Podcasts about Gene therapy

Al Gianchetti, CEO of Nuevocor,  discusses genetic cardiomyopathies, an underdiagnosed condition that might explain multigenerational heart failure. Current treatments include beta blockers, pacemakers, and heart transplants but do not alter the course of the disease.  Because of multiple gene mutations, Nuevocor is developing a precision therapy for LMNA-related cardiomyopathy based on mechanobiology that prevents cardiac cell death using a method different from gene replacement and gene editing. Al explains, "We're focused on genetic cardiomyopathies, and our lead drug, NVC001 in particular, is focusing first on LMNA cardiomyopathy, and LMNA is a specific gene mutation. We're focusing on LMNA-related cardiomyopathy for several reasons. First of all, it's one of the most severe forms of genetic cardiomyopathy, with a very poor prognosis for patients and their families who have the genetic mutation."   "The second reason is that although it's a rare disease, it affects a fairly large number of people. For example, about 50,000 people in the United States are estimated to have LMNA-related cardiomyopathy. The third reason is that this disease is not amenable to gene replacement strategies where you're replacing the defective gene. The size of the trans gene that's required doesn't make it amenable to current gene therapy viral vectors. Finally, because there are multiple gene mutations, it's not amenable to gene editing." #Nuevocor #DrugDiscovery #Innovation #Biotech #Cardiology #GeneTherapy #GeneticCardiomyopathy #HeartFailure #LMNACardiomyopathy #PrecisionMedicine #RareDisease #ClinicalTrials nuevocor.com Download the transcript here

Al Gianchetti, CEO of Nuevocor,  discusses genetic cardiomyopathies, an underdiagnosed condition that might explain multigenerational heart failure. Current treatments include beta blockers, pacemakers, and heart transplants but do not alter the course of the disease.  Because of multiple gene mutations, Nuevocor is developing a precision therapy for LMNA-related cardiomyopathy based on mechanobiology that prevents cardiac cell death using a method different from gene replacement and gene editing. Al explains, "We're focused on genetic cardiomyopathies, and our lead drug, NVC001 in particular, is focusing first on LMNA cardiomyopathy, and LMNA is a specific gene mutation. We're focusing on LMNA-related cardiomyopathy for several reasons. First of all, it's one of the most severe forms of genetic cardiomyopathy, with a very poor prognosis for patients and their families who have the genetic mutation."   "The second reason is that although it's a rare disease, it affects a fairly large number of people. For example, about 50,000 people in the United States are estimated to have LMNA-related cardiomyopathy. The third reason is that this disease is not amenable to gene replacement strategies where you're replacing the defective gene. The size of the trans gene that's required doesn't make it amenable to current gene therapy viral vectors. Finally, because there are multiple gene mutations, it's not amenable to gene editing." #Nuevocor #DrugDiscovery #Innovation #Biotech #Cardiology #GeneTherapy #GeneticCardiomyopathy #HeartFailure #LMNACardiomyopathy #PrecisionMedicine #RareDisease #ClinicalTrials nuevocor.com Listen to the podcast here

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. The landscape of these industries is currently experiencing a wave of transformative changes, from strategic corporate relocations and groundbreaking partnerships to pivotal regulatory approvals and promising clinical trial outcomes. Let's begin with a significant corporate strategy shift. Lundbeck has made the decision to relocate its U.S. headquarters to Boston. This move is strategically designed to establish a global innovation hub that enhances its capabilities in neuroscience and artificial intelligence. Boston's burgeoning status as a biotech innovation nexus makes it an attractive destination, offering access to a rich ecosystem of research institutions and technological expertise. Such an environment is crucial for facilitating advancements in neurological research, reflecting the city's growing influence in the biotech world. Turning to regulatory updates, Merck & Co. and Eisai have achieved a notable milestone with FDA approval for their combination therapy of Welireg (belzutifan) and Lenvima (lenvatinib) as a second-line treatment for clear cell renal cell carcinoma. This approval is particularly significant as it marks the introduction of the first HIF-2α inhibitor combined with a tyrosine kinase inhibitor, providing a novel therapeutic approach for patients who have limited options after initial therapies. The combination therapy exemplifies the promise of innovative treatments in oncology, broadening therapeutic landscapes and potentially improving patient outcomes. In business development news, Genentech has partnered with Earendil Labs in a groundbreaking agreement valued at over $1.5 billion to co-develop AI-driven bispecific antibodies for cancer treatment. This collaboration underscores the industry's shift towards harnessing artificial intelligence and machine learning to optimize drug discovery and development processes. By leveraging AI, this partnership aims to accelerate the creation of targeted therapies with enhanced efficacy, highlighting a transformative trend in the industry. Clinical trials continue to yield encouraging results. Johnson & Johnson and Legend Biotech's CAR-T therapy, Carvykti (cilta-cel), has demonstrated a five-year survival benefit in a Phase 2 study for multiple myeloma patients. This milestone emphasizes the long-term efficacy of CAR-T treatments in hematological malignancies, showcasing their potential to significantly extend patient survival rates. In parallel, Regenxbio has reported positive three-year durability data for its gene therapy candidate RGX-314 in non-proliferative diabetic retinopathy, signaling advancements in gene therapy delivery systems that could revolutionize treatments for chronic conditions. Financial activities within the sector also reflect robust investment trends. Sadarx Pharmaceuticals has raised $446 million through an IPO to advance its siRNA and complement-mediated disease pipelines, indicative of sustained investor interest in RNA-based therapeutics. Additionally, Kasvu Therapeutics secured €30 million in Series A funding to propel its neuroplastogen depression candidate KTX-0141 forward, highlighting a growing focus on mental health therapeutics. However, challenges persist amid these advancements. GSK faces scrutiny from the UK self-regulatory body over transparency issues related to vaccine compliance, while Eli Lilly received criticism concerning promotional language about Orforglipron GLP-1 press releases. These incidents underscore the necessity of ongoing regulatory vigilance to maintain industry standards. Overall, these developments reflect a dynamic landscape characterized by strategic realignments, pioneering scientific collaborations, and significant regulatory milestones. The integration of AI into drug development processes and continuous innovation in therapeutic modalities like CAR-T and gene therapies are poised to enhance patient care significantly. As companies navigate complex regulatory landscapes and foster strategic partnerships, these efforts collectively highlight the sector's commitment to addressing unmet medical needs through advanced science and technology. In summary, the pharmaceutical and biotech industries are poised at an exciting juncture where scientific innovation meets strategic business acumen. With each advancement comes the potential not only to enhance patient care but also to redefine healthcare paradigms globally. As these sectors continue to evolve, their contributions promise a future where biotechnology plays an increasingly pivotal role in improving quality of life worldwide. Thank you for tuning into Pharma Daily—your source for vital updates from the frontiers of pharmaceutical and biotech innovation. Stay informed and join us next time as we explore more breakthroughs shaping healthcare's future.Support the show

HEADLINES:• Truck Movement Will Be Banned During Peak Hours On Major Dubai Roads From October 1 • 10-Year-Old Youssef Haider Completes Life-Saving Gene Therapy Treatment in Dubai • Dubai Run Clubs Pay Tribute To Sheikh Ahmed bin Rashid Al Maktoum • Hundreds Gathered In Dubai For The Funeral Of A Mum Who Died Giving Birth

Results for weight loss treatment combo cagrilintide and semaglutide; positive results for treatment of rare form of ALS, and for sefaxersen in adults with primary IgA nephropathy; gene therapy approved for Sanfilippo syndrome; and Lyrfigtu gains approval for FGFR2+ advanced cholangiocarcinoma.

Hear from Ryan McCreery and John Lee, two researchers working toward intercepting hearing loss early. Both will present on their work at the 2026 ASHA Research Symposium on Hearing, to be held at the ASHA Convention and streamed online.First, hear McCreery of Boys Town National Research Hospital talk about his research on intervention for children with mild hearing loss. Then, Lee of Regeneron Pharmaceuticals discusses a gene therapy clinical trial that's seeing participants regain hearing.TranscriptLearn More:ASHA Research Symposium on HearingASHA Voices: How Do Audiologists Counsel Patients Interested in Gene Therapy?ASHA Voices: Researchers Discuss Speech in Noisy Environments, Spatial Awareness, and Bilateral Cochlear ImplantsSupport for this episode of ASHA Voices comes from AMN Healthcare.

Gene therapy has been used to treat many previously incurable conditions, like correcting deadly genetic disorders or giving children born with hearing loss the ability to hear. The field has massive potential, but there are still many economic, regulatory, and scientific hurdles to overcome to adequately address a variety of both rare and more common conditions. In this episode, host Samantha Laine Perfas talks with gene editing researcher David Liu and ear, nose, and throat surgeon Eliot Shearer about how gene therapy can help treat diseases that were previously considered too rare to research.

Professor Robin Ali, Director of the Centre for Gene Therapy & Regenerative Medicine, KCL.

Beacon Therapeutics reported positive results from the Phase 3 pivotal trial of laru-zova, its potential gene therapy treatment for X-linked retinitis pigmentosa. A significant proportion of patients improved dim-light vision by 15 or more letters after treatment with either high or low doses of laru-zova compared to the group that was not treated, according to preliminary results from the VISTA clinical trial. Beacon plans to begin seeking FDA approval later this year. If approved, this would be the first treatment available for XLRP, one of the more severe forms of retinitis pigmentosa that predominantly affects males.

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 period of profound transformation within the industry, marked by scientific advancements, strategic acquisitions, and a shifting regulatory landscape that promises to redefine the future of healthcare. Telix Pharmaceuticals has made headlines with its acquisition of ITM Isotope Technologies Munich SE, valued at up to $2.35 billion. This move positions Telix at the forefront of the radiopharmaceutical sector—a field gaining momentum due to its precision medicine potential in oncology. Radiopharmaceuticals use radioactive substances for targeted cancer therapies, offering promising alternatives to traditional treatments by minimizing side effects and enhancing therapeutic efficacy. This acquisition could revolutionize oncology treatment protocols, providing more precise diagnostic and therapeutic options for patients. In another significant development, Ultragenyx has secured FDA approval for Fayuvi, marking the first-ever gene therapy for Sanfilippo Syndrome Type A. This devastating neurodegenerative condition previously had no effective treatment. Gene therapies like Fayuvi represent a paradigm shift in addressing genetic disorders by tackling root causes at the molecular level rather than merely alleviating symptoms. This milestone not only offers hope to affected families but also sets a precedent for future gene therapies targeting similar rare diseases. China's ambitious five-year plan to capture 25% of global innovative drug approvals by 2030 underscores its growing role in the pharmaceutical industry. With a focus on expanding research and development capabilities across multiple disease areas, this initiative aims to foster international collaborations and accelerate novel therapies' introduction worldwide. The plan reflects China's commitment to becoming a major player in pharmaceutical innovation, potentially reshaping global market dynamics. Alkermes' recent clinical trial results highlight another promising development in ADHD treatment. Its orexin agonist ALKS 7290 has shown potential in Phase 1b trials, with Phase 2 trials underway. Orexin agonists could represent a new approach to managing ADHD beyond traditional stimulant medications by addressing underlying neurobiological pathways rather than just symptoms. Beacon Therapeutics is preparing for an FDA filing following successful Phase 2/3 trials of laruparetigene zovaparvovec for X-linked retinitis pigmentosa (XLRP), a progressive eye disease leading to blindness. This advancement underscores gene therapy's potential to significantly slow disease progression or restore vision in patients with inherited retinal diseases, offering renewed hope for affected individuals. Strategically, Novo Nordisk is realigning its focus through acquisitions like Kallyope's early-stage obesity assets and Bayer's divestment of the cancer drug Stivarga to Grunenthal. These transactions reflect ongoing efforts within companies to concentrate on core competencies and address competitive pressures effectively. AstraZeneca's $30 million investment in its Wuxi facility aims to expand cardiovascular drug production capacity, responding to rising global demand driven by increasing incidence rates. Such investments highlight a broader trend towards enhancing production capabilities in response to changing market needs. Eli Lilly has also made strides in oncology with FDA approval of an oral SERD combination therapy involving Inluriyo and Verzenio. This combination offers longer progression-free survival for breast cancer patients compared to monotherapy, representing an important advancement in developing patient-centric therapeutic regimens. Finally, technological advancements continue redefining clinical trial processes with automation and digital innovations accelerating drug development timelines and improving data integration capabilities. These efforts will likely lead to transformative impacts on drug development processes and patient outcomes globally. Overall, these developments illustrate an industry at the forefront of innovation, grappling with complex challenges such as regulatory changes and competitive pressures while continuously pushing the boundaries of medical science to improve patient care. As biotechnologies advance and new therapeutic areas are explored, stakeholders across the sector must navigate these changes strategically to capitalize on emerging opportunities. Thank you for tuning into Pharma Daily. Stay informed and engaged as we continue bringing you the latest insights from the pharmaceutical and biotech world—it's an exciting time for science and medicine alike!Support the show

The promise of cell and gene therapies is big. They have the ability to address the underlying genetic causes of...[…]

The promise of cell and gene therapies is big. They have the ability to address the underlying genetic causes of...

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 groundbreaking advancements and strategic shifts shaping the future of this dynamic industry. The FDA has recently approved Ultragenyx's gene therapy, Fayuvi, for a rare neurodegenerative disorder, marking a significant milestone in the realm of precision medicine. Gene therapies like Fayuvi offer a beacon of hope for conditions with limited treatment options. This approval underscores the burgeoning promise of personalized medicine, where treatments are meticulously tailored to individual genetic profiles, vastly improving patient outcomes. Across the Atlantic, Europe's Committee for Medicinal Products for Human Use has given the nod to several new medicines, including notable contributions from Novo Nordisk and AstraZeneca. Novo Nordisk's latest hemophilia A treatment exemplifies its ongoing commitment to enhancing therapeutic solutions for blood disorders. These CHMP endorsements are pivotal; they pave the way for European Commission approvals, thereby bringing these advanced treatments one step closer to patients throughout Europe. In a significant breakthrough for diabetes-related complications, Bayer's Kerendia has become the first drug in three decades approved by the FDA specifically for chronic kidney disease linked to type 1 diabetes. This approval highlights a critical advancement in addressing long-standing unmet medical needs, emphasizing the necessity of continuous innovation in chronic disease management. However, not all developments have been straightforward. Bristol Myers Squibb has opted to discontinue its degrader-antibody conjugate following an evaluation of Phase 1 data. This decision illustrates the high stakes of drug development where early-stage data critically inform strategic directions and resource allocations. It serves as a reminder of the inherent risks involved in pioneering new therapeutic approaches. In obesity management, oral GLP-1 receptor agonists are seeing fluctuating prescription trends as market competition intensifies between Novo Nordisk's Wegovy pill and Eli Lilly's Foundayo. A weekly tracker launched by Fierce Pharma seeks to illuminate these trends, offering valuable insights into market dynamics and the evolving landscape of weight-loss therapies. From an investment perspective, Electra Therapeutics' $350 million IPO marks investor confidence in biotech companies focused on inflammation-related diseases. The funds raised are earmarked for advancing its monoclonal antibody Ipsoprubart aimed at treating immune-mediated conditions. This trend towards leveraging financial markets reflects a broader industry movement aimed at fueling innovation and expanding therapeutic portfolios. Notably, despite geopolitical tensions, drug licensing deals in China remain largely unaffected by new U.S. Treasury rules. This stability is crucial for maintaining cross-border collaborations essential to global drug development efforts. On another front, Xenon Pharmaceuticals has paused its phase 3 depression trials due to psychosis events, underscoring the complexities inherent in psychiatric drug development. This pause highlights the rigorous safety evaluations necessary during clinical trials to ensure patient welfare is prioritized. Exploring broader industry trends, cyclic peptides are gaining attention for their potential to break into new drug frontiers despite manufacturing challenges. Their versatility across various therapeutic areas underscores their promising future in drug development. Moreover, the FDA's open stance towards emerging fields like psychedelics reflects a progressive regulatory approach towards novel therapeutic avenues while ensuring safety standards are upheld. This openness holds promise for groundbreaking treatments targeting complex conditions such as PTSD and depression. These developments collectively signify a dynamic period within the pharmaceutical and biotech sectors marked by rapid innovation and strategic recalibration. As companies adeptly navigate regulatory landscapes and clinical challenges, successful approvals and strategic pivots underscore their resilience and commitment to advancing patient care through cutting-edge science. The continual evolution of technologies like gene therapy and monoclonal antibodies promises transformative impacts on disease management and patient outcomes as we move forward into an exciting future for healthcare advancements globally. Thank you for listening to Pharma Daily. Stay tuned for more updates on how these developments continue to shape our industry and transform patient care around the world.Support the show

Join us as Dr. Noah Davidsohn, Co-Founder and Chief Scientific Officer of Rejuvenate Bio, reveals how systemic multi-gene therapy and in vivo partial cellular reprogramming can reverse age-related chronic diseases and double remaining lifespan. Discover the breakthrough science of AAV vector delivery, FGF21 and sTGFβR2 synergy, and companion animal clinical translation.What if a single gene therapy injection could simultaneously treat heart failure, kidney disease, and diabetes? In this episode, we sit down with Dr. Noah Davidsohn, CSO and Co-Founder of Rejuvenate Bio, spun out of George Church's lab at Harvard Medical School. Dr. Davidsohn breaks down their landmark 2024 study in Cellular Reprogramming, which demonstrated a 109% increase in the remaining lifespan of extremely aged (124-week-old) wild-type mice using systemic AAV9 delivery of Yamanaka factors (OSK). He explains the mechanics of intermittent doxycycline pulsing to avoid tumor risks, the power of multi-gene master regulators like FGF21 and sTGFβR2 in RJB-01, and how treating companion dogs with Mitral Valve Disease serves as the ultimate translational bridge to human clinical trials.

Interview with Dana S. Guggenheim, BA, and Justin S. Golub, MD, MS, authors of Newborn Hearing Screening in the Otoferlin Gene Therapy Era. Hosted by Paul C. Bryson, MD, MBA. Related Content: Newborn Hearing Screening in the Otoferlin Gene Therapy Era

Interview with Dana S. Guggenheim, BA, and Justin S. Golub, MD, MS, authors of Newborn Hearing Screening in the Otoferlin Gene Therapy Era. Hosted by Paul C. Bryson, MD, MBA. Related Content: Newborn Hearing Screening in the Otoferlin Gene Therapy Era

Guest: Lawrence Lustig, M.D. On this episode of Advances in Care, Erin Welsh speaks with Dr. Lawrence Lustig, otolaryngologist-in-chief at NewYork-Presbyterian and Columbia. Dr. Lustig has spent decades working to advance various interventions to treat genetic deafness and after years of research, his team was the first to show that gene therapies could be effective at reversing hearing loss in mouse models. Dr. Lustig focused his initial study on otoferlin, a gene that is missing in people with a rare form of genetic deafness, and how to safely deliver it back to patients. Starting with mouse models and solving for the uniquely large size of the gene, they discovered that packaging otoferlin with an adeno-associated virus and injecting it into the ear completely reversed deafness. From there, they began the process of trialing this approach in humans at NewYork-Presbyterian and Columbia. The trials were successful, leading to natural hearing restoration in most patients, and in April 2026 the work culminated with an FDA approval of the first ever gene therapy for genetic deafness based on the research pushed forward by Dr. Lustig and his team. Dr. Lustig's team has trialed the therapy in patients across a range of ages, from six years old to …

In this episode, Kleopas Kleopa speaks with Jo Ng about gene therapy for rare neurological disorders.

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 advancements and challenges that are shaping the future of drug development, regulatory landscapes, and industry innovations. Starting with a major milestone in personalized cancer therapy, the FDA has expanded Bayer's approval for Hyrnuo (zipalertinib) to include its use as a first-line treatment for HER2-mutant non-small cell lung cancer (NSCLC). This approval is based on promising Phase 1/2 clinical data, highlighting zipalertinib's role as a tyrosine kinase inhibitor targeting specific genetic mutations. This development is part of a broader trend towards precision medicine in oncology, allowing treatments to be specifically tailored to patients' genetic profiles. The potential impact on patient care is substantial, offering more effective treatment options for those with this particular HER2 mutation. In parallel, Johnson & Johnson's Imbruvica (ibrutinib) regimen has been endorsed by NICE for mantle cell lymphoma. These advancements represent a significant shift towards integrating precision medicine into oncology, aiming to improve outcomes by focusing on individual patient needs. On the business front, Samsung Biologics has secured a $262 million manufacturing deal with an unnamed European pharmaceutical company. This highlights an increasing demand for biologics manufacturing capabilities and reflects a growing reliance on contract development and manufacturing organizations (CDMOs) to meet complex therapeutic needs. Meanwhile, EMD Serono's acquisition of PostEra's AI-discovered fertility programs marks a pivotal move towards incorporating artificial intelligence in drug discovery, potentially revolutionizing women's health and fertility treatments. Clinical trial successes continue to drive momentum in the sector. AbbVie's Qulipta (atogepant) achieved its primary endpoint in a Phase 3 trial focused on menstrual migraines through CGRP receptor antagonism. This positions Qulipta as a promising new therapy within neurological disorders. Similarly, Rezera's Ruvonoflast met its primary endpoint in treating peripheral artery disease via NLRP3 inhibition, showcasing innovative anti-inflammatory approaches within cardiovascular medicine. Investment remains robust across the industry landscape. Frazier Life Sciences has successfully raised $1.1 billion to support small to mid-cap biotech companies, aiming to foster innovation and support emerging firms through critical phases of drug development. Additionally, CordenPharma's €80 million investment in enhancing aseptic fill-finish capacity illustrates strategic expansions within pharmaceutical manufacturing infrastructure. However, regulatory challenges persist. NICE's rejection of Gilead Sciences' lenacapavir due to cost concerns underscores ongoing debates around drug pricing and accessibility within healthcare systems. Furthermore, Biohaven Pharmaceuticals faces a partial clinical hold by the FDA on its epilepsy drug trial due to safety concerns. These instances highlight the rigorous scrutiny required throughout drug development processes. In response to these challenges, companies are increasingly adopting sophisticated strategies to navigate the evolving regulatory landscape. The "most favored nation" pricing policy in the U.S., designed to align domestic drug prices with those abroad, is prompting pharmaceutical firms to reassess their market strategies amid shifting economic conditions. On an optimistic note, Encoded Therapeutics has raised $275 million in Series F financing aimed at advancing gene therapies for conditions like Dravet syndrome. This substantial investment underscores confidence in gene therapy as a transformative approach for treating complex neurological and rare diseases. Finally, Ionis Pharmaceuticals achieved a breakthrough with its therapy approval for Alexander disease, illustrating the growing emphasis on targeting genetic disorders through precision medicine approaches. Such developments reflect both the dynamic nature of scientific innovation and the inherent challenges that accompany it. As these advancements unfold across various therapeutic areas and technological innovations continue to transform industry practices, it becomes increasingly clear that strategic planning and investment in research are crucial for translating scientific breakthroughs into tangible patient benefits. The ability of companies to innovate while ensuring compliance and economic viability will be pivotal as they strive to redefine treatment paradigms within this highly competitive sector. Thank you for joining us today on Pharma Daily. Stay tuned for more updates as we continue to bring you the latest insights from the ever-evolving pharmaceutical and biotech industries.Support the show

Send us Fan MailCAR-T therapy can be extraordinarily powerful against cancer - but there's a catch. We currently have to take a patient's T cells out of their body, genetically engineer them in a laboratory, manufacture billions of cells, and then put them back. But what if we could skip the factory entirely - and genetically program the T cells inside the patient's body?Dr. Luke Russell, Ph.D. is President of Vyriad ( https://vyriad.com/ ), a biotechnology company developing viral therapies designed to deliver genetic payloads directly to cells in the body.Dr. Russell has spent much of his career at the intersection of cancer immunotherapy, oncolytic virotherapy, gene delivery, and biotechnology strategy. Before moving into executive leadership, his research focused on using viruses to attack tumors and stimulate antitumor immunity. He earned his PhD in Neuroscience from The Ohio State University, where his work included developing an immunostimulatory oncolytic herpesvirus for brain cancer, and later earned an MBA in Finance and Strategy from Carnegie Mellon University's Tepper School of Business.Dr. Russell joined Vyriad in 2018 and has held increasingly senior roles spanning research, alliance management, business development, operations and corporate strategy. During that time, he has helped build strategic relationships with major pharmaceutical companies including Novartis, Regeneron and Merck KGaA.And now, as President, Dr. Russell is helping lead Vyriad's effort to develop a new generation of viral medicines - including VV169, the company's third clinical program, and its G-Link platform, which is designed to give viral vectors a modular, “plug-and-play” ability to retarget which cells they deliver their genetic payloads to.At the center of today's conversation is a particularly provocative possibility: in-vivo CAR-T - using a targeted viral vector to genetically reprogram a patient's own T cells inside the body.#CAR T #CAR TCellTherapy #Cancer #CancerResearch #Immunotherapy #ImmunoOncology #GeneTherapy #GeneDelivery #CellTherapy #InVivoCAR T #CAR TResearch #TCells #ViralVectors #OncolyticVirotherapy #Biotechnology #Biotech #PrecisionMedicine #MedicalInnovation #DrugDevelopment #FutureOfMedicine #Vyriad #LukeRussell #ProgressPotentialAndPossibilities #PPPSupport the show

Inherited retinal diseases can profoundly affect a child's vision and development, yet there are no approved drug treatments for most of these conditions in children. In this episode of Eye on Innovation, host Carey Powers speaks with Carmen Carrichio, CEO and founder of Raresight, about why pediatric inherited retinal diseases have historically received less attention and investment than adult diseases — and how advances in genetic testing and clinical assessment are beginning to change that.In this episode, learn about: Why treatments developed for adults cannot simply be applied to childrenWhere gene therapy may fit into the future of careRaresight's approach to developing treatments designed to remain in the eye longerHow these treatments will change the trajectory of a child's life RESOURCES: Carey Powers: Carey Powers, MBA | Ophthalmology Innovation Source  

In this episode of Reimagine Childhood from the Early Childhood Christian Network, host Monica Healer talks with grandmother Kate Mobell, whose grandson Shepherd was diagnosed with Duchenne muscular dystrophy just before kindergarten, and Dr. Diana Castro, a board-certified neurologist and neuromuscular physician. They explain why early educators' daily observations can help families pursue quicker evaluations and referrals, especially when concerns have been minimized. Dr. Castro outlines common early signs of Duchenne—delayed walking, speech delays, fatigue at recess, frequent falls, difficulty with stairs or jumping, toe walking, enlarged calves, and Gower's sign—and notes possible cognitive and learning impacts. The conversation also covers why early intervention matters, how treatments like gene therapy aim to increase dystrophin, and how teachers can document specific behaviors, encourage OT/PT or specialist evaluation, plan for safety needs, and compassionately support families and classmates through education and community resources like CureDuchenne.   00:00 Welcome and Purpose 02:34 Shepherd Diagnosis Story 08:17 Signs Teachers Can Spot 18:47 Learning Library Promo 19:50 Treatments and Gene Therapy 22:42 Access to Gene Therapy 25:05 Schools as Early Advocates 28:01 Supporting Families at School 35:13 Teacher Action Steps and Wrap Up Download milestone lists by age from birth to age 5 HERE! These can be super helpful in documenting what you see! https://cureduchenne.org/ has tons of resources for parents who might have a recent diagnosis. https://neuromdcenter.com/ is Dr. Castro's clinic in Dallas. She says patients from all over the world! Connect with Dr. Castro on Instagram https://www.instagram.com/dianacastromd/ or Facebook https://www.facebook.com/DianaCastroMD

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 updates that are shaping the future of medicine and patient care. First on our agenda is uniQure's ambitious step toward accelerated U.S. marketing approval for AMT-130, a gene therapy targeting Huntington's disease. This move signifies a potential breakthrough in treating rare neurological disorders with genetic roots. The approval of AMT-130 would highlight gene therapy's transformative power, not just for Huntington's disease but also for a broader range of genetic conditions, offering hope to patients where traditional treatments have fallen short. In the realm of oncology, Summit Therapeutics, in collaboration with Akeso, has reported that their bispecific antibody ivonescimab has surpassed Merck's Keytruda in a Phase 3 trial for non-small cell lung cancer. The trial revealed superior overall survival and progression-free survival rates, marking a significant milestone in cancer treatment. Bispecific antibodies like ivonescimab open new avenues for targeting multiple pathways simultaneously, potentially leading to more effective therapies with reduced side effects. Collaboration continues to be a pivotal strategy in drug development. GSK and Hutchmed have formed a licensing agreement for HMPL-A830, an innovative cancer therapy. This partnership involves an upfront payment of $110 million, with the potential to reach $1.3 billion based on performance milestones. Such alliances highlight the importance of strategic partnerships in accelerating therapeutic advancements and expanding the arsenal of available cancer treatments. Moving to regulatory milestones, Samsung Bioepis has secured approval in Japan for its biosimilar ustekinumab, aimed at treating moderate-to-severe Crohn's disease by targeting IL-12/IL-23 pathways. This approval is part of a larger trend towards biosimilars as cost-effective alternatives to biologics, increasing access to essential treatments for autoimmune diseases. In cardiovascular health, Everest Medicines has received China NMPA approval for Cardamyst (etripamil), a self-administered nasal spray for paroxysmal supraventricular tachycardia. The approval underscores innovations in patient-centric drug delivery systems that offer easier administration methods and empower patients to manage their conditions effectively. On the business front, Fortrea has acquired Worldwide Clinical Trials' early-phase division for $45 million. This acquisition aims to bolster Fortrea's clinical research organization platform, reflecting the growing demand for comprehensive clinical pharmacology services that can accelerate drug development timelines. Meanwhile, Pfizer's divestment of its Seagen antibody-drug conjugate PF-08046031 to Medicus Pharma in a deal exceeding $1 billion exemplifies strategic realignments within large pharmaceutical companies. These transactions allow companies to streamline operations and focus on core therapeutic areas where they can make the most impact. Research advancements are also making headlines as Revolution Medicines reports promising Phase 1/2 data for Rasonque (daraxonrasib) in NSCLC patients with KRAS mutations. KRAS has been notoriously difficult to target, so these findings highlight Rasonque's potential as a breakthrough small molecule therapy addressing critical needs in oncology. Despite these strides forward, challenges remain evident. Ultragenyx's GTX-102 did not meet primary or secondary endpoints in its Phase 3 Angelman syndrome study. This setback highlights the complexities involved in developing effective treatments for rare neurological conditions and underscores the inherent risks in high-stakes clinical trials. Turning our attention back to regulatory dynamics, FDA deliberations over Replimune's melanoma treatment revealed nuanced decision-making processes balancing clinical evaluation with strategic considerations. Even amidst internal disagreements, FDA leadership endorsed an accelerated nod for Replimune's therapy—an illustration of how complex these processes can be. In terms of funding innovation, ARPA-H's substantial $125 million investment into personalized RNA-based drug production reflects a commitment to advancing RNA therapeutics despite recent challenges. The initiative could become a catalyst for new approaches in personalized medicine by leveraging RNA technologies to tailor treatments more precisely to individual profiles. Additionally, Roche is making headway in obesity treatment with its UCN2 analog, which shows promise in reducing weight without sacrificing lean muscle mass—an essential factor given obesity's global health implications. This development may introduce new mechanisms of action that could revolutionize existing therapies by overcoming metabolic challenges inherent in obesity treatment. Overall, these developments underscore a dynamic period within the pharmaceutical and biotech sectors characterized by rapid scientific progress and strategic realignments responding to evolving market demands. As companies navigate these changes, their ability to leverage new technologies and refine their strategic focus will be crucial in maintaining competitive advantage and driving future growth—all while aiming to improve patient outcomes globally.Support the show

In this episode of the Phelan-McDermid Syndrome Podcast: Sharing Research, Progress, and Hope, Lauren is joined by Dan Gallo, Executive Vice President and Head of Clinical Development and Medical Affairs at Jaguar Gene Therapy, and Tessa Clarkson, Clinical Development Consultant at Jaguar Gene Therapy.They discuss Jaguar's Developmental Milestone Survey, a study designed to better understand how people with Phelan-McDermid syndrome gain, lose, and potentially regain meaningful developmental skills. The goal is to identify developmental patterns that could help researchers measure meaningful treatment effects and inform future clinical trials—including potentially reducing the need for placebo-controlled designs.The conversation covers the survey's scientific rationale, lessons from a similar approach in Rett syndrome, eligibility and participation, and why family participation is especially important as Jaguar prepares for discussions with the FDA about future JAG201 development.JAG201 update: As of August 21, 2026, Jaguar reported that Cohort 1 dosing in its Phase 1/2 trial was complete and Cohort 2 dose escalation was underway. Jaguar described early indications of clinical benefit across neurodevelopmental domains and an emerging safety profile with no treatment-related serious adverse events reported to date, while emphasizing that more follow-up and complete analysis are needed before conclusions can be drawn.Learn more about the Developmental Milestone Survey: pmscaregiverstudy.com

In this episode, we review the current status of gene therapy for inherited retinal degenerations with Dr. Ninel Gregori, Professor of Clinical Ophthalmology, Bascom Palmer Eye Institute

A special conversation with Troy Rohn on his book “Brain Medicine: Breakthroughs in Gene Therapy for Anxiety & Other Neuropsychiatric Disorders.”Most anxiety treatments manage the symptoms — but what if you could actually rewire what's causing them? That's the question driving neuroscientist Troy's book, Brain Medicine, which argues gene therapy could move anxiety treatment beyond present medications toward fixing the underlying brain circuitry itself. He talks about why he wrote the book, the ethical questions this science raises — access, cost, and where treatment ends and enhancement begins — and also talks about his own lifelong struggle with anxiety.Support KAJ Masterclass — help sustain independent editorial work: https://kajmasterclass.com/support=========================================About KAJ Masterclass MediaKAJ Masterclass Media is home to a video-first, live-first, editorially independent conversation ecosystem exploring leadership, business, AI, careers, health, creativity, and the evolving human experience through thoughtful, unscripted conversations grounded in lived experience, clarity, and real-world insight.This show is part of the KAJ Masterclass ecosystem, which includes The KAJ Masterclass LIVE, The Author's Voice with KAJ, and a growing collection of editorially independent conversation platforms.Every conversation is designed to leave you with something meaningful to think about, understand, or apply.

Wellness trends move fast, yet volume two of The Next Frontier Files proves some predictions age well. Revisiting year two of the show, host Jenn Trepeck tracks the guests who forecasted metabolic health, gut health, and body positivity long before they became mainstream conversations, and shows how precision medicine and permission became the defining threads of an entire year.Jenn Trepeck brings listeners of Salad with a Side of Fries a look back at the guest predictions that shaped the year two, August 2020 - August 2021, grading them against what really happened since.What You Will Learn in This Episode:✅ How telehealth and virtual training went from pandemic workaround to a permanent part of everyday healthcare✅ Why metabolic health and gut health became two of the most repeated topics in wellness over the following years✅ What guests predicted about wearable technology, gene editing, and the future of human biology✅ How body positivity and permission-based wellness reshaped the conversation around health and self-imageThe Salad With a Side of Fries podcast, hosted by Jenn Trepeck, explores real-life wellness and weight-loss topics, debunking myths, misinformation, and flawed science surrounding nutrition and the food industry. Let's dive into real-life wellness and weight loss, including drinking, eating out, and skipping the grocery store.TIMESTAMPS:00:00 Year two overview explains how telehealth shifted from workaround to permanent wellness trends infrastructure04:42 Chad Levy discusses virtual training, and Amy Marshall shares her perspective on Telehealth and Zoom technology06:55 Chris Burres introduces longevity as a frontier, and Natasa Billeci mentions AI and propels the precision medicine conversation08:49 Toxin exposure and cellular absorption enter the conversation through Tim James' product launch12:01 Corey Phelps highlights her views on meditation and breathwork12:58 Dr. Casey Means discusses metabolic health tools and using technology to return to basics15:53 Gut health gets its first mention by Mike Millner, predicting it is only 2% understood16:53 Dr. Anthony Balduzzi predicts gene therapy, CRISPR, and implantable technology merging with human biology18:25 Catharine Arnston embraces preventative medicine19:13 Sarah Nilsen expounds about CBD and cannabinoid research20:12 Jay Shifman comments on BMI and asks why is it still being used21:17 Sarah Malouf and Farah M. Green raise body positivity and the pressure to redefine beauty standards23:20 Jenn closes by tying the year's predictions together under precision and permissionKEY TAKEAWAYS:

What happens when life-changing therapies cost millions of dollars, but the healthcare system was never built to manage them? In this episode of Entrepreneur Rx, host John Shufeldt sits down with Annie Collins, Co-founder and Chief Commercial Officer of Aradigm, to discuss her journey from healthcare consulting and venture capital to building a company tackling one of the most complicated challenges in modern medicine. Annie shares how her early interest in medicine led her from pre-med studies to Bain & Company and eventually to Andreessen Horowitz, where she invested in early-stage healthcare and life science companies. While at a16z, she became increasingly focused on the disconnect between groundbreaking therapies and the healthcare system's ability to sustainably pay for and deliver them. Eventually, Annie and her co-founders stopped looking for a company solving the problem and decided to build one themselves. The conversation explores the complex world of cell and gene therapies, where a single treatment can cost hundreds of thousands to more than $4 million. Annie explains how Aradigm works across payers, providers, manufacturers, and patients to create a more predictable and sustainable model while helping patients navigate an incredibly complicated treatment journey. John and Annie also discuss what it was like to leave one of the world's leading venture capital firms to become a founder, why entrepreneurship turned out to be even harder than she expected, and what she has learned moving from evaluating companies to actually building one. They also talk about persistence in venture capital, female founders and leadership, and why having other founders and mentors to lean on matters when the startup journey gets lonely. About Annie Collins: Annie Collins is a Co-founder and Chief Commercial Officer at Aradigm, where she oversees launches of new programs, the success of existing customers, and operational execution of Aradigm's program. She began her career at Bain & Company as a strategy consultant for large healthcare and life science companies before joining Andreessen Horowitz as an investment partner focused on early-stage healthcare and life science companies. While there, she was introduced to the commercial complexities posed by high-cost drugs like cell and gene therapies and the largely unsolved challenge of managing those therapies on the medical benefit. That experience ultimately led her and Will Shrank to leave a16z and start Aradigm alongside Spencer Carrucciu. About Aradigm: Aradigm is an incentive-aligned, cost-plus platform managing the financial and clinical complexities of cell and gene therapies (CGTs). Our mission is to create sustainable, affordable access to life-saving medicines through a novel financial and delivery model that brings together every stakeholder — payers, providers, manufacturers, and, most importantly, patients. About Xcellerant Ventures:  Xcellerant Ventures is a venture capital firm that invests in innovative companies across healthcare, technology, defense, and other emerging industries. Through multiple funds and investment vehicles, including the Jetstream Venture Fund, Xcellerant partners with visionary founders, provides strategic guidance, and helps connect investors with opportunities in the private markets. About Deal Titan:  This week's episode is brought to you by Deal Titan. Deal Titan is an AI-powered platform that helps investors evaluate early-stage companies faster by turning pitch decks into structured diligence reports covering the market, team, financials, competition, and key risks. Use code EntrepreneurRx for a free solo membership, including 10 full analysis reports.

Have you heard about the radioactive wolves in Chernobyl? Or the latest gene-editing tech that's curing rare diseases? Or that male birth control might be on the horizon? No?!Well, get ready, because while we've been busy living our lives, genomics has been moving – fast! Things that once read like science fiction are moving on over to the non-fiction section.This season on Nice Genes!, join Dr. Kaylee Byers as she engages in some big genomics breakthroughs – from personalized cancer treatments, to how our bodies react in space, to how animals adapt to the most extreme environments, and whether we can actually change how we age.It's a whole new gene-eration (if you will) of discoveries that you won't want to miss!New episodes every two weeks, starting September 8th. Make sure you follow Nice Genes! so you don't miss an episode!

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 of the latest advancements and achievements across this dynamic industry, where innovation is reaching new heights and regulatory landscapes are continuously evolving. In a groundbreaking moment for gene therapy, Ultragenyx has secured the first FDA approval for its gene therapy product, Genglycos (DTX401), aimed at treating Glycogen Storage Disease Type Ia. This rare metabolic disorder has long posed significant challenges due to the body's inability to convert glycogen into glucose. Using an adeno-associated virus vector to deliver the therapeutic gene, Genglycos has shown significant clinical efficacy in Phase 3 studies, offering new hope for patients with limited options. This achievement underscores gene therapy's potential to transform the management of metabolic diseases and sets a precedent for future innovations in treating genetic disorders. Shifting focus to monoclonal antibodies, Regeneron has received FDA approval for Pasatru (Garetosmab), a treatment for Fibrodysplasia Ossificans Progressiva (FOP), a rare and debilitating bone disease. Pasatru targets Activin A to reduce unwanted bone formation outside the normal skeleton, marking a strategic use of monoclonal antibodies in managing rare conditions. This approval provides another therapeutic avenue for FOP patients and highlights the critical role monoclonal antibodies play in addressing complex medical challenges. On the technological front, Vitestro's Aletta has become the first FDA-approved robotic blood draw device. This innovation addresses the current shortage of phlebotomists and promises enhanced efficiency in healthcare settings. The integration of robotics into routine medical procedures exemplifies how technological advancements can optimize healthcare delivery, improving patient experiences and operational workflows. In regulatory news from the UK, NICE's endorsement of Eli Lilly's once-weekly insulin Onswik for NHS coverage marks a significant step forward in diabetes management. By facilitating access to innovative treatments with more convenient dosing regimens, this approval aims to enhance patient compliance and outcomes significantly. Business development continues to drive innovation across various therapeutic areas. Eli Lilly's collaboration with Amplitude Therapeutics on an RNA vaccine platform signifies a focused effort to harness cutting-edge technologies against infectious diseases. Similarly, Chai Discovery's partnership with Bristol Myers Squibb leverages AI and machine learning to accelerate antibody discovery, showcasing how AI is reshaping drug discovery paradigms. Funding initiatives further reveal industry trends. Kynexis's successful EUR97 million Series A raise will advance its cognitive impairment schizophrenia drug toward registrational development. Such investments underscore confidence in neuroscience therapeutics and emphasize a growing focus on addressing cognitive disorders through novel small molecules. Despite these advancements, challenges persist. Amgen's decision to terminate its collaboration with TScan Therapeutics on a Crohn's disease project due to strategic realignments highlights ongoing industry shifts. Moreover, regulatory recalls impacting product safety continue to underscore the importance of maintaining rigorous standards. In other significant developments, Novo Nordisk is exploring smaller doses of its weight management drug Wegovy through a Phase 3 trial. This study reflects an industry trend towards optimizing drug formulations for enhanced efficacy and patient compliance. On the legal front, Aurinia Pharmaceuticals reached a settlement with Teva Pharmaceuticals to delay a generic version of its lupus drug Lupkynis until late 2036. This agreement secures Aurinia's market position while providing a buffer period to maximize revenue from its patented formulation. Amid geopolitical tensions, Chinese biotech companies remain confident in navigating international markets, reflecting resilience and underscoring China's growing influence in global biotech innovation. The field also observes notable movements such as Boehringer Ingelheim's improved standing in rare disease reputation rankings and B. Braun Medical's IV solution recall due to contamination concerns—highlighting ongoing challenges in product safety and quality assurance. As we wrap up, these discussions showcase an industry rapidly evolving through scientific breakthroughs, strategic collaborations, and regulatory successes. The implications are profound: from more effective therapies for rare conditions to leveraging technological advances for streamlined R&D processes. The pharmaceutical and biotech sectors are on a promising trajectory that could redefine patient care and drug development paradigms globally. Thank you for tuning into Pharma Daily; stay informed about the latest developments shaping our industry.Support the show

Dr. Sid Kerkar, CEO and Founder of AllerGene AI Therapeutics, is developing in vivo gene therapy for severe food allergies, where symptoms are currently treated rather than root causes. AllerGene AI is targeting mast cells, which are immune cells responsible for triggering allergic reactions, and their approach represents a significant opportunity to prevent these reactions. Unlike traditional cell therapy, this technology modifies cells within the body to recognize and eliminate armed mast cells, allowing the body to repopulate with non-reactive cells, preventing severe allergic reactions and establishing long-lasting immune tolerance. Sid explains, "Today, millions of people suffer from food allergies in the US and worldwide. And that's a spectrum from just the mild symptoms that cause discomfort to the really severe kind, which can lead to life-threatening issues. And for us as a company, that's where we focus and where the biggest need is to come up with new treatments that can help prevent these life-threatening events from happening."   "A lot of it is symptom management, and EpiPens are a staple. Everyone who has had a severe reaction or may have one has to carry one around that can help prevent you from having the cardiac and lung issues that cause death. But there's no real treatment that can prevent someone from having these severe reactions. And this is where we're focused: treating the root cause of these severe reactions in the hope that we can develop a treatment that can prevent these terrible reactions from happening." "The triggers can be wide-ranging, from many different food products such as peanuts or seafood or milk. It could be anything really that can sensitize one to have a reaction. And even beyond food, all other types of allergens like bee stings can cause a reaction. We know the cell type that is central to these reactions, and it's a cell type called a mast cell."  #AllerGeneAI #MastCells #AllergicReaction #SevereAllergy #FoodAllergy #AllergyGeneTherapy #GeneTherapy allergene.ai Download the transcript here

Dr. Sid Kerkar, CEO and Founder of AllerGene AI Therapeutics, is developing in vivo gene therapy for severe food allergies, where symptoms are currently treated rather than root causes. AllerGene AI is targeting mast cells, which are immune cells responsible for triggering allergic reactions, and their approach represents a significant opportunity to prevent these reactions. Unlike traditional cell therapy, this technology modifies cells within the body to recognize and eliminate armed mast cells, allowing the body to repopulate with non-reactive cells, preventing severe allergic reactions and establishing long-lasting immune tolerance. Sid explains, "Today, millions of people suffer from food allergies in the US and worldwide. And that's a spectrum from just the mild symptoms that cause discomfort to the really severe kind, which can lead to life-threatening issues. And for us as a company, that's where we focus and where the biggest need is to come up with new treatments that can help prevent these life-threatening events from happening."   "A lot of it is symptom management, and EpiPens are a staple. Everyone who has had a severe reaction or may have one has to carry one around that can help prevent you from having the cardiac and lung issues that cause death. But there's no real treatment that can prevent someone from having these severe reactions. And this is where we're focused: treating the root cause of these severe reactions in the hope that we can develop a treatment that can prevent these terrible reactions from happening." "The triggers can be wide-ranging, from many different food products such as peanuts or seafood or milk. It could be anything really that can sensitize one to have a reaction. And even beyond food, all other types of allergens like bee stings can cause a reaction. We know the cell type that is central to these reactions, and it's a cell type called a mast cell."  #AllerGeneAI #MastCells #AllergicReaction #SevereAllergy #FoodAllergy #AllergyGeneTherapy #GeneTherapy allergene.ai Listen to the podcast here

Joining Kiri Pritchard-McLean this week is comedian Stephen Bailey who explains how fighting transformed his confidence. Professor Ed Wild shares a groundbreaking gene therapy that can treat Huntington's disease for the first time, and Professor Alessandra Devoto explores how plants like basil could help unlock future treatments for liver fibrosis.Best Medicine is your weekly dose of laughter, hope and incredible medicine. Award-winning comedian Kiri Pritchard-McLean is joined by a funny and fascinating panel of comedians, doctors, scientists, experts and historians to celebrate medicine's inspiring past, present and future.Each week Kiri challenges a panel of experts and a comedian to make a case for what they think is 'the best medicine', and each guest champions anything from world-changing science or an obscure invention to an everyday treatment, an uplifting worldview, an unsung hero or a futuristic cure.Whether it's groundbreaking surgery, seaweed underwear, AI glasses to help people with dementia, horse therapy, sports, revolutionary gene therapy for Huntington's disease or yesterday's rubbish becoming tomorrow's medicines - it's always something worth celebrating.Hosted by Kiri Pritchard-McLeanFeaturing: Stephen Bailey, Professor Alessandra Devoto and Professor Ed Wild Written by  Edward Easton, Jordan Gray, Frances Keyton, Kiri Pritchard-McLean, Ben Rowse and Rhys ThorneProducer: Tashi RadhaExecutive Producer: Ben WorsfieldTheme tune composed by Andrew JonesA Large Time production for BBC Radio 4

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 of the latest breakthroughs, regulatory updates, and clinical trial results shaping our industry. Recently, a significant milestone has been achieved in gene therapy. A new study showcases a groundbreaking approach to treating rare genetic disorders using CRISPR-Cas9 technology. This innovative method involves directly editing genes within the body, offering a potential cure rather than simply managing symptoms. The trial, conducted on individuals with a rare liver disease, demonstrated not only safety but also preliminary efficacy, marking a pivotal moment in gene therapy. The implications are vast; by honing in on genetic abnormalities in situ, this approach could revolutionize treatment paradigms for a host of monogenic diseases, potentially extending to more complex conditions in the future. In regulatory news, there's an update from the European Medicines Agency regarding the approval of a novel cancer immunotherapy. This new treatment leverages the patient's immune system to combat tumors more effectively. It is based on a combination of checkpoint inhibitors and personalized vaccines tailored to an individual's tumor profile. The approval was granted following compelling phase 3 trial results showing significant improvement in overall survival rates for patients with advanced melanoma. This development underscores the growing importance of personalized medicine as a cornerstone of modern oncology treatment strategies. The conversation around Alzheimer's disease has taken an intriguing turn with recent findings from a phase 2 clinical trial. Researchers have been investigating an experimental drug that targets amyloid plaques in the brain, long considered a hallmark of Alzheimer's pathology. The trial results revealed that participants receiving the drug showed a marked slowing in cognitive decline compared to the placebo group. While these findings are promising and add to the body of evidence supporting amyloid-targeting therapies, experts caution that larger trials are necessary to confirm these effects and assess long-term outcomes. Meanwhile, an exciting development in vaccine technology is underway as researchers explore mRNA platforms beyond COVID-19 applications. A new mRNA vaccine targeting respiratory syncytial virus (RSV) has shown strong immunogenicity in early-stage trials. Given RSV's significant health burden on infants and older adults globally, this advancement could fill a critical gap in infectious disease prevention. The adaptability of mRNA technology offers hope for rapid development of vaccines against other elusive viruses as well. In another key development, artificial intelligence continues to make strides within drug discovery processes. A biotech company has successfully utilized AI algorithms to predict molecular structures that could inhibit enzymes linked to various cancers. This approach significantly accelerates the initial discovery phase, potentially reducing years off traditional timelines. By integrating AI into early-stage research, there's potential not just for faster drug discovery but also for identifying novel therapeutic targets that might have been overlooked by conventional methods. Additionally, there is news from regulatory fronts with the FDA's recent fast-track designation for an investigational treatment for non-alcoholic steatohepatitis (NASH). This chronic liver disease affects millions worldwide and currently lacks approved pharmacological treatments. The investigational drug works by modulating specific metabolic pathways involved in liver fat accumulation and inflammation, addressing key aspects of NASH pathophysiology. Fast-track designation is significant as it facilitates expedited review processes, reflecting both strong preliminary data and an urgent unmet medical need. Finally, let's touch upon an evolving trend within our industry—sustainability in pharmaceutical manufacturing. Several companies are now adopting green chemistry principles to reduce environmental impact during production processes. This shift not only benefits ecological health but also aligns with broader corporate social responsibility goals increasingly demanded by stakeholders. By minimizing waste and using renewable resources where possible, these practices aim to create more sustainable models for drug production without compromising efficacy or safety. That's all for today's episode of Pharma Daily. Thank you for tuning in to stay informed about these pivotal developments reshaping our industry landscape. Stay curious and connected as we continue to explore how science and innovation intersect to improve global health outcomes. Until next time, stay informed and stay inspired!Support the show

Kendra Riley's two daughters were diagnosed with metachromatic leukodystrophy (MLD), a rare terminal genetic disorder. With no FDA-approved treatment available in the U.S., Kendra's family had to raise nearly half a million dollars and relocate to Italy so their youngest daughter could receive a life-saving gene therapy. While that treatment succeeded, their older daughter's disease had progressed too far — she is now in hospice care at age eight. Kendra joins Finding Freedom to advocate for Right to Try 2.0, federal legislation that would give rare disease patients access to individualized gene-based therapies at home without crossing an ocean. She also shares advice for families navigating rare disease diagnoses and resources that can help along the way. Video Chapters: 0:00 — Intro & Guest Introduction 3:19 — The MLD Diagnosis 4:55 — Moving to Italy for Gene Therapy 6:43 — Right to Try 2.0 Explained 8:07 — Two Daughters, Two Outcomes 13:53 — Advice for Rare Disease Families 17:32 — How to Help & Where to Follow Links: Kendra's family blog: rileysroad.com Instagram: @MrsKendraRiley Right to Try 2.0 — introduced by Sen. Ron Johnson (WI) and Rep. Diana Harshbarger (TN) Goldwater Institute — lead organization behind Right to Try 2.0 MLD Foundation — resources for metachromatic leukodystrophy Armer Foundation for Kids — financial assistance for families of children with life-threatening illnesses Contact your representatives: House.gov | Senate.govBecome a supporter of this podcast: https://www.spreaker.com/podcast/heartland-newsfeed-radio-network--2904397/support.

Rejuvenate Bio CEO Dan Oliver joins Chris Lustrino to discuss the company's gene therapy platform for aging-related disease. A spinout from George Church's lab at Harvard Medical School, Rejuvenate Bio is developing therapies designed to change gene expression profiles associated with aging rather than edit or replace a patient's underlying genome. Dan explains how the company uses viral delivery to help the body produce beneficial proteins over time. The conversation explores Rejuvenate Bio's dual strategy across animal health and human health. Dan explains why dogs offer both a meaningful commercial market and a scientifically useful path for developing aging-related therapies, especially because dogs age faster than humans and owners increasingly view pets as family members. Chris and Dan also discuss Rejuvenate Bio's partnerships with animal health companies, its development and commercial milestone agreements, its recently closed round backed by Merck Animal Health, the FDA approval process for veterinary products, and the company's long-term plan to generate first-in-human clinical data that could create major partnering, acquisition, or public market opportunities.

Stay informed on current events, visit www.NaturalNews.com  - AI-Generated Viruses and Their Implications (0:10) - The Nature of Viruses and AI-Generated Proteins (3:57) - The Role of AI in Protein Synthesis and Its Risks (8:44) - The Depopulation Agenda and Medical Establishment (12:32) - The Impact of AI on Society and Personal Freedom (59:41) - The Role of AI in Enhancing Human Capabilities (59:53) - The Future of AI and Its Implications for Society (69:00) - The Role of AI in Addressing Global Challenges (69:15) - The Impact of AI on the Economy and Workforce (69:32) - The Ethical Considerations of AI Development (69:44) - Pandora's Box and Technological Advancements (76:48) - AI and Vaccine Dangers (78:55) - AI and Gene Therapy (82:02) - Global Implications and Historical Context (84:57) - Nuclear War and DNA Damage (85:08) - Survival and Human Resilience (95:24) - Convergence of Technologies and Future Predictions (98:38) - Support and Resources (100:36) Watch more independent videos at http://www.brighteon.com/channel/hrreport  ▶️ Support our mission by shopping at the Health Ranger Store - https://www.healthrangerstore.com ▶️ Check out exclusive deals and special offers at https://rangerdeals.com ▶️ Sign up for our newsletter to stay informed: https://www.naturalnews.com/Readerregistration.html Watch more exclusive videos here:

Why is gene therapy still so difficult to manufacture at scale after decades of progress?Phillip Ramsey, Chief Technical Officer at Sangamo Therapeutics, joins BioTalk Unzipped for a candid conversation about one of the central challenges facing advanced therapies: turning extraordinary science into reproducible, scalable medicines.Thank You to Our SponsorsLeucentra - https://leucentra.com/Inspired by Science. Empowered by IT. Leucentra provides specialized IT consulting and services for life sciences and healthcare, helping organizations use technology more effectively to support innovation.TruTechnologies - https://trutechnologies.ai/Clinical Trial Execution, Live. TruTechnologies provides live clinical trial execution technology that gives sponsors greater visibility into protocol activities, samples, and study data as they happen.About This EpisodeRecorded onsite at the AAPS National Biotechnology Conference in Boston, Phillip explains why gene therapy manufacturing has not yet reached the maturity of monoclonal antibody production.As technologies have evolved across retroviral vectors, lentiviral vectors, adenovirus, AAV, lipid nanoparticles, and other advanced modalities, development teams have repeatedly been required to rethink fundamental manufacturing and analytical questions.Phillip discusses why analytics are critical, why experience across multiple manufacturing lots and products matters, and why there simply is no shortcut to developing the scientific knowledge necessary to manufacture advanced therapies reliably.He also reflects on the uncertainty of biotechnology.Early in his career, Phillip was walking down a hallway when his CFO asked if he had cashed his paycheck.When he said no, she told him not to.It's a remarkable story that captures something rarely discussed about biotechnology: behind transformative science are people willing to operate through considerable uncertainty while trying to build something that has never existed before.Gregory and Phillip Discuss• Viral vector manufacturing and scale-up• Why analytics are essential to gene therapy development• AAV manufacturing challenges• Lentiviral vectors and other evolving delivery technologies• Lipid nanoparticles and changes in advanced therapy manufacturing• Why gene therapy cannot simply adopt the established monoclonal antibody manufacturing playbook• Building process knowledge across lots and products• Career resilience in biotechnology• Progress across AAV, CAR-T, and other advanced therapies• Gene therapy affordability• The importance of therapeutic durability• Outcomes-based payment models for advanced therapiesPhillip's message for scientists and industry leaders is straightforward: don't assume the manufacturing problem has already been solved simply because a technology has existed for decades.The field is still evolving, and some knowledge cannot be shortcut.About Phillip RamseyPhillip Ramsey is Chief Technical Officer at Sangamo Therapeutics and brings more than 35 years of experience across biotechnology and pharmaceutical manufacturing, technical development, operations, quality, and advanced therapies.Connect with Phillip Ramsey:https://www.linkedin.com/in/phillip-ramsey-a2074711/Sangamo Therapeutics:https://www.sangamo.com/Connect With BioTalk UnzippedGregory Austin on LinkedIn:https://www.linkedin.com/in/gregoryaustin1/Dr. Chad Briscoe on LinkedIn:https://www.linkedin.com/in/chadbriscoe/BioTalk Unzipped:https://www.biotalkunzipped.com/BioTalk Unzipped unzips, unlocks, and uncovers the stories behind medical progress through conversations with scientists, executives, entrepreneurs, and innovators advancing biotechnology, pharmaceuticals, and medical technology.Recorded onsite at the AAPS National Biotechnology Conference in Boston.Special thanks to AAPS and Rebecca Stauffer for supporting the BioTalk Unzipped interview series.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Recent weeks have been a whirlwind of activity in the pharmaceutical and biotech sectors, showcasing significant strides in scientific breakthroughs, regulatory advancements, and strategic industry maneuvers. Replimune's Vusolimogene Oderparepvec has emerged as a beacon of hope in the fight against advanced melanoma. This gene therapy, utilizing an oncolytic virus, has recently received a positive nod from an advisory committee for its Phase 1/2 trial. When combined with PD-1 checkpoint inhibitors, it holds promise for enhancing immune responses against tumors—a critical development in cancer treatment that may redefine therapeutic strategies. This advancement follows two prior FDA rejections, underscoring the multifaceted evaluation process drugs undergo before approval and demonstrating that perseverance can eventually lead to success. Meanwhile, AstraZeneca and Daiichi Sankyo have achieved a milestone with the European Union's approval of Datopotamab Deruxtecan for treating unresectable or metastatic triple-negative breast cancer (TNBC). As an antibody-drug conjugate targeting the Trop-2 protein, it offers new hope to TNBC patients who often have limited options. This approval underscores the potential of antibody-drug conjugates in oncology, supported by promising Phase 3 trial data. Regeneron Pharmaceuticals is enjoying notable revenue growth thanks to Dupixent, a monoclonal antibody addressing autoimmune and respiratory diseases. Such financial success underscores the expanding role of biologics in tackling chronic illnesses, reflecting a broader industry trend towards biologically-based treatments. In business news, Pharmanovia's licensing deal with Impact Therapeutics for Senaparib signifies strategic expansion in oncology therapeutics. Similarly, Kaigene's agreement with Taisho Pharmaceutical highlights ongoing interest in autoimmune therapies, illustrating how companies are leveraging partnerships to broaden their therapeutic horizons. Pfizer is on the brink of expanding its label for Ritlecitinib (Litfulo), a JAK3 inhibitor that showed positive results in Phase 3 trials for nonsegmental vitiligo. By targeting autoimmune pathways, this small molecule could offer new hope to vitiligo patients seeking effective treatments. Litfulo is advancing through regulatory stages following successful trials, poised to provide an oral alternative for non-segmental vitiligo—a potential game-changer that could improve patient compliance and expand treatment options beyond Incyte's Opzelura. However, not all news is positive. Novo Nordisk faced disappointment when its Ziltivekimab failed a Phase 3 trial for atherosclerotic cardiovascular disease with chronic kidney disease. Despite this setback, Novo Nordisk remains optimistic about ongoing trials Artemis and Hermes as potential lifelines for their CKD program. Success could diversify their portfolio and introduce novel therapeutic options for CKD patients. Additionally, financial hurdles surfaced as Daiichi Sankyo saw an 11% share drop due to an accounting error, and Alnylam Pharmaceuticals' market value took a hit following a lowered revenue forecast for its TTR drug. On a global scale, the Trump administration has allocated $600 million to GAVI for childhood immunizations worldwide. This funding reversal emphasizes ongoing efforts to ensure vaccine accessibility across the globe—an essential component of public health initiatives. The dynamic nature of these developments reflects both the potential and challenges within the pharmaceutical landscape. The promising avenues presented by gene therapy, antibody-drug conjugates, and JAK inhibitors are tempered by clinical setbacks and financial volatility, underscoring the rigorous demands faced by industry stakeholders. In other industry news, AbbVie's Skyrizi continues to thrive despite increasing competition in the psoriasis market. With sales reaching $5.55 billion in Q2 2026, Skyrizi demonstrates the sustained demand for innovative biologics that enhance treatment efficacy and safety profiles. Karyopharm Therapeutics remains committed to its XPO1 inhibitor Xpovio despite setbacks in other indications like endometrial cancer. Their focus on myelofibrosis illustrates a strategic pivot towards hematologic malignancies where unmet needs persist. Oral GLP-1 receptor agonists are gaining ground as Novo Nordisk's Wegovy pill and Eli Lilly's Foundayo vie for dominance in weight management solutions. These oral formulations promise increased accessibility over traditional injectables. Regulatory arenas continue to evolve with AbbVie's Rinvoq gaining European clearance for alopecia and vitiligo. Such approvals highlight regulatory bodies' critical role in expanding market access across diverse therapeutic areas. Strategic partnerships also abound as WellSpan Health collaborates with Hippocratic AI to integrate artificial intelligence into healthcare solutions—a growing trend aimed at enhancing clinical decision-making capabilities. Meanwhile, Apnimed's $192 million IPO success indicates strong investor confidence in novel therapeutics like its sleep apnea treatment—a reflection of biopharma's financing dynamics where targeted therapies draw significant interest. Overall, these updates paint a vivid picture of an industry characterized by relentless innovation through scientific research, strategic alliances, and regulatory progress—all aimed at improving patient outcomes amidst evolving market demands. The industry's focus on breakthrough technologies continues to shape global health initiatives by offering new opportunities for patient care across various conditions. As companies navigate this dynamic environment marked by both promise and challenge—their ability to innovate scientifically while engaging strategically with regulators will be crucial to bringing novel therapies effectively into clinical practice where they can make meaningful impacts on patients' lives worldwide.Support the show

Dr. Panicos Shangaris is from King's College London, where he is a senior clinical lecturer and consultant in maternal and fetal medicine.  He discusses the treatment options for sickle cell disease, an inherited blood condition, and his work on the Pericles project, which aims to treat it before birth. In-utero gene therapy offers a third option for parents of an affected fetus alongside postnatal treatment or pregnancy termination. Panicos emphasizes key advantages of fetal therapy within the first 16 weeks of gestation, the need to address ethical concerns, and ensuring the safety of both the mother and fetus during the procedure.  Panicos explains, "So sickle cell disease is an inherited condition that affects the blood and the molecule in the red cell, which is basically the red blood cell that carries oxygen. So what happens is the red cell has a biconcave shape, and for people who have sickle cell disease, that biconcave shape is lost, and the erythrocyte red cells are sickle-shaped. So because of that, the sickle-shaped red cells have difficulty going through small capillaries and small vessels. They can cause strokes, they can cause pain because they block the blood flow to the small capillaries." "Yes, so the cause is because of an abnormal gene, which basically makes the hemoglobin, the molecule that carries oxygen around the different organs and around the body, instead of there being a problem in the genetic code, if I can put it like that. And that's why the erythrocyte, the red cells, have an abnormal shape. Very well-known what's causing it: a genetic problem." "So Pericles stands for prenatal therapy for Sickle Cell Disease. And what we are trying to achieve is to offer a third option to people who might carry a fetus affected by sickle cell disease. At the moment, the two options are stopping the pregnancy, termination of pregnancy, or having an affected fetus and an unaffected baby and sick postnatal care. So our aim is to offer a third option, which is correcting the disease inside the womb so that the parents have a healthy baby. So correct the gene while the baby is inside the womb. And this is the main aim of the project." #KingsCollegeLondon #KCLResearch #GeneTherapy #PrenatalCare #Hematology #Genomics #EmpoweredPatient #PERICLES #SickleCellDisease #SickleCellAwareness #PreconceptionCare #BeforePregnancy #PregnancyPlanning #CarrierScreening #GeneticCounselling #ReproductiveChoices #PrenatalDiagnosis #PrenatalTherapy #InUteroTherapy #GeneEditing #StemCellTherapy #FetalMedicine #MaternalFetalMedicine #HighRiskPregnancy #IVFJourney #PGTM #PatientVoice #PatientEmpowerment #SharedDecisionMaking #HealthEquity #PrecisionMedicine #PersonalisedMedicine #FutureOfMedicine  kcl.ac.uk Download the transcript here 

Dr. Panicos Shangaris is from King's College London, where he is a senior clinical lecturer and consultant in maternal and fetal medicine.  He discusses the treatment options for sickle cell disease, an inherited blood condition, and his work on the Pericles project, which aims to treat it before birth. In-utero gene therapy offers a third option for parents of an affected fetus alongside postnatal treatment or pregnancy termination. Panicos emphasizes key advantages of fetal therapy within the first 16 weeks of gestation, the need to address ethical concerns, and ensuring the safety of both the mother and fetus during the procedure.  Panicos explains, "So sickle cell disease is an inherited condition that affects the blood and the molecule in the red cell, which is basically the red blood cell that carries oxygen. So what happens is the red cell has a biconcave shape, and for people who have sickle cell disease, that biconcave shape is lost, and the erythrocyte red cells are sickle-shaped. So because of that, the sickle-shaped red cells have difficulty going through small capillaries and small vessels. They can cause strokes, they can cause pain because they block the blood flow to the small capillaries." "Yes, so the cause is because of an abnormal gene, which basically makes the hemoglobin, the molecule that carries oxygen around the different organs and around the body, instead of there being a problem in the genetic code, if I can put it like that. And that's why the erythrocyte, the red cells, have an abnormal shape. Very well-known what's causing it: a genetic problem." "So Pericles stands for prenatal therapy for Sickle Cell Disease. And what we are trying to achieve is to offer a third option to people who might carry a fetus affected by sickle cell disease. At the moment, the two options are stopping the pregnancy, termination of pregnancy, or having an affected fetus and an unaffected baby and sick postnatal care. So our aim is to offer a third option, which is correcting the disease inside the womb so that the parents have a healthy baby. So correct the gene while the baby is inside the womb. And this is the main aim of the project." #KingsCollegeLondon #KCLResearch #GeneTherapy #PrenatalCare #Hematology #Genomics #EmpoweredPatient #PERICLES #SickleCellDisease #SickleCellAwareness #PreconceptionCare #BeforePregnancy #PregnancyPlanning #CarrierScreening #GeneticCounselling #ReproductiveChoices #PrenatalDiagnosis #PrenatalTherapy #InUteroTherapy #GeneEditing #StemCellTherapy #FetalMedicine #MaternalFetalMedicine #HighRiskPregnancy #IVFJourney #PGTM #PatientVoice #PatientEmpowerment #SharedDecisionMaking #HealthEquity #PrecisionMedicine #PersonalisedMedicine #FutureOfMedicine  kcl.ac.uk Listen to the podcast here 

Podcast Host and Interviewee: Shahad Abdulsahib, PhD Graduate, UT Health San Antonio Podcast Description: Shahad Abdulsahib discusses a landmark preclinical study evaluating Synthetic Super-Enhancer-based gene therapy for glioblastoma, published in Nature in April 2026, and the rapid translation of these findings into the first-in-human Phase 1/2 ADePT clinical trial with first patient dosing in May 2026.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're navigating through a myriad of transformative advancements reshaping the industry. From artificial intelligence-driven research breakthroughs to strategic acquisitions in emerging therapeutic areas, these developments are setting new benchmarks in drug discovery and patient care. **Strategic Acquisitions and Expansions:** Samsung Biologics' recent proposal to acquire Swiss CDMO Polypeptide for $1.8 billion marks a significant expansion into peptide production, underscoring the growing therapeutic importance of peptides. Their specificity and efficacy make them particularly appealing for metabolic disorders such as obesity. This acquisition reflects a strategic move to capture emerging market opportunities as demand for innovative metabolic treatments rises. Eli Lilly's bold $2.8 billion acquisition of Atai-Beckley highlights the pharmaceutical industry's shifting focus towards psychedelics. As mental health disorders like depression and PTSD become more prominent, psychedelics hold great promise for new therapeutic approaches. This acquisition may pave the way for further research and acceptance of psychedelics within mainstream medicine, offering new hope for patients. Another notable development is Tempus' acquisition of Personalisis for $1.5 billion. This move strengthens Tempus' cancer treatment portfolio by leveraging genomics-driven insights, emphasizing precision medicine's critical role in oncology. **AI Integration and Technological Innovation:** Bristol Myers Squibb is doubling down on AI integration by expanding its collaboration with Nvidia to build what promises to be the most powerful AI supercomputer dedicated to life sciences. This development is pivotal as it signifies a deeper commitment to harnessing computational power in R&D. By accelerating drug discovery and optimizing clinical trial processes, AI stands to revolutionize how treatments are developed and tailored to individual patients. Overall, these developments indicate a dynamic phase characterized by technological innovation and strategic collaborations. The integration of AI into drug development processes stands out as a transformative force, promising enhanced efficiency and efficacy in bringing new treatments to market. **Regulatory Milestones and Clinical Trials:** In regulatory news, Takeda's Qdenga has achieved a milestone by becoming India's first approved dengue vaccine. This approval is crucial in addressing neglected tropical diseases, with significant implications for public health in regions where dengue is prevalent. By reducing dengue fever incidence, this vaccine could play a vital role in improving health outcomes in many tropical countries. Promising results have emerged from clinical trials across various therapeutic areas. 4D Molecular Therapeutics reported an 87% reduction in treatment burden for wet AMD using their gene therapy candidate 4D-150 in Phase 2b trials—demonstrating potential as a transformative approach with reduced intervention frequency. Latigo Biotherapeutics' Nav1.8 inhibitor LTG-001 outperformed Vicodin in Phase 2 trials for acute pain, offering a non-opioid alternative amidst the opioid crisis. Meanwhile, Regenxbio's five-year data on surabgene lomparvovec shows sustained efficacy in wet AMD and diabetic retinopathy—underscoring its potential as a long-term treatment option. **Financial Performance and IPO Activity:** Financially, Abbott exceeded expectations with strong sales from their device and diagnostic divisions, prompting an upward revision of their full-year profit forecast. This reflects sustained demand for innovative diagnostic solutions and medical devices—highlighting Abbott's strategic market positioning. The IPO landscape remains active with companies like Nuvox Therapeutics seeking to raise funds for advancing oxygen therapeutics targeting hypoxia-related diseases—showcasing renewed investor interest and confidence within biotech innovation. Latigo Biotherapeutics is preparing for an IPO to advance its pipeline, demonstrating a strategic response to the opioid crisis. By developing alternatives that minimize addiction risks while providing effective relief, Latigo underscores the industry's commitment to safer pain management solutions. Turning to clinical trials, Scribe Therapeutics plans a $96 million IPO to advance its CRISPR-based lipid-lowering therapies, marking significant interest in genetic solutions for cardiovascular diseases like hyperlipidemia. The precision offered by CRISPR technology could revolutionize treatment options for conditions contributing significantly to cardiovascular health burdens. **Geopolitical Influences:** Geopolitical factors are also influencing market dynamics, particularly in the UK biotech sector where IPO plans are being shaped by global uncertainties despite robust venture capital backing. This situation underscores the complex interplay between regional economic conditions and global investment trends in biotech innovation. As these trends continue unfolding across the industry landscape—from AI-driven research advancements to strategic acquisitions—the potential impact on patient care paradigms remains profound. These developments not only promise new possibilities within drug development but also influence broader healthcare delivery models aimed at improving patient outcomes globally.Support the show

The first person has been treated with a highly anticipated new gene therapy that aims to turn back the clock on ageing cells. The trial is aimed at retinal cells, with the hope that encouraging them to behave as if they were young again could improve sight in the affected patients. If it proves to be safe, it could open the door to a whole raft of therapies based on the emerging field of cellular rejuvenation. To understand more about this cutting edge research, Madeleine Finlay hears from science editor Ian Sample and from Paul Knoepfler, professor of cell biology and human anatomy at the university of California, Davis.. Help support our independent journalism at theguardian.com/sciencepod

Gene therapy has changed what is possible for people living with sickle cell disease. New treatments have shown that the condition can be successfully treated. But the therapy is costly and available to only a small number of patients. Millions of people around the world still rely on early diagnosis and preventative medicines, with access to basic care remaining a challenge in many places, including sub-Saharan Africa, which has almost 80% of global sickle cell cases. Are we closer to gene therapy for sickle cell disease being accessible?We speak to Dr Natasha Archer, director of the Sickle Cell Program at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, US; Dr Alexis Thompson, paediatric haematologist at the Children's Hospital of Philadelphia and a professor of paediatrics at the University of Pennsylvania, US; Dr Leon Tshilolo, professor of paediatrics and haematology at the University of Mbuji-Mayi, DR Congo and Prof Baba Inusa, consultant paediatrician in the School of Life Course & Population Sciences at King's College London, UK(Photo: Cell lines are prepared in a laboratory in Maryland, US. Credit: Brendan Smialowski/Getty Images)Presenter: Daniel Rosney Producer: Matt Toulson Researcher: Evie Yabsley Sound engineer: Cameron Ward Production co-ordinator: Phoebe Lomas and Liam Morrey Editor: Tom Bigwood

The case of baby KJ Muldoon, an infant born with a lethal genetic metabolic disorder, demonstrates the potential to compress years of therapeutic development into months using an in vivo base editing approach. Jeff Coller, director of the Johns Hopkins RNA Innovation Center, wrote about the case in a New York Times op-ed, arguing that CRISPR-based base editors—delivered via lipid nanoparticles as mRNA molecular surgery payloads—could be generalized to thousands of monogenic disorders. He further explains that realizing this promise will be constrained less by scientific limitations than by regulatory, manufacturing, and reimbursement challenges. Coller also outlines what it will take to make bespoke therapies economically and operationally viable, and argues that failure to scale this approach would reflect not a failure of science, but of imagination.

AI and Generative Biology: Authoring Life, Redesigning Healthcare, and Building Guardrails: Physician and molecular biologist Dr. Adrian Woolfson, co-founder of Genyro, a biotechnology company specializing in synthetic genome design and construction, and author of “On the Future of Species: Authoring Life by Means of Artificial Biological Intelligence,” discusses the convergence of AI with synthetic/generative biology that could make biology programmable and enable genome design and construction beyond traditional gene editing. Woolfson argues that the technology is morally neutral but is arriving rapidly and requires public awareness, governance, and guardrails due to risks, including misuse by authoritarian regimes and biological warfare. He discusses evolution's trade-offs and why he favors limiting human applications to curing disease and extending healthy longevity, while opposing germline modification; he explains germline vs somatic editing and cites the flawed, unethical case of a Chinese scientist attempting inherited HIV resistance. They examine sickle cell disease cures costing $2–$3 million, scalability issues, and trade-offs like malaria protection, and highlight non-medical benefits such as engineered crops, biomaterials, desalination, and DNA-based information storage.

Guys, check out b-Vibe's 360 Plug — the male equivalent of the rabbit. It's a remote-controlled vibrator with 360° rotation, a smooth silicone body, and simultaneous prostate and perianal pleasure. Grab yours at bvibe.com and use code TABOO25 for 25% off!Erectile dysfunction affects tens of millions of men in the U.S., yet stays wrapped in silence and shame. In this episode, I bust the biggest ED myth — that it's a permanent, unfixable part of aging — and walk through the full range of treatment options available today. I cover penile implants, vacuum erection devices, testosterone replacement therapy, and FDA-approved medications like Viagra and Cialis, then move into natural approaches: therapy, lifestyle changes, key nutrients, and sex toys that keep intimacy alive during treatment. I close with a look at emerging treatments on the horizon — shockwave therapy, stem cells, PRP, and gene therapy — and a clear call to action: talk to your doctor, because ED is treatable and no one has to navigate it alone.In This Episode:00:00 — bvibe 360 Plug (code TABOO25)00:50 — Podcast Intro / Welcome to Taboo to Truth01:10 — Welcome Back + ED Stats01:55 — What This Episode Will Cover02:15 — Myth-Busting: ED Is NOT a Permanent Part of Aging03:00 — Treatment #1: Penile Implants04:05 — Treatment #2: Vacuum Erection Devices (Pumps)05:00 — Treatment #3: Testosterone Replacement Therapy (TRT)05:50 — Treatment #4: FDA-Approved Medications (Viagra, Cialis, Levitra, Stendra)06:35 — Natural Approaches: Counseling & Sex Therapy07:35 — Lifestyle Changes & Key Nutrients (Citrulline, Vitamin D/E, Zinc)08:20 — Sex Toys: Staying Intimate During Treatment09:10 — The Future of ED Treatment (Shockwave, Stem Cells, PRP, Gene Therapy)10:05 — Fastest Way to Fix ED + Key Takeaway11:00 — Outro: Subscribe + Hotter, Wiser, Wilder CourseWant a deeper look? Watch the full episode on YouTube for a more visual experience of today's discussion. This episode is best enjoyed on video—don't miss out!

Dr. William Li: Eat to Beat Disease, Extend Longevity, and Hack Your Vascular Health Your body already has a built-in cancer defense system, and the foods you eat can rival prescription drugs at activating it. In this episode, you'll discover what 720,000 living centenarians reveal about longevity, why microplastics are reaching your brain through your nose, and how your blood vessels hold the real key to biological aging. -Watch this episode on YouTube for the full video experience: https://www.youtube.com/@DaveAspreyBPR Host Dave Asprey sits down with Dr. William Li, an internationally renowned physician, scientist, and two-time New York Times bestselling author of "Eat to Beat Disease" and "Eat to Beat Your Diet." As President and Medical Director of the Angiogenesis Foundation, Dr. Li's groundbreaking research has led to the development of more than 40 new medical treatments impacting care for over 70 diseases including diabetes, heart disease, blindness, and obesity. His TED Talk, "Can We Eat to Starve Cancer?", has surpassed 11 million views, making him one of the most trusted voices at the intersection of functional medicine and food as medicine science. Together they dig into the biology of SuperAgers, the vascular markers that predict how long you'll live, and why flow-mediated dilation may be the most underrated biohacking measurement tool available today. Dave shares what happened after four separate gene therapies, including klotho and VEGF, and Dr. Li explains why your metabolism, mitochondria, and gut microbiome show up in the data on every centenarian ever studied. They also get into the microplastic crisis, the cribriform plate pathway that delivers airborne particles directly to your brain, and why your tongue is the first place your body quietly stores fat. You'll Learn: What centenarian research reveals about immune function, gut health, and vascular resilience Why 50% of food extracts matched or outperformed cancer drugs in head-to-head angiogenesis testing How microplastics travel through your nose directly to your central nervous system What flow-mediated dilation and pulse wave velocity tell you about your true biological age Why your hind-third tongue fat is an overlooked early warning sign for sleep apnea and metabolism problems What Dave's four gene therapies (klotho, VEGF, follistatin) actually did to his body How the vagus nerve connects gut health to brain optimization, stress resilience, and longevity Why genetics now accounts for roughly half of longevity outcomes, and what that means for the future of gene therapy How to use biohacking tools to compound your health the same way you compound financial returns Thank you to our sponsors! - Beyond Wonderland Conference | Oct 13 - 14, 2026. Get your ticket now at wonderlandconference.com. - Amp | If you're ready to make fitness fit into your life, go to amp.ai to check it out - Caldera + Lab | A small habit with big results. Go to CalderaLab.com/DAVE and use code DAVE for 20% off your first order. - Calroy | Go to Calroy.com/DAVE for exclusive discounts on Arterosil HP, Vascanox HP and all Calroy products. Dave Asprey is a four-time New York Times bestselling author, founder of Bulletproof Coffee, and the father of biohacking. With over 1,000 interviews and 1 million monthly listeners, The Human Upgrade brings you the knowledge to take control of your biology, extend your longevity, and optimize every system in your body and mind. Each episode delivers cutting-edge insights inhealth, performance, neuroscience, supplements, nutrition, biohacking, emotional intelligence, and conscious living. New episodes are released every Tuesday, Thursday, Friday, and Sunday (BONUS). Dave asks the questions no one else will and gives you real tools to become stronger, smarter, and more resilient. Keywords: William Li, Dr. William Li, Eat to Beat Disease, angiogenesis, food as medicine, centenarians, SuperAgers, longevity, anti-aging, biohacking, vascular health, flow-mediated dilation, pulse wave velocity, microplastics, cribriform plate, gene therapy, klotho, VEGF, follistatin, mitochondria, metabolism, gut health, vagus nerve, sleep apnea, functional medicine, human performance, brain optimization, cancer prevention, inflammation, immune system Resources: • Learn More About Dr. Li's Work At: https://drwilliamli.com • Get My 2026 Clean Nicotine Roadmap | Enroll for free at https://daveasprey.com/2026-clean-nicotine-roadmap/ • Dave Asprey's Latest News | Go to https://daveasprey.com/ to join Inside Track today. • Danger Coffee: https://dangercoffee.com/discount/dave15 • My Daily Supplements: SuppGrade Labs (15% Off) • Favorite Blue Light Blocking Glasses: TrueDark (15% Off) • Dave Asprey's BEYOND Conference: https://beyondconference.com • Dave Asprey's New Book – Heavily Meditated: https://daveasprey.com/heavily-meditated • Join My Substack (Live Access To Podcast Recordings): https://substack.daveasprey.com/ • Upgrade Labs: https://upgradelabs.com Timestamps: 00:00 – Trailer 02:33 – William's Background 06:21 – Food vs. Cancer Drugs 14:09 – Gene Therapy 23:50 – Centenarian Research 27:41 – Stress & the Vagus Nerve 37:31 – Vascular Health 42:34 – Microplastics 53:19 – Novel Biomarkers See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.