gene editing capabilities—focusing on TSRA-196 targeting the SERPINA1 gene linked to AATD showcases potential advancing therapeutic options for genetic disorders through gene editing technologies offering new disease treatment avenues.AI-driven platforms were spotlighted at RSNA 2025 by industry leaders such as GE Healthcare, Philips, and Siemens. These innovations promise to revolutionize radiological workflows by enhancing diagnostic accuracy and operational efficiency through AI integration. As AI continues to permeate healthcare technologies, its potential to transform diagnostic processes marks a significant leap towards personalized medicine.Akebia Therapeutics' acquisition ofSupport the show

What if an Alzheimer's therapy did not just slow decline, but restored memory and physical health back to baseline?In this episode of Spark Time, we sit down with Dan Salain, President and CEO of Recall Therapeutics, and Dr. Atul Chopra, Co-founder and CSO, to unpack one of the boldest ideas in neuroscience today: RTX-100, a gene therapy designed to restore cognition and systemic health in Alzheimer's disease. Dan and Atul walk us through the science behind targeting memory circuits rather than just clearing amyloid, how a liver-expressed protein naturally crosses the blood–brain barrier, and why their preclinical data show full recovery of associative, recognition, and spatial memory in advanced mouse models.We also explore the overlooked systemic side of Alzheimer's cachexia, sarcopenia, frailty, UTIs, pneumonia and how RTX-100 appears to normalize body weight, muscle strength, endurance, and fluid balance alongside cognitive recovery. From serendipity in the lab to regulatory strategy and potential paths to market, this conversation opens up a very different vision for what treating Alzheimer's and even aging itself could look like.

In this uplifting episode, hosts Beth Glassman and Cathy Gildenhorn sit down with Leanna Scaglione, a powerhouse rare disease advocate and marathon runner living with NF2-Related Schwannomatosis (NF2-SWN), formerly known as neurofibromatosis type 2. Diagnosed at just 16 years old, Leanna's life changed dramatically when tumors were discovered in her nervous system, a hallmark of NF2-SWN. Many would have slowed down. Instead, Leanna sped up. Today, she has: Completed multiple marathons, including Berlin and New York City Participated in clinical trials Become an advocate and 2025 Ambassador for the Children's Tumor Foundation Set her sights on becoming the first person with NF2 to complete all 7 Abbott World Marathon Majors Her message is powerful: a diagnosis doesn't define the finish line. In This Episode, We Discuss: What NF2-SWN is and how it impacts the nervous system The emotional toll of receiving a life-altering diagnosis as a teenager Losing a dream — and finding a new one Running marathons through surgeries, treatments, and uncertainty How advocacy and visibility can change the rare disease landscape Building a life rooted in resilience, purpose, and possibility About Our Guest: Leanna Scaglione is 34 years old living with NF2-Related Schwannomatosis (NF2-SWN). Ever since being diagnosed at 16 years old, she has refused to live defined by her diagnosis. From relearning to walk, participating in immunotherapy drug trials, and going through numerous surgeries she has defied the odds against her. Most recently she has undergone surgery to remove her right acoustic neuroma. She continues to live her best life as an endurance runner, sharing her story and experiences living with NF2 in hopes to spread awareness, motivate her community and rally for a cure. Resources:  Children's Tumor Foundation NF2 Accelerator: A Strategic Portfolio Approach to End NF2 Articles Explaining NF2 MedlinePlus GeneReviews National Organization for Rare Disorders (NORD) Research from China that Leanna reference for a gene therapy for NF2 Yuan R, Wang B, Wang Y, Liu P. Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions. Oncol Ther. 2024 Jun;12(2):257-276. doi: 10.1007/s40487-024-00279-2. Epub 2024 May 17. PMID: 38760612; PMCID: PMC11187037. Additional Research Articles Alexandra K. O'Donohue, Samantha L. Ginn, Gaetan Burgio, Yemima Berman, Gabriel Dabscheck, Aaron Schindeler, The evolving landscape of NF gene therapy: Hurdles and opportunities, Molecular Therapy Nucleic Acids, Volume 36, Issue 1, 2025, 102475 ,ISSN 2162-2531, https://doi.org/10.1016/j.omtn.2025.102475. Connect With Us:   Stay tuned for the next new episode of “It Happened To Me”! In the meantime, you can listen to our previous episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “It Happened To Me”.  “It Happened To Me” is created and hosted by Cathy Gildenhorn and Beth Glassman. DNA Today's Kira Dineen is our executive producer and marketing lead. Amanda Andreoli is our associate producer. Ashlyn Enokian is our graphic designer. See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, ItHappenedToMePod.com. Questions/inquiries can be sent to ItHappenedToMePod@gmail.com. 

Host Matt Burgess speaks with genetic counsellor and NSGC president Sara Pirzadeh‑Miller about her early research on values in genetic counselling, how life experience shapes practice, and the evolving role of genetic counsellors. They also discuss the clinical challenges of the CDH1 gene, risk interpretation, screening versus prophylactic surgery, the rise of genetic counselling assistants, and future genomic innovations such as gene therapies and improved risk stratification.

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today we dive into some of the most significant and exciting advancements shaping the industry.Let's start with a breakthrough coming out of recent clinical trials. A novel approach to treating Alzheimer's disease is making waves. Researchers have been focusing on a monoclonal antibody designed to target amyloid-beta plaques in the brains of patients suffering from this debilitating condition. The latest Phase 3 trial results have shown a promising reduction in cognitive decline among participants, offering a glimmer of hope for millions affected by Alzheimer's worldwide. The study, conducted over 18 months, involved more than 1,500 patients and demonstrated a statistically significant slowing of disease progression compared to placebo. This could mark a pivotal moment in Alzheimer's treatment, shifting the paradigm from symptomatic relief to disease modification.In regulatory news, the FDA has granted approval to a new gene therapy for hemophilia B. This therapy, developed using cutting-edge CRISPR technology, offers a potential cure for patients who have long relied on regular infusions to manage their bleeding disorder. By directly editing the genes responsible for clotting factor production, this therapy can potentially provide long-term relief with just a single administration. The approval follows extensive clinical trials that showed sustained increases in clotting factor levels and a dramatic reduction in bleeding episodes among participants. This development not only underscores the growing impact of genomic medicine but also highlights the regulatory body's commitment to advancing innovative treatments that meet unmet medical needs.Shifting focus, there's an intriguing trend emerging in oncology drug development. The industry is increasingly adopting personalized medicine approaches, tailoring treatments based on individual genetic profiles. This shift is particularly evident in the rise of targeted therapies and combination regimens designed to tackle cancer at its genetic roots. A recent study demonstrated how combining targeted therapies with immune checkpoint inhibitors can enhance treatment efficacy in certain types of cancer, such as melanoma and non-small cell lung cancer. By understanding the specific mutations driving tumor growth and leveraging the body's immune response, these combinations are setting new standards for cancer care.In another significant development, researchers have unveiled promising results from an innovative mRNA vaccine trial aimed at combating infectious diseases beyond COVID-19. The technology, which gained prominence during the pandemic, is now being applied to other viral threats like influenza and RSV. Early-phase clinical trials have shown robust immune responses and favorable safety profiles, suggesting mRNA vaccines could soon revolutionize how we approach vaccination for a variety of pathogens. This versatility and rapid development timeline make mRNA platforms particularly attractive for addressing emerging infectious diseases swiftly.Meanwhile, a new study has raised awareness about antibiotic resistance—a growing concern globally. Scientists have developed an advanced diagnostic tool capable of rapidly identifying bacterial infections and their resistance profiles within hours instead of days. This innovation can significantly impact how clinicians prescribe antibiotics, ensuring targeted treatments that minimize resistance development. By providing real-time insights into bacterial genetics and resistance mechanisms, this tool empowers healthcare providers to make informed decisions that preserve antibiotic efficacy for future generations.Lastly, let's toSupport the show

Happy Thanksgiving… Thursday, November 27, 2025. Week 48.   Continued from #S10e189…   And the AAV Paper (https://pubmed.ncbi.nlm.nih.gov/40988338/) from #S10e187… https://curesyngap1.org/podcasts/syngap10/clinical-research-ai-dx-nl47-survey-autism-press-6-days-to-register-for-syngap1conf-s10e187/   https://curesyngap1.org/blog/ Issac's story, Transmitter reprint, Scramble 4 write up and JK on #Autism, #MustRead   https://curesyngap1.org/resources/webinars/ 119 - 112 Register for livestream of the conference, AAV from Allen Inst., dos en espanol, Missense, Unlock and Rare-X for ProMMiS.   https://curesyngap1.org/podcasts/syngap1-stories/  A gold mine have you listened to #38, the Virginie Pod, really must listen, she is our leader. https://www.linkedin.com/posts/graglia_syngap1stories-syngap1-syngap1storiesty-activity-7387203351907708928-liNL    CLINICAL TRIAL & GENETIC MEDICINE CORNER Example of Ultragenyx FAST Angelman follow on trial to look at other ages and genotypes, key message, never give up. https://www.linkedin.com/posts/cureangelman_the-global-aurora-study-will-enroll-approximately-activity-7389647402690957312-Bihi Congrats to Novartis on approval of the first Gene Therapy to Cure SMA! https://www.linkedin.com/posts/graglia_sma-fdaapproval-rarediseaseinnovation-activity-7398939783005347840-Ocd_ Remember Spinraza was approved in December 2019.   TODOS Sign up for Citizen Health: https://www.citizen.health/partners/srf USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/   SOCIAL MATTERS 4,468 LinkedIn.  https://www.linkedin.com/company/curesyngap1/  1,480 YouTube.  https://www.youtube.com/@CureSYNGAP1    11.2k Twitter https://twitter.com/cureSYNGAP1  45k Insta https://www.instagram.com/curesyngap1/    $CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ Episode 190 of #Syngap10 #CureSYNGAP1

This week, a world first gene therapy treats rare Hunter syndrome. Could these personalised medicines be used more widely? We speak to Claire Booth, professor in Gene Therapy at Great Ormond Street Hospital. And high in the Chilean desert, the last bit of 13 billion year old light has hit the mirror of the Atacama Cosmology Telescope for the last time. Dr Jenifer Millard, a science communicator and host of the Awesome Astronomy podcast, tells us what it's been up to for the past 20 years.And Penny Sarchet, managing editor at New Scientist brings her pick of the latest new discoveries.Think you know space? Head to bbc.co.uk, search for BBC Inside Science, and follow the links to the Open University to try The Open University Space Quiz. Presenter: Tom Whipple Producers: Alex Mansfield, Ella Hubber, Jonathan Blackwell, Tim Dodd and Clare Salisbury Editor: Martin Smith Production Co-ordinator: Jana Bennett-Holesworth

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve deep into a series of transformative events that underscore the dynamic nature of our industry, where scientific innovation meets regulatory evolution and market adaptation.We begin with significant regulatory news from Medicare, which recently announced price reductions for 15 prescription drugs, including Novo Nordisk's semaglutide products, Ozempic and Wegovy. This initiative is part of the Inflation Reduction Act aimed at making essential medications more affordable. By potentially increasing accessibility to these treatments, this move highlights a growing trend towards cost containment in drug pricing within the U.S. healthcare system. It reflects a broader effort to ensure that life-saving treatments remain within reach for more patients, emphasizing the need for balance between innovation and affordability.Turning to approvals, Otsuka has secured FDA clearance for Voyxact, a first-in-class treatment targeting IgA nephropathy (IgAN). This positions Otsuka in an increasingly competitive market space populated by major players like Novartis and Vertex. The entry of Voyxact could pave the way for innovative therapeutics in kidney diseases, offering new hope to patients who have had limited treatment options until now.On the other side of the Atlantic, French authorities have conducted a raid on Sanofi's headquarters as part of a tax fraud investigation. This development sheds light on ongoing scrutiny in the pharmaceutical sector regarding financial practices and regulatory compliance. Such investigations can have far-reaching implications on corporate governance and transparency, reminding us of the importance of ethical practices in maintaining industry trust.Novo Nordisk has strategically used its FDA national priority voucher to expedite the review process for a high-dose formulation of Wegovy. This move underscores the importance of regulatory incentives in accelerating drug development timelines, allowing for quicker patient access to potentially life-changing therapies. It's a testament to how strategic navigation through regulatory pathways can significantly impact drug availability.In clinical trials, Sarepta Therapeutics received FDA clearance to conduct a study combining its gene therapy Elevidys with sirolimus in patients with Duchenne muscular dystrophy. The study aims to address liver safety issues associated with Elevidys, which had led to previous label restrictions. This reflects the industry's commitment to enhancing therapeutic safety profiles while expanding treatment indications.In oncology advancements, AstraZeneca's Imfinzi received FDA approval for use in early-stage stomach cancer, marking its third perioperative indication. This approval underscores the expanding role of immunotherapy across various cancer types and stages, offering new treatment paradigms that could improve surgical outcomes and long-term patient survival.Despite these advances, there is skepticism regarding artificial intelligence's role in regulatory compliance submissions among pharmaceutical professionals. A survey reveals that 65% express distrust towards AI-generated outputs, highlighting challenges that AI technologies face in gaining acceptance within highly regulated environments such as pharmaceuticals. However, federal recommendations to revamp U.S. biotechnology research emphasize incorporating AI into scientific processes to maintain global competitiveness. This call reflects concerns over potential declines in innovation leadership and underscores the need for strategic investment in research infrastructure.In antitrust news, the Federal Trade Commission (FTC) outlined its case agaiSupport the show

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a landscape rich with scientific innovation, regulatory scrutiny, and strategic business decisions shaping the future of healthcare.Let's begin with Novartis, which has achieved a significant milestone by securing FDA approval for Itvisma. This is an intrathecal formulation of its gene therapy Zolgensma, designed to treat older patients with spinal muscular atrophy (SMA). Priced at $2.59 million, this approval is a critical advancement in gene therapy for SMA, broadening the treatment horizon for a wider patient demographic. Gene therapies are increasingly crucial in addressing rare genetic disorders, offering transformative potential in patient care. Novartis's SMA market expansion post-FDA approval of Itvisma not only enhances its SMA portfolio but also signifies growing acceptance towards gene therapies as viable treatment options for genetic disorders. Meanwhile, Novartis is strategically restructuring, planning to cut 550 jobs at a Swiss plant by 2027 while expanding its workforce in North Carolina. This move reflects broader industry trends toward optimizing global operations and investing in regions with strategic manufacturing capabilities.In the sphere of regulatory scrutiny, lawmakers are questioning the FDA's National Priority Voucher Program amid concerns about corruption and expedited reviews. This situation highlights ongoing challenges within regulatory frameworks to balance innovation speed with rigorous safety assessments. An investigation into the FDA's new priority review voucher program has been initiated due to concerns over corruption and expedited review processes potentially compromising drug safety. This inquiry could influence future regulatory frameworks and underscores balancing accelerated drug approvals with rigorous safety standards. Richard Pazdur expressed concerns about expedited drug approval programs' safety and legality as he takes on his new role as director of the FDA's Center for Drug Evaluation and Research. These initiatives aim to accelerate drug reviews but spark debate over patient safety implications—underscoring an ongoing tension between innovation speed and regulatory diligence.Turning to Novo Nordisk, their expansion of the Amycretin program demonstrates a commitment to tackling chronic conditions like diabetes. Following promising Phase 2 data showcasing dual agonist capabilities, Novo Nordisk is advancing pivotal trials focused on obesity. This strategic pivot aligns with market needs and scientific discoveries that could significantly enhance diabetes management options. Further emphasizing Novo Nordisk's commitment to diabetes management, their expansion of the amycretin program after promising Phase 2 results demonstrates the efficacy of a dual agonist originally focused on obesity. This underscores a trend toward multifunctional biologics addressing metabolic disorders by targeting multiple pathways—indicative of broader industry shifts towards integrated therapeutic approaches. Novo Nordisk's recent mid-stage clinical trial results for Amycretin—a weight loss treatment—are noteworthy as they demonstrated sustained efficacy over 36 weeks in type 2 diabetes patients without a plateau in weight loss. Analysts highlight its potential as a superior therapeutic option in the burgeoning weight loss market due to its durable solution for weight management.On a contrasting note, SK Life Science encounters regulatory hurdles as the FDA scrutinizes advertising practices related to its antiseizure medication Xcopri. This scenario underscores the complex interplay between marketing strategies and regulatory compliance within the pharmaceuticSupport the show

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Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a host of transformative events reshaping the landscape, from strategic acquisitions and funding infusions to regulatory maneuvers and scientific breakthroughs.Johnson & Johnson has taken a decisive step in its oncology strategy with the $3 billion acquisition of Halda's cell death technology. This acquisition, focusing on the "hold and kill" bifunctional small molecule platform, is poised to enhance J&J's prostate cancer pipeline significantly. It underscores J&J's commitment to expanding its oncology portfolio through innovative platforms designed to improve therapeutic outcomes. The move highlights a broader industry trend toward personalized medicine and targeted cancer therapies, which are becoming pivotal in improving patient care.In another domain of cancer treatment, Nuvalent has unveiled promising Phase 1/2 data for its candidate neladalkib, which could position the company as a formidable competitor to Pfizer's established lung cancer drug, Lorbrena. The promising data might expedite regulatory discussions with the FDA, potentially leading to an accelerated approval process. This development illustrates the competitive landscape in oncology, where firms strive to introduce novel therapies with improved efficacy and safety profiles.The field of antibody-drug conjugates (ADCs) is also experiencing significant advancements. A San Diego-based biotech has secured $120 million in funding to develop a best-in-class ADC formula, with support from Merck & Co. This initiative aims to refine the precision and efficacy of ADCs by delivering cytotoxic agents directly to cancer cells while minimizing collateral damage to healthy tissues. Such innovations are crucial as they represent a new frontier in targeted cancer therapy.In terms of financial activities, Artios Pharma's successful $115 million Series D funding round is set to bolster its clinical efforts in exploring DNA damage response inhibitors for cancer treatment. These inhibitors target cancer cells' ability to repair DNA damage, holding potential for more effective therapies against resistant cancer types. Meanwhile, Sofinnova Partners' €650 million raise for biotech and medtech investments amid a volatile economic environment underscores continued investor confidence in life sciences despite market uncertainties.Bayer is making strategic moves in China by opening an incubator in Beijing. This facility will host local biopharma companies such as Suzhou Puhe Biopharma and Beijing Youngen Technology, fostering innovation and collaboration within China's burgeoning biotech landscape. Such initiatives reflect global efforts to leverage regional strengths and foster cross-border collaborations.On the operational side, Nxera Pharma is restructuring its workforce by laying off 15% of its staff as part of a strategic pivot towards profitability. This decision mirrors broader industry trends where companies refocus resources on core projects to streamline operations and enhance financial stability.A recent study has highlighted the impact of NIH grant cuts on clinical trials across the United States. Over 383 trials involving more than 74,000 patients have been disrupted due to funding terminations under the current administration. This situation raises concerns about the sustainability of clinical research funding and its implications for ongoing medical advancements.Jazz Pharmaceuticals has reported practice-changing Phase 3 results for its HER2-targeted drug Ziihera for gastroesophageal adenocarcinoma. These findings reaffirm Jazz's confidence in positioning Ziihera as a preferred first-line treatment option for HER2-positive cancers, poSupport the show

In this episode of the Parkinson's Podcast, learn about gene therapy for Parkinson's, including common misunderstandings and ongoing research about it.  This episode contains some technical terms and abbreviations. For your convenience, here is a short list of definitions that might be helpful.   AADC – Aromatic L-Amino Acid Decarboxylase: An enzyme that helps make dopamine (not a rock and roll band)  AAV – Adeno-Associated Virus: A harmless virus used to deliver gene therapy  CDNF – Cerebral Dopamine Neurotrophic Factor: A protein that helps keep dopamine-producing cells healthy  DBS – Deep Brain Stimulation: A surgical treatment that uses electrical signals to help control symptoms  GAD – Glutamic Acid Decarboxylase: An enzyme that helps rebalance brain circuits.   GBA – A gene linked to Parkinson's risk  GCH1 – GTP Cyclohydrolase 1: An enzyme that supports dopamine production.   GDNF – Glial Cell Line-Derived Neurotrophic Factor: A protein that helps protect brain cells  LRRK2 – A gene linked to Parkinson's risk  PARK2 – A gene linked to Parkinson's risk  PET – Positron Emission Tomography: A brain scan that shows chemical activity   PINK1 – A gene linked to Parkinson's risk   SNCA – A gene linked to Parkinson's risk  More information about Gene Therapy for Parkinson's is available here:  https://davisphinneyfoundation.org/blog/gene-therapy-parkinsons/   https://youtu.be/n2KNtRK3SIk  https://youtu.be/v9mJiO4EEag    Sign up for updates on webinars, events, and resources for the Parkinson's community: https://dpf.org/newsletter-signup

The Foundation funded preclinical studies for the approach and launched Opus.

For the first time in human history, we can diagnose thousands of genetic diseases—often for under $1,000—but we still can't treat most of them. The problem isn't understanding what's broken; it's delivering the fix to the right cells.Eric Kelsic, CEO of Dyno Therapeutics, joins a16z's Jorge Conde to explain how AI-designed protein shells are solving gene therapy's delivery crisis. They explore why Huntington's patients can now get 15 extra years of healthy life, how Dyno inverted the liver-to-brain delivery ratio by 1000x, and why capsids evolved by nature are now being designed by machine learning models trained on millions of variants.Eric introduces the concept of genetic agency—humanity's first-ever ability to take action at the DNA level—and details why solving delivery for common diseases will make ultra-rare disease treatments economically viable. Plus: what happens when gene therapy requires neurosurgery today but could be a simple injection tomorrow, why recent deaths in clinical trials prove we need better technology now, and how genetic medicine could become as routine as surgery within our lifetimes. Resources:Follow Eric on X: https://x.com/ekelsicFollow Jorge on X: https://x.com/JorgeCondeBioLearn more about GATC 2025: https://www.dynotx.com/gatc2025 Stay Updated:If you enjoyed this episode, be sure to like, subscribe, and share with your friends!Find a16z on X: https://x.com/a16zFind a16z on LinkedIn: https://www.linkedin.com/company/a16zListen to the a16z Podcast on Spotify: https://open.spotify.com/show/5bC65RDvs3oxnLyqqvkUYXListen to the a16z Podcast on Apple Podcasts: https://podcasts.apple.com/us/podcast/a16z-podcast/id842818711Follow our host: https://x.com/eriktorenbergPlease note that the content here is for informational purposes only; should NOT be taken as legal, business, tax, or investment advice or be used to evaluate any investment or security; and is not directed at any investors or potential investors in any a16z fund. a16z and its affiliates may maintain investments in the companies discussed. For more details please see a16z.com/disclosures.  Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.