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Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfesor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this 8-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th-7th 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Roberts will discuss immune responses and other safety concerns related to gene therapies.Mark Roberts, MDUndoubtedly, the immune system is a major issue in these patients. It would be fantastic if we could immunotolerize our patients and indeed prevent the rejection of the therapy. We've talked about the fact that these are viral vectors and of course there may be high seroprevalence of antibodies to these viral vectors, and it's very important in the pre-screening of patients who might be eligible to understand that at the beginning. These of course can have developed over the years and of course can be part of immunological memory and therefore extremely difficult and probably impractical to actually shift.On giving the treatment though as I think we're all aware there is this problem of the innate immunity and potential therefore for acute toxicities and then a learned or adaptive response with cytotoxic T cells and antibodies which may of course become high tighter neutralizing antibodies and potentially antibodies not only against the viral vector, even the functional protein, even the transgene are all theoretical possibilities with time. The capsid, the transgene, and even the protein product can all potentially induce an immunological event. Of course, all of these would lead to both potential patient changes and then a lack of efficacy of the treatment.Indeed, there have been some serious and indeed fatal problems in the gene therapy development program as I think we're all aware. Though many of these are thankfully been overcome. Spinal muscular atrophy has a gene therapy which is licensed, but there were early patients who actually had significant problems. A patient of just 6 months of age who developed kidney failure, two other patients who actually developed liver failure.In Duchenne muscular dystrophy, a very common condition, again there were significant issues and crucially in these patients who all have cardiomyopathy, it was heart failure and cardiac arrest that were big concerns and pulmonary edema and this was seen even with a CRISPR-based technology and is perhaps is best known but has been addressed the excellent myotubular myopathy patients, four patients died and crucially quite a long time after the gene therapy emphasizing the need to monitor these patients extremely carefully and these patients died of cholestatic liver failure albeit that they had a degree of liver dysfunction.That's changed our screening of course of patients, we're now all looking in myotubular patients for liver involvement and Rett syndrome as well. Now these immunoprophylaxis treatment regimes to hopefully try and reduce the immunological reaction against the gene are certainly evolving.This is just a summary of some of the other immunosuppressive regimes used in other disorders, for example, spinal muscular atrophy, but Pompe and MPS as examples of LSDs. Certainly these regimes will continue to evolve and are going to be very important in seeking to make sure that these treatments are effective. It reminds me somewhat of what's happened with enzyme replacement therapy that the use of these immunological strategies in infants has revolutionized the utility of those treatments in early patients.In the next part, Dr. Roberts will discuss lessons learned from gene therapy trials.
Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs.Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lessons learned from gene therapy trials.Mark Roberts, MDWhen we think about the challenges of actually doing clinical trials with these gene therapies, there's a huge development stage in terms of picking the right viral vector with the right surface receptor. That's a major piece of work. That can often take years. The preclinical work is obviously very important as indeed is understanding the natural history because it's really not practical to do placebo-controlled trials of gene therapies.In contrast to other studies, when we turn to phase 1 and phase 2, you'll notice that the patient numbers are often quite small. One is having to think carefully about surrogate measurements of response. Especially when in phase 3 studies, we may be thinking about withdrawing the existing, for example, enzyme replacement therapy because we believe the gene therapy will then be effective.That's just a few snapshots of where we've come and there's a lot more work to be done.In the next part, Dr. Longo will discuss the current treatment landscape and limitations in lysosomal disorders.
Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss ongoing gene therapies in lysosomal disorders.Nicola Longo MD, PhDI'm going to present to discuss some example of ongoing gene therapy for lysosomal disorder. There are gene therapy in development for both Fabry disease and some of this involve ex vivo gene therapy, many others involve systemic administration with an AAV, Gaucher disease type 1 that affect the periphery, and Gaucher disease type 2, where the replacement should occur within the central nervous system because this condition affects the brain. There is already one approved gene therapy for lysosomal disorder, which is for the early onset metachromatic leukodystrophy. This has been approved both in Europe and now even in the United States, which consists of ex vivo gene therapy with the administration of an extra gene that restore the function of the defective enzyme. Now there are many others that are ongoing for the same indication. There are gene therapy programs for GM1 and GM2 gangliosidosis, and at least one for Krabbe disease. It is important to know that some of these condition are actually included in the recommended uniform screening panel. Basically, we would have access to patients in a timely manner for some of these conditions. Then there are several gene therapy under development for the mucopolysaccharidoses, including MPS-IH, MPS-II, MPS-IIIA and MPS-IV.There are different type of lysosomal disorders, the one caused by mutation, integral membrane protein, not enzyme within the lysosome, but protein that are present on the membrane of the lysosome. This gene therapy that have been tested, it is for cystinosis, that it is caused by a defective lysosomal and for Danon disease, which is caused by a deficiency of an integral membrane part. Finally, one lysosomal disorder, which obviously seems a metabolic condition, but it is really not, is glycogen storage disease type 2 or Pompe disease, in which there is the intralysosomal accumulation of glycogen. There are several ongoing clinical trials to try to correct the problem in this condition.Now, I'm going to discuss some of the most advanced program in the lysosomal storage disorder. This include one for Fabry, which is on an accelerated approval pathway with phase 1 and 2 data, one for Gaucher disease type 1. Obviously, I'm going to discuss the one that has been already approved for metachromatic leukodystrophy. There is one for Hunter syndrome, and the difference of the one for Hunter syndrome, it is an example of the direct administration of gene therapy within the central nervous system.Finally, there is one ongoing for glycogen storage disease type 2 or Pompe disease in adult patients. In gene therapy for metachromatic leukodystrophy, it was the first gene therapy approved for lysosomal disorder in human, and this requires harvesting the CD34 cell from affected patient and then introducing the [inaudible 00:04:32] gene back in this cell, and then placing them back inside the patient again. This has been very effective in patients who were treated early, and obviously, the treatment needs to occur before there is irreversible brain damage in this patient.In the next part, Dr. Roberts and Longo will discuss treatment with gene therapies.
Nicola Longo MD, PhD, and Mark Roberts, MDDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Doctors Roberts and Longo will discuss treatment with gene therapies.Question: Can one administer AAV-mediated gene therapy repeatedly?Mark Roberts, MDI think the traditional view would have been no. One can think of gene therapy as a silver bullet. Hopefully, it will reach its target. But if it's not effective, that bullet has been shot, the immunological response has occurred, and it means redosing, at least with that particular vector, may become difficult. But this situation is changing and evolving as we have better understanding of immunological modulation for repeat testing. We were discussing this yesterday evening, weren't we, Professor Longo?Nicola Longo MD, PhDCorrect. Basically, the current AAV-based gene therapy cannot be readministered. It is either effective, or it doesn't work. The other thing is that even though in theory, one could utilize a different AAV vector with different immunogenicity, there is many times cross-reactivity among the different adenovirus, adeno-associated viruses. Now, there are approaches in animal models in which you give a strong immune suppression to prevent the creation of the immune response against the adeno-associated virus, and at least in the animal model, it has been possible to give some of the gene therapy repeatedly.The second approach that is being tested is with gene correction therapy, in which by using an RNA guide and the CRISPR/Cas9 system delivered by lipid nanoparticles, you basically correct some of the effective genetic information. Obviously, since this is done by lipid nanoparticles and not by an AAV, the immunity that you create is really not there. You can give this one repeatedly, and in theory, it can be given more than one time. But again, you are absolutely correct. The current gene therapy cannot be given twice, and either it works or it doesn't work.Question:vWill gene-therapy-treated patients be able to go back to the standard of care or enzyme replacement therapy?Mark Roberts, MDI think when we're talking to patients about the potential benefits of gene therapy and the amelioration of the requirement to have these infusions on a regular basis of ERT, the hope is that will work, but they need to be reassured that we can potentially go back to the ERT. Gene therapy is an important treatment, but we don't know the destination of the patient at the beginning, and we have to make it available to them to go back to ERT.One of the crucial questions, of course, though, is the basis of the immunological reaction that perhaps prevented the gene therapy being effective. If it's against the viral vector, well, okay. If it's against the transgene, not great. If it's against the functional protein, that becomes more difficult. It is somewhat, I think at this time, to be fair to say to patients, think of gene therapy as a trial treatment. It is somewhat a leap of faith and an important observation, of course, for the patient community, but just be aware there may be downsides.Nicola Longo MD, PhDThey totally agree with Dr. Roberts. In general, they should be able to go back to enzyme replacement therapy if the gene therapy is not effective. However, what we are starting to appreciate is that we need to understand the immune response, not just to the enzyme replacement therapy, but also to gene therapy. What this field is doing is forcing geneticists to deal with the immune response. I feel that historically has not been dealt together. The two things need to be integrated. The advantage of the gene therapy is that the protein is produced endogenously. There should be the development of some degree of tolerance with time in the body towards the endogenous continuous production of a protein.Now, will that happen all the time? I still do not know. Again, we need to understand much better what is the integration of the immune system with the response to gene therapy in the ongoing clinical trials.
In this episode, we sit down with Dr. Jean Bennett, Professor Emeritus of Ophthalmology at Penn Medicine and a true pioneer in gene therapy research. Dr. Bennett led the team that developed the first FDA-approved gene therapy to treat inherited blindness, transforming the lives of patients with retinitis pigmentosa and other retinal diseases. Her groundbreaking work spans over three decades and has opened doors for gene therapy applications far beyond ophthalmology. Dr. Bennett shares her remarkable journey from a curious child in New Haven, Connecticut—who chose seeds over candy at age four—to becoming a world-renowned scientist. She opens up about the challenges she faced as a woman in science, including being advised to leave her field entirely when seeking tenure at Penn. With candor and humor, she discusses how mentorship from previous Girl Power Gurus guest, Dr. Virginia Lee, helped her persevere, the emotional eureka moment when blind dogs first regained their sight, and what it felt like to witness blind children see for the first time. Dr. Bennett also offers invaluable advice for young women pursuing STEM careers, emphasizing the importance of finding work you love, building collaborative teams, and never giving up on your dreams despite the obstacles. This is a must-listen for anyone interested in science, medicine, or the power of determination.
In this episode, we discuss promising results of gene therapy, administered suprachoroidally, for the treatment of nonproliferation diabetic retinopathy without macular edema with Charles Wykoff, MD, Ph.D., Retina Consultants of Houston.
The company has also fully enrolled the Phase 2/3 VISTA clinical trial for its XLRP gene therapy.
In the final episode of 2025, the GEN team came together to reflect on some of the stories over the last year that they each found impactful. Some of the stories were covered in GEN's December issue, but our editors took the time to discuss some topics in a bit more depth during the podcast. The discussion began with an update on the progress of AI use in drug discovery. Cell and gene therapy was a big topic with the positive outcome for Baby KJ, while Sarepta dealt with a rollercoaster of a year with their DMD treatment, Elevidys. Research and biotech in Africa are growing and the discussion ranged from challenges to promise in research and development on the continent. This year's NIH budget cuts and impacted institutions rounded out the conversation.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, Phd, Kevin Davies, Phd, John Sterling, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base: GEN Editors Reflect on Six of the Biggest Stories of the YearBy Alex Philippidis, Savannah Wiegel, Fay Lin, PhD, Kevin Davies, PhD, Uduak Thomas, and Julianna LeMieux, PhD, GEN, December 4, 2025StockWatch: Sarepta Says Deaths of Two Elevidys Patients Posted on FDA Database Unrelated to Treatment By Alex Philippidis, GEN Edge, August 10 StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT Stocks By Alex Philippidis, GEN Edge, August 3 FDA Probes Death of Brazilian Boy Linked to Sarepta's Elevidys GEN Edge, July 27 Second DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, June 15 DMD Patient Dies After Treatment with Sarepta Gene Therapy By Alex Philippidis, GEN Edge, March 18From The Editor in ChiefJohn Sterling, December Issue of GEN Gene Therapy in AfricaThe State of CRISPR & Genome Editing Summit, June 11, 2025TOP 50 NIH By Alex Philippidis, GEN, December 4, 2025 FDA Cell & Gene Therapy roundtable: Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions” By Alex Philippidis, and Kevin Davies, PhD, GEN Edge, June 5, 2025Scientists in NYC Rally to Defend and Stand Up for ScienceBy Corinna Singleman, GEN, March 10, 2025Touching Base Podcast Hosted by Corinna Singleman, PhD Behind the Breakthroughs Hosted by Jonathan D. Grinstein, PhD Hosted on Acast. See acast.com/privacy for more information.
Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established, and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lysosomal disorders and the potential for gene therapies.Mark Roberts, MDI'm going to give an overview of what is gene therapy, emphasizing the current challenges and the development issues and needs that there will be as we try and enable gene therapy for our patients, particularly those with lysosomal storage disorders.I'm going to try and make a case for why lysosomal storage disorders are an extremely good group of conditions for the potential benefits of gene modifying therapies. Firstly, whilst we all recognize that these conditions are inherently individually rare, they're certainly severe. Collectively, with over 70 LSD disorders, 1 in 5,000 may be afflicted by these conditions ultimately in their life and can be detected, for example, by newborn screening programs.Secondly, there's certainly a significant clinical burden with these patients with the current standard of care, so a large unmet need exists. Existing enzyme replacement therapies have undoubtedly changed the natural history of many of these conditions, but there are limitations and often initial benefits and later deteriorations.Unfortunately, for most lysosomal storage disorders, it's only symptomatic treatments and indeed, care that is available for these patients with no specific treatment. Thirdly, these conditions are extremely well-characterized, monogenic singleton and problems of inborn errors of metabolism. We know the functional protein that is deficient in these conditions. Because of that, and knowing that these are critical for lysosomal function, and using preclinical models, we can model the potential benefits of gene therapies very well in a number of systems, including, of course, soon, muscle chip experiments as well.Finally, with these conditions, they may potentially be really useful targets whilst not perhaps curing the condition, at least ameliorating the phenotype, and enabling the addition of other treatments as well, potentially. I've noted, some of these therapies can be directly delivered to certain tissues, so muscle tissue, which is my main interest, but also, crucially, the central nervous system, which is very important when we consider ameliorated phenotypes, for example, treated by enzyme replacement therapy, but where the children who become the adults have significant learning disability as a major component to their problems.In the next part, Dr. Roberts will discuss vectors, different strategies, modes of administration, and targets in gene replacement therapies.
Genflow Biosciences Ltd (LSE:GENF, OTCQB:GENFF) CEO Dr Eric Leire talked with Proactive's Stephen Gunnion about the company's progress in its canine gene therapy trial targeting sarcopenia. Leire confirmed that the dosing phase has now been completed with no adverse effects, calling it a key milestone in de-risking the SIRT6 programme for potential pharma partners and investors. He said, “We demonstrated some kind of safety, with the full dosing, we had to observe no side effect, no adverse side effect, even minor event.” Leire explained the study's scientific rigour, highlighting its randomised, controlled and blinded design using aged dogs not previously involved in clinical trials. This, he said, addresses a major unmet need in the life extension field, with industry experts previously considering such a study “impossible to do.” Looking ahead, efficacy data is expected in January, with analysis focused on multiple endpoints. These include mitochondrial function, clinical data, muscle biopsies, and epigenetic markers like the methylation clock in collaboration with UCLA. Leire said the results could influence other Genflow programmes, including preclinical work on NASH, Werner syndrome and ophthalmology. He noted that Genflow is in licensing discussions with several animal health companies and reiterated that while Genflow is not an animal health business, a potential deal could be shaped by the strength of the efficacy data. Visit Proactive's YouTube channel for more exclusive interviews. Don't forget to like this video, subscribe to the channel, and turn on notifications so you never miss an update. #GenflowBiosciences #SIRT6 #GeneTherapy #Sarcopenia #LifeExtension #CanineTrial #BiotechNews #PharmaDeals #AgeingResearch #BiotechInvesting #ClinicalTrials #MitochondrialFunction #BiotechUpdate #ProactiveInvestors
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the exciting, yet challenging, landscape of gene therapies and their potential to revolutionize healthcare. Recent scientific advancements have pushed the boundaries of what's possible, offering potential lifetime cures for diseases once considered incurable. However, this breakthrough comes with a significant economic caveat: the staggering cost of these therapies, often ranging between $3 to $4 million per patient. This price tag presents a formidable challenge to current healthcare infrastructures.The disconnect between these innovative treatments and existing payment systems is evident in what industry experts term the "$4 million payment problem." Therapies like Lenmeldy and Hemgenix highlight this issue. Lenmeldy, for instance, can prevent metachromatic leukodystrophy with a single infusion priced at $4.25 million, while Hemgenix offers a cure for hemophilia B at $3.5 million. These therapies effectively convert lifelong treatment costs into a singular, substantial payment, challenging traditional insurance models that are built to spread costs over time.The primary obstacle is not the efficacy of these treatments but rather the financial and logistical infrastructures needed to support them. The current insurance model is ill-equipped to handle such large, one-time payments. Employers who often provide health insurance face a dilemma: investing millions in curing an employee who might leave the company shortly after receiving treatment could result in significant financial risk and disincentivizes employers from covering such therapies.Enter Aradigm Health, which has emerged as a potential solution to this conundrum. Aradigm aims to create an "infrastructure layer" specifically for these high-cost cures. With $20 million in funding backing their initiative, Aradigm seeks to pool financial risk across multiple employers, thus mitigating the impact of substantial individual claims. Their model involves employers contributing a fixed monthly fee into a shared fund that covers these expensive treatments when needed. This approach distributes financial volatility across a broader base rather than placing it on individual employers.Aradigm's strategy is not only about financial solutions but also about streamlining logistical complexities associated with delivering gene therapies. Their patient journey management includes coordinating with biotech companies for manufacturing schedules, arranging travel and accommodation for patients and families, and ensuring seamless insurance paperwork handling. This comprehensive support system reduces barriers that often delay or disrupt treatment delivery.Operating as a public benefit corporation with a "cost-plus" model, Aradigm ensures that any surplus from lower-than-expected claims is returned to employers rather than kept as profit. This aligns incentives towards patient care rather than profit maximization. Their approach highlights a critical need within the biotech and pharmaceutical industries: developing adaptable infrastructures that align with rapid scientific advancements.Meanwhile, Amgen has secured significant ground in 2023 with its second FDA approval for Uplizna in treating generalized myasthenia gravis—a chronic autoimmune neuromuscular disorder characterized by varying degrees of skeletal muscle weakness. Uplizna's mechanism involves targeting CD19 on B cells implicated in autoimmune diseases' pathogenesis. This expansion marks an advancement in therapeutic options for patients and underscores Amgen's growing footprint in treating complex autoimmune conditions.GlaxoSmithKline has also made headlines with Blujepa, marking it as the first new class of antibiotics for gonorrhea in over three decades while receiving approval for treating uncomplicSupport the show
The Smart 7 is an award winning daily podcast, in association with METRO that gives you everything you need to know in 7 minutes, at 7am, 7 days a week...With over 19 million downloads and consistently charting, including as No. 1 News Podcast on Spotify, we're a trusted source for people every day and the Sunday 7 won a Gold Award as “Best Conversation Starter” in the International Signal Podcast Awards If you're enjoying it, please follow, share, or even post a review, it all helps...Today's episode includes the following guests:Anthony Albanese - Australian Prime Minister Julie Inman Grant - Australia's ESafety Commissioner Dr Rachel Murrihy - Clinical Psychologist and Director of the KIdman Centre at Sydney's University of Technology Melanie Dawes - Chief Executive of Content Regulator Ofcom Lisa Nandy - UK's Culture Secretary Will Guyatt - The Smart 7's Tech Guru Reid Wiseman - NASA astronaut and member of Artemis crew Dr David Burtt - Postdoctoral Fellow at NASA's Goddard Space Flight CentreDr Mike Thorpe - Assistant Research Scientist at the University of Maryland, and the Planetary Environments Lab Division at NASADouglas Gonzaga de Sousa - Co-ordinator of the Centre for Specialty Coffees of Espirito SantoEdmond Rhys Jones - Co-lead at Boston Consulting Groups Centre for Climate and Sustainability Policy & RegulationBill Gates - Co-founder of Microsoft and high profile philanthropist Alyssa Tapley - Took part in the gene therapy trialProfessor Waseem Qasim - Consultant in Paediatric Immunology, and Professor of Cell and Gene Therapy at UCLDr Robert Chiesa - Consultant in Bone Marrow Transplantation at Great Ormond Street HospitalDr Roberto Biaggi - Co-author of the study from California's Loma Linda University.Celso Aguilar - Superintendent of the Toro Toro Park in BoliviaContact us over @TheSmart7pod or visit www.thesmart7.com or find out more at www.metro.co.uk Presented by Ciara Revins, written by Liam Thompson, researched by Lucie Lewis and produced by Daft Doris. Hosted on Acast. See acast.com/privacy for more information.
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a myriad of transformative advancements and strategic shifts within these industries, illustrating the profound impact of innovation and regulatory changes on healthcare.Eli Lilly's recent strides in obesity treatment highlight a significant scientific breakthrough with their novel drug, retatrutide. Currently in phase 3 trials, this triple agonist targets GLP-1, GIP, and glucagon receptors, achieving an extraordinary 28.7% weight loss in participants. Additionally, it demonstrated a 75.8% reduction in knee osteoarthritis pain. Yet, the journey to this milestone wasn't without challenges. The trials saw a higher discontinuation rate than earlier studies, reminding us of the delicate balance between efficacy and patient tolerability—a consistent theme in obesity pharmacotherapy as developers strive to maximize benefits while minimizing adverse effects.In a testament to the global nature of drug development, Zealand Pharma has embarked on a $2.5 billion collaboration with a burgeoning Chinese biotech firm. This partnership aims to advance oral cardiometabolic therapies, underscoring the crucial role of strategic alliances in accessing innovative scientific platforms and expanding market reach.Rezolute faced a significant setback with a disappointing phase 3 trial for its hypoglycemia treatment, resulting in an 87% drop in stock value. This starkly illustrates biotech's inherent volatility and the critical importance of robust clinical trial design to mitigate financial risks associated with unsuccessful outcomes.Meanwhile, Moderna is leveraging Nanexa's expertise in long-acting formulations to enhance injectable therapy delivery systems. This collaboration is indicative of a broader industry trend focused on optimizing drug delivery technologies to boost efficacy and patient compliance.Operational restructuring is also evident as Pfizer implements cost-cutting measures, including layoffs in Switzerland, as part of broader strategic initiatives to optimize operations amid rising R&D costs and pricing pressures. Simultaneously, Chris Boulton's transition from Amgen to Prolynx underscores the fluid movement of talent within the industry—a reflection of ongoing strategic realignments.Regulatory landscapes are shifting as well. The FDA's approval of the first non-drug at-home treatment for depression marks an expansion into alternative therapeutic modalities beyond traditional pharmaceuticals. This wearable device offers adults with major depressive disorder a novel treatment option, integrating technology into mental health care—a promising addition to holistic treatment strategies.In another significant regulatory update, Teva Pharmaceuticals agreed to delist numerous patents from the FDA's Orange Book following an FTC ruling. This move is anticipated to foster increased generic competition and potentially lower medication costs for conditions like asthma and diabetes—a critical shift towards greater market accessibility.The FDA has also finalized guidance on promotional materials for biologics and biosimilars, standardizing advertising practices to ensure accurate representation of these products' efficacy and safety profiles amidst an expanding biologics market.Moreover, the FDA has launched its Commissioner's National Priority Voucher Pilot Review Program to expedite critical drug approvals. The first beneficiary under this program was USAntibiotics with Augmentin XR, signaling a potential shift towards more rapid access to essential medications.On the international policy front, recent developments between the UK and US have led to reductions in medicine rebate rates within the UK. This adjustment could lead to increased spending on new medicines, indicating more favorable conditions for pharmaceutical Support the show
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. In the ever-dynamic landscape of these industries, recent advancements have underscored both the scientific ingenuity and strategic foresight shaping patient care today.Pfizer has unveiled promising clinical trial data for Tukysa, indicating its potential as a first-line maintenance therapy in HER2-positive breast cancer. This development suggests that Tukysa could delay disease progression, offering patients extended survival prospects and an improved quality of life. Additionally, Pfizer's recent licensing agreement with Yaopharma for YP05002—a small molecule GLP-1 agonist currently in Phase 1 trials aimed at obesity treatment—highlights their strategic push into the rapidly evolving obesity treatment market.Meanwhile, Fondazione Telethon, an Italian nonprofit organization, has achieved a significant milestone with FDA approval for Waskyra—the first gene therapy for Wiskott-Aldrich syndrome. This ex vivo gene therapy directly targets the genetic roots of this rare disease, shifting treatment from symptomatic management to addressing underlying causes. This approval is transformative not only for patients suffering from this condition but also for the broader field of gene therapies, heralding a new era in treating rare genetic disorders.On the strategic front, Eli Lilly's decision to establish a $6 billion active pharmaceutical ingredient manufacturing facility in Huntsville, Alabama, marks a pivotal investment in U.S. manufacturing capabilities. This site will be critical in producing APIs for small molecule and peptide medicines, a testament to Lilly's commitment to meeting growing therapeutic demands while bolstering domestic production resilience—a trend gaining momentum across the industry. In oncology, Eli Lilly's Jaypirca demonstrated an impressive reduction in disease progression during Phase 3 trials for chronic lymphocytic leukemia.Biocon's acquisition of Viatris' stake in their biosimilar subsidiary exemplifies the shifting dynamics within the biosimilars market. This move allows Biocon to consolidate its market position as biosimilars gain traction as cost-effective alternatives to branded biologics. Such strategic realignments are indicative of competitive maneuvering aimed at capturing greater market share and driving down healthcare costs.Roche has made strides with compelling results from its Phase 3 trial of giredestrant, an oral selective estrogen receptor degrader showing a 30% reduction in risk for invasive breast cancer recurrence or death. The significance of this development lies in offering an oral alternative to injectable treatments, potentially improving patient adherence and reshaping standard care protocols for hormone receptor-positive breast cancer. Furthermore, Roche has achieved another regulatory milestone with its monoclonal antibody Gazyvaro gaining EU approval for treating lupus nephritis following successful Phase 3 trials.Innovation continues unabated as Formation Bio forms a new subsidiary through a $605 million deal with Lynk Pharmaceuticals. By securing rights to a next-generation immunology asset, Formation Bio positions itself at the forefront of immunological research developments. Concurrently, BioNTech and Bristol Myers Squibb have reported positive results from Phase 2 trials of Pumitamig for triple-negative breast cancer—validating bispecific antibodies' efficacy within oncology.Collaborative efforts are also reshaping industry landscapes. Bora and Corealis have partnered to create an end-to-end contract development and manufacturing organization for oral solid dose drug development. This collaboration aims to streamline processes and provide scalable solutions through a single contracting source, reflecting a shift towards integrated service models that enhance efficiencySupport the show
Guest: Victoria Coleman-Cowger, PhD Gene therapy offers transformative potential for individuals with sickle cell disease, but the psychosocial challenges that accompany such a major decision are often underestimated. Hear from Dr. Victoria Coleman-Cowger as she highlights recommendations for supporting emotional wellbeing during and after gene therapy and explains the importance of recognizing both the psychological and social impacts of curative treatment. Dr. Coleman-Cowger is a licensed clinical psychologist and the Associate Vice President of Neurology and Cell and Gene Therapy at Emmes, a full-service clinical research foundation.
Guest: Victoria Coleman-Cowger, PhD Gene therapy offers transformative potential for individuals with sickle cell disease, but the psychosocial challenges that accompany such a major decision are often underestimated. Hear from Dr. Victoria Coleman-Cowger as she highlights recommendations for supporting emotional wellbeing during and after gene therapy and explains the importance of recognizing both the psychological and social impacts of curative treatment. Dr. Coleman-Cowger is a licensed clinical psychologist and the Associate Vice President of Neurology and Cell and Gene Therapy at Emmes, a full-service clinical research foundation.
In Part 2 of the 2026 pipeline series, host Carolyn Liptak welcomes Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient, to explore the advanced therapies pipeline: cell therapies, gene therapies, tissue-engineered products, and combination advanced therapy products. The discussion explores major pipeline trends, six leading products to watch, and the growing innovation expected to shape clinical practice in 2026. Guest speakers: Amanda Frick, PharmD, BCPS Senior Clinical Manager, Strategic Clinical Intelligence Spend Management Vizient Host: Carolyn Liptak, MBA, BS Pharm Pharmacy Executive Director Center for Pharmacy Practice Excellence (CPPE) Vizient Show Notes: [00:05] — Introduction Announcer opens the episode. Host Carolyn Liptak introduces the focus on advanced therapies: cell & gene therapies, tissue-engineered products, and combination products. Guest: Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient. [01:07] — Defining Advanced Therapies FDA groups cell and gene therapies within advanced therapies. Total FDA-approved advanced therapies: 46. Amanda monitors 29 drug-like therapies within that group. [02:01] — Pipeline Size and Approval Activity S. pipeline: 264 agents in development. About 10 agents approach FDA decision annually. Actual approvals: 5–7 per year on average. [02:56] — Big-Picture Trends in Cell & Gene Therapy Oncology dominates 40–50% of all CGTs in development. Expanding into autoimmune, neurology, and earlier-phase therapies for diabetes, angina, osteoarthritis. Movement toward allogeneic ("off-the-shelf") therapies Designed to overcome limits of autologous cell manufacturing. Reduces wait time and manufacturing failures. Resurgence of therapeutic vaccines Currently 3 approved (Sipuleucel-T, Talimogene, Papzimeos ). 20+ vaccines in the pipeline, largely targeted to cancer. CE program coming Jan 29. [06:13] — Therapy #1: Tabelecleucel or Tab-cel (Allogeneic EBV-Specific T-Cell Therapy) First allogeneic T-cell therapy expected in the U.S. For EBV-positive post-transplant lymphoproliferative disorder (PTLD). “Off-the-shelf” and donor-derived. [07:07] — Clinical Need & Outcomes Currentstandard of care: rituximab. After relapse, survival
In this podcast, we spoke with Troy Ostreng, Senior Product Manager and David Burdge, Director of Cell and Gene Therapy at CPC about the development of the MicroCNX® aseptic micro-connectors and how they're helping biopharma teams streamline closed-system operations for cell and gene therapies. What unfolded was a detailed and forward-looking conversation that touched on CPC's 47-year legacy, the technical demands of advanced therapies, and the company's plans to drive the future of automation and sterility in manufacturing. A Legacy That Positioned CPC for Today's Advanced Therapy Boom When asked how CPC's long history in biologics and hospital environments prepared the company for today's cell and gene therapy landscape, David took us back to CPC's roots. “CPC was founded in 1978, so that's 47 years of innovation within connection technologies,” he said. “The first biologic was released in 1982, synthetic insulin, and we were there supporting the industry with open-format connectors on single-use bags.” From the early development of biologics through the shift to single-use and the rise of stainless-steel/single-use hybrid systems, CPC continuously evolved its connection technologies. They launched steam-through connectors as bioprocessing grew more complex, released their first aseptic connector in 2009, and introduced their first connector specifically targeted for the cell and gene therapy market in 2017. David explained how that history matters today: “Biologics has about a 35-year head start on advanced therapies. So the question becomes, what lessons can we transfer from biologics to cell and gene therapy as that industry grows at three to four times the rate biologics did in its first decade?” That perspective, combining biological manufacturing experience with the needs of new therapy modalities, forms the foundation for CPC's MicroCNX platform. MicroCNX: The First Aseptic Connector Built for Small-Format Tubing As cell and gene therapy developers began scaling up manufacturing, they quickly discovered a problem: the connectors used for biologics were not designed for small-volume, patient-specific therapies. Troy described it plainly: “Several years ago, we started hearing rumblings that current connectors weren't meeting what cell and gene therapy required.” CPC responded with a deep Voice of Customer (VOC) initiative, interviewing process engineers, operators, manufacturing leaders, and platform developers. Over and over, the same needs emerged. Operators wanted something simple. “Ease of use was the number one requirement,” Troy said. “Operators needed a product that was easy to use so they could make sterile connections in a short amount of time.” Processes demanded robustness. “Customers needed a connection they could trust—no contamination, no failures, no weak spots in the connection process,” he added. Small-volume precise applications required connectors actually designed for them. With autologous therapies, he noted, “We aren't talking about 1,000 liters; we're talking about 250 milliliters. And if there's a mishap, that could mean the difference between life and death for a patient.” All of this laid the groundwork for MicroCNX, which became the first aseptic connector engineered for small-format tubing. The “Pinch-Click-Pull” Process: Sterility Meets Speed One of the standout features of MicroCNX is its elegantly simple pinch-click-pull operation. Troy explained how simplicity came directly from user feedback. “As operators walked us through their pain points, what they needed was clear: a connector they could learn immediately. So MicroCNX has a three-step process—pinch, click, pull. You can literally do it as fast as I say it.” He continued,“Once someone does it one time, they're basically an expert. That ease of use dramatically reduces operator error.” For an industry where operator variability remains one of the biggest sources of risk and batch loss, eliminating complexity is critical. Cryogenic Challenges Call for Cryo-Rated Solutions As the conversation shifted to cryopreservation, a critical component of cell therapy manufacturing,Troy introduced the MicroCNX® ULT and MicroCNX® Nano variants. “These were really developed because therapies were being frozen to –150°C, even –190°C. You need a connector that can be frozen to those temperatures, thawed, and still be as robust as it was before.” The ULT and Nano were engineered with: Low-profile geometries to fit inside freezing cassettes Specialized materials to withstand thermal stress Chemical compatibility with DMSO and other cryoprotectants Enhanced durability to survive impacts while frozen Troy emphasized how critical it was to get the materials right: “We searched extensively for a material that could handle those harsh chemicals and temperatures. What we landed on was PPSU—polyphenylsulfone. It's chemically sound, and it's incredibly impact-resistant at very low temperatures.” CPC built these connectors because customers repeatedly told them: existing solutions were cracking, leaking, or becoming brittle. MicroCNX was engineered to overcome all of that. True Closed Systems vs. Functionally Closed Systems: Why the Difference Matters A substantial part of the conversation focused on the differences between closed, functionally closed, and open systems—distinctions that are often overlooked but critically important. Troy broke down the differences clearly: “An open system is exposed at some point. A functionally closed system is inherently open but gets closed temporarily to let fluid transfer. In comparison, a closed system is never open at any point.” Examples of functionally closed systems include: Biosafety cabinets (BSCs) Luer-based connections Closed system transfer devices These approaches require: Sanitization Careful environmental controls Operator expertise And, as Troy noted, “a mishap in one of these can mean losing a very valuable therapy.” CPC's sterile connectors—including MicroCNX minimize these risks: “Our connectors allow the system to remain closed 100% of the time. That greatly reduces contamination risk.” This distinction isn't merely academic—it has direct regulatory implications as well. David added,“In Annex 1, they refer to intrinsically sterile connection devices—like sterile connectors and tube welders—that allow operations normally requiring Grade A or B to occur in a Grade C or D environment.” That ability to operate safely in lower-grade spaces is increasingly critical as the industry tries to overcome facility and labor bottlenecks. Why Teams Are Moving Away from Tube Welding Tube welding has been part of bioprocessing for decades, but David explained why its era may be ending for CGT. “Tube welding was born out of the blood banking industry when no other solution existed. But sterile connectors don't require capital investment. They're faster. They eliminate issues like tubing alignment or pinhole leaks. They're simply more reliable.” As biologics manufacturers have already done, CGT teams are now transitioning toward connectors like MicroCNX® that provide sterile, consistent, low-burden operations. The MicroCNX® Luer Variant: Supporting Transitional Workflows Not all workflows are ready to move away from luer-based devices. That's where the MicroCNX Luer variant fits in. Troy described how it works.“You connect a syringe or bag with a luer inside the BSC, but then because the MicroCNX® connector itself is sterile, you can take it outside the hood and make a sterile connection elsewhere.” This capability bridges legacy workflows and fully closed systems—critical during process development, technology transfer, or when working with specific devices. Co-Development: The Heart of CPC's Innovation Process As the conversation returned to CPC's broader philosophy, David highlighted how important customer collaboration is. “It's all about the customer for CPC,” he said. “We start with Voice of Customer. Our business and applications managers are out in the field understanding real applications and guiding them to the right products.” This feedback fuels CPC's two major development tracks: Catalog product development (platforms like MicroCNX) Custom-engineered solutions for unique applications David added: “We maintain a full new product introduction roadmap. Some products will be released broadly. Others will be developed specifically for one customer. But both are driven by real application requirements.” This process ensures CPC's products evolve in lockstep with the needs of advanced therapy teams. Looking Ahead: Designing Connectors for Robotics and Automation Toward the end of the conversation, David turned to one of CPC's biggest focus areas: the future of automation. “The ultimate customer in this industry is the patient,” he said. “And right now we face barriers—capacity, speed, accessibility, cost. Process automation can significantly reduce those barriers.” Automation requires connectors designed not just for human hands but for robotics: Predictable geometries Features optimized for machine vision Forces and actuation steps compatible with robotic grippers Designs intended for automated loading and unloading David summarized CPC's future direction: “We're taking a fresh look at our connectors, reimagining them as something designed for robotic manipulation. It's a high priority for us.” Troy echoed the sentiment: “Our connectors are awesomely designed for humans. But automation is coming, and we're focused on the features robots need.” A Future Built on Innovation and Patient Impact The interview closed with both guests reflecting on CPC's mission. “We're incredibly passionate about innovation and meeting the needs of our customers through thoughtful product development,” Troy said.
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a series of compelling advancements and challenges that are shaping this dynamic industry.Pfizer has recently unveiled phase 3 results for its hemophilia drug Hympavzi, positioning it as a formidable competitor against Sanofi's Qfitlia and Novo Nordisk's Alhemo. The data suggests Hympavzi offers robust efficacy, potentially revolutionizing hemophilia treatment and enhancing patient outcomes significantly. This development is not just about competition; it represents a critical stride forward in patient care for those affected by this debilitating condition.UCB plans to seek regulatory approval for Fintepla to treat an additional epileptic disorder following positive phase 3 trial results in patients with CDKL5 deficiency disorder. This decision reflects promising results and could offer new hope to patients with limited treatment options, further cementing Fintepla's position in epilepsy management.Gene therapy continues to shine with CSL's Hemgenix demonstrating sustained long-term benefits. After five years of follow-up, a single dose has reduced annualized bleeding rates by an impressive 90% in hemophilia B patients within the Hope-B study. Such long-term efficacy highlights gene therapy's transformative potential, offering lasting improvements in quality of life for patients with genetic disorders.Regulatory frameworks are also evolving, as evidenced by the CDC's Advisory Committee on Immunization Practices (ACIP) voting to modify hepatitis B vaccine guidance for newborns. This decision advocates an individualized approach, sparking debate over vaccination strategies, which reflects the complexities and delicate balance required in public health policies today.Regeneron is making strides to simplify treatment regimens for T-cell engagers Lynozyfic and Ordspono. By reducing regimen complexity, they aim to improve patient compliance and expand access, thus enhancing the potential impact on cancer care—a crucial step toward broader therapeutic accessibility.In an effort to address cost barriers and stimulate domestic production of generics, Mark Cuban has proposed lowering FDA fees. This proposal highlights ongoing discussions around regulatory reforms needed to boost generics manufacturing in the United States. Such initiatives align with broader industry goals of increasing access to affordable medications.Despite these advancements, industry insiders have expressed concerns about "unprecedented turmoil" within the FDA. These challenges underscore the critical role of stable leadership in maintaining public trust and ensuring effective regulation amidst rapid scientific progress.Obesity treatments are gaining significant attention as companies like Wave Life Sciences and Structure Therapeutics report promising data, capturing increased investor interest. This trend underscores a growing focus on innovative pharmacological approaches to address obesity—a complex, multifactorial condition that affects millions globally.The strategic landscape of cancer diagnostics is also evolving, as evidenced by Natera's acquisition of Foresight Diagnostics. This deal underscores ongoing industry consolidation efforts aimed at enhancing technological capabilities and expanding market presence—a testament to the critical role diagnostics play in comprehensive cancer care strategies.As we observe these developments, it's clear that scientific innovation coupled with strategic regulatory maneuvers is shaping the future of healthcare. These advancements offer profound implications for patient care, providing new hope through advanced therapies while highlighting the importance of effective regulation and strategic partnerships.Precision medicine continues to extend beyond oncology into fields like cardiometabolic and neSupport the show
Send us a textDr. Norman Putzki, MD is Senior Vice President, Global Clinical Development Head, and U.S. Development Site Head at Novartis ( https://www.novartis.com/ ) where he oversees global teams working on next-generation gene therapies, RNA-based medicines, targeted biologics, and innovative small molecules.Dr. Putzki most recently served as Global Head of Development for Neuroscience and Gene Therapy at Novartis, where he oversaw one of the world's most ambitious pipelines aimed at transforming the lives of patients with neurological, neuromuscular, and rare genetic diseases. A physician–scientist by training, with an MD from University of Duisburg Essen, Dr. Putzki has built a career at the intersection of clinical medicine, translational research, and large-scale drug development. Before joining Novartis, Dr. Putzki led programs across multiple therapeutic areas at Biogen Idec and has played key roles in advancing clinical treatments for conditions long considered intractable including MS and Parkison's disease.Beyond his scientific and clinical contributions, Dr. Putzki is widely recognized for his commitment to patient-centered innovation and his ability to bring together cutting-edge science, regulatory acumen, and real-world medical insight. Important Episode Links - Zolgensma Web Site - https://www.zolgensma.com/Itvisma Web Site - https://www.itvisma.com/Nature Medicine Papers For Itvisma Phase III study - Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial - https://www.nature.com/articles/s41591-025-04103-wIntrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial - https://www.nature.com/articles/s41591-025-04119-2#NormanPutzki #Novartis #Neuroscience #GeneTherapy #SpinalMuscularAtrophy #SMN1 #SurvivalMotorNeuron1 #Zolgensma #OnasemnogeneAbeparvovec #AdenoAssociatedVirusSerotype9 #AAV9 #SMA #AveXis #Itvisma #NewbornScreening #ProgressPotentialAndPossibilities #IraPastor #Podcast #Podcaster #ViralPodcast #STEM #Innovation #Technology #Science #ResearchSupport the show
Send us a textOn the latest WTR Small-Cap Spotlight podcast episode, we welcomed Jan Eryk Umiastowski, CFO of GenSight Biologics (Euronext Paris: SIGHT), a late clinical-stage biopharma company based in Paris, France, primarily focused on developing and commercializing novel disease-modifying gene therapies aimed at reversing vision loss from severe neurodegenerative retinal diseases. He discusses GenSight's development pipeline, what's unique about the company's approach to gene therapy and upcoming target milestones.
Novo Nordisk's highly anticipated data for semaglutide in Alzheimer's dashed hopes that the GLP-1 therapy could become a game changer in the disease. On the latest BioCentury This Week podcast, Executive Editor Selina Koch discusses the Phase III readout including what it says about the mechanism's use in the neurodegenerative disease, Novo's decision to skip Phase II and enroll a large patient group in a later stage trial, and what other datasets for GLP-1s in the indication have shown.Washington Editor Steve Usdin explains why FDA's new vaccine policies, driven by CBER Director Vinay Prasad, could have impacts more far-reaching than expected, including making it more difficult to develop or modify vaccines. And Lauren Martz, Executive Director of Biopharma Intelligence, analyzes the bleak investment outlook for cell and gene therapy companies in the U.S. and why China's biotech ecosystem offers a glimmer of hope for sponsors of these assets.View full story: https://www.biocentury.com/article/657721#Semaglutide #GLP1Therapies #VaccinePolicy #CellAndGeneTherapy #AlzheimersResearch00:00 - Introduction01:53 - Novo's Alzheimer's Miss13:19 - FDA's New Vaccine Policy19:53 - Funding C> BiotechsTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text
Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we'll explore the latest advances and strategic moves shaping the industry, providing you with insights into how these developments might influence drug development and patient care.The pharmaceutical and biotech sectors are currently experiencing a wave of transformative changes. A significant development is the trade agreement between the United States and the United Kingdom, which excludes medicines from import tariffs. This strategic move, orchestrated by the Trump administration, is set to reduce costs and bolster investments in pharma sectors across both nations. By enhancing market accessibility, it aims to stimulate cross-border investment in pharmaceutical research and production.On the clinical front, Eli Lilly is making headlines by joining Novo Nordisk in reducing self-pay prices for its GLP-1 receptor agonist, Zepbound. This reflects a broader industry trend towards patient-centric pricing models aimed at improving affordability. With healthcare costs on the rise, these measures could ease financial burdens for patients requiring long-term medication regimens.Regulatory updates are also making waves, with the FDA planning stricter vaccine regulations under Dr. Vinay Prasad's leadership at the Center for Biologics Evaluation and Research (CBER). These updates come amid concerns over vaccine safety during COVID-19-related incidents, underscoring a commitment to maintaining public trust in vaccines. Regulatory scrutiny continues as Prasad focuses on COVID-19 vaccine safety in children amid reports linking 10 child deaths to vaccines via VAERS—emphasizing challenges interpreting safety data while highlighting the need for robust methodologies ensuring reliable causality conclusions.In corporate restructuring news, Valneva is streamlining its operations by closing a site and eliminating 30 roles. This move highlights an industry focus on optimizing resources to bolster vaccine development pipelines. Meanwhile, Microsize and Schedio's acquisition of Lonza's Swiss micronization plant underscores ongoing investments in advanced manufacturing technologies critical for high-quality pharmaceuticals.In ophthalmology, Belite Bio is advancing with promising Phase 3 results for tinlarebant in treating Stargardt disease—a rare genetic eye disorder. This success positions Belite to file for FDA approval, potentially expanding treatment options for this underserved patient population. Such advancements in targeted therapies emphasize the need for ongoing research in genetic disorders.Regeneron is betting $150 million on Tessera's gene writing technology targeting alpha-1 antitrypsin deficiency (AATD). This collaboration highlights the industry's growing interest in gene therapy as a frontier for treating rare diseases, marking a shift towards precision medicine where tailored genetic interventions offer hope for previously untreatable conditions. Regeneron's strategic move investing $275 million partnering with Tessera exploring gene editing capabilities—focusing on TSRA-196 targeting the SERPINA1 gene linked to AATD showcases potential advancing therapeutic options for genetic disorders through gene editing technologies offering new disease treatment avenues.AI-driven platforms were spotlighted at RSNA 2025 by industry leaders such as GE Healthcare, Philips, and Siemens. These innovations promise to revolutionize radiological workflows by enhancing diagnostic accuracy and operational efficiency through AI integration. As AI continues to permeate healthcare technologies, its potential to transform diagnostic processes marks a significant leap towards personalized medicine.Akebia Therapeutics' acquisition ofSupport the show
What if an Alzheimer's therapy did not just slow decline, but restored memory and physical health back to baseline?In this episode of Spark Time, we sit down with Dan Salain, President and CEO of Recall Therapeutics, and Dr. Atul Chopra, Co-founder and CSO, to unpack one of the boldest ideas in neuroscience today: RTX-100, a gene therapy designed to restore cognition and systemic health in Alzheimer's disease. Dan and Atul walk us through the science behind targeting memory circuits rather than just clearing amyloid, how a liver-expressed protein naturally crosses the blood–brain barrier, and why their preclinical data show full recovery of associative, recognition, and spatial memory in advanced mouse models.We also explore the overlooked systemic side of Alzheimer's cachexia, sarcopenia, frailty, UTIs, pneumonia and how RTX-100 appears to normalize body weight, muscle strength, endurance, and fluid balance alongside cognitive recovery. From serendipity in the lab to regulatory strategy and potential paths to market, this conversation opens up a very different vision for what treating Alzheimer's and even aging itself could look like.
It Happened To Me: A Rare Disease and Medical Challenges Podcast
In this uplifting episode, hosts Beth Glassman and Cathy Gildenhorn sit down with Leanna Scaglione, a powerhouse rare disease advocate and marathon runner living with NF2-Related Schwannomatosis (NF2-SWN), formerly known as neurofibromatosis type 2. Diagnosed at just 16 years old, Leanna's life changed dramatically when tumors were discovered in her nervous system, a hallmark of NF2-SWN. Many would have slowed down. Instead, Leanna sped up. Today, she has: Completed multiple marathons, including Berlin and New York City Participated in clinical trials Become an advocate and 2025 Ambassador for the Children's Tumor Foundation Set her sights on becoming the first person with NF2 to complete all 7 Abbott World Marathon Majors Her message is powerful: a diagnosis doesn't define the finish line. In This Episode, We Discuss: What NF2-SWN is and how it impacts the nervous system The emotional toll of receiving a life-altering diagnosis as a teenager Losing a dream — and finding a new one Running marathons through surgeries, treatments, and uncertainty How advocacy and visibility can change the rare disease landscape Building a life rooted in resilience, purpose, and possibility About Our Guest: Leanna Scaglione is 34 years old living with NF2-Related Schwannomatosis (NF2-SWN). Ever since being diagnosed at 16 years old, she has refused to live defined by her diagnosis. From relearning to walk, participating in immunotherapy drug trials, and going through numerous surgeries she has defied the odds against her. Most recently she has undergone surgery to remove her right acoustic neuroma. She continues to live her best life as an endurance runner, sharing her story and experiences living with NF2 in hopes to spread awareness, motivate her community and rally for a cure. Resources: Children's Tumor Foundation NF2 Accelerator: A Strategic Portfolio Approach to End NF2 Articles Explaining NF2 MedlinePlus GeneReviews National Organization for Rare Disorders (NORD) Research from China that Leanna reference for a gene therapy for NF2 Yuan R, Wang B, Wang Y, Liu P. Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions. Oncol Ther. 2024 Jun;12(2):257-276. doi: 10.1007/s40487-024-00279-2. Epub 2024 May 17. PMID: 38760612; PMCID: PMC11187037. Additional Research Articles Alexandra K. O'Donohue, Samantha L. Ginn, Gaetan Burgio, Yemima Berman, Gabriel Dabscheck, Aaron Schindeler, The evolving landscape of NF gene therapy: Hurdles and opportunities, Molecular Therapy Nucleic Acids, Volume 36, Issue 1, 2025, 102475 ,ISSN 2162-2531, https://doi.org/10.1016/j.omtn.2025.102475. Connect With Us: Stay tuned for the next new episode of “It Happened To Me”! In the meantime, you can listen to our previous episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “It Happened To Me”. “It Happened To Me” is created and hosted by Cathy Gildenhorn and Beth Glassman. DNA Today's Kira Dineen is our executive producer and marketing lead. Amanda Andreoli is our associate producer. Ashlyn Enokian is our graphic designer. See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, ItHappenedToMePod.com. Questions/inquiries can be sent to ItHappenedToMePod@gmail.com.
Host Matt Burgess speaks with genetic counsellor and NSGC president Sara Pirzadeh‑Miller about her early research on values in genetic counselling, how life experience shapes practice, and the evolving role of genetic counsellors. They also discuss the clinical challenges of the CDH1 gene, risk interpretation, screening versus prophylactic surgery, the rise of genetic counselling assistants, and future genomic innovations such as gene therapies and improved risk stratification.
Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today we dive into some of the most significant and exciting advancements shaping the industry.Let's start with a breakthrough coming out of recent clinical trials. A novel approach to treating Alzheimer's disease is making waves. Researchers have been focusing on a monoclonal antibody designed to target amyloid-beta plaques in the brains of patients suffering from this debilitating condition. The latest Phase 3 trial results have shown a promising reduction in cognitive decline among participants, offering a glimmer of hope for millions affected by Alzheimer's worldwide. The study, conducted over 18 months, involved more than 1,500 patients and demonstrated a statistically significant slowing of disease progression compared to placebo. This could mark a pivotal moment in Alzheimer's treatment, shifting the paradigm from symptomatic relief to disease modification.In regulatory news, the FDA has granted approval to a new gene therapy for hemophilia B. This therapy, developed using cutting-edge CRISPR technology, offers a potential cure for patients who have long relied on regular infusions to manage their bleeding disorder. By directly editing the genes responsible for clotting factor production, this therapy can potentially provide long-term relief with just a single administration. The approval follows extensive clinical trials that showed sustained increases in clotting factor levels and a dramatic reduction in bleeding episodes among participants. This development not only underscores the growing impact of genomic medicine but also highlights the regulatory body's commitment to advancing innovative treatments that meet unmet medical needs.Shifting focus, there's an intriguing trend emerging in oncology drug development. The industry is increasingly adopting personalized medicine approaches, tailoring treatments based on individual genetic profiles. This shift is particularly evident in the rise of targeted therapies and combination regimens designed to tackle cancer at its genetic roots. A recent study demonstrated how combining targeted therapies with immune checkpoint inhibitors can enhance treatment efficacy in certain types of cancer, such as melanoma and non-small cell lung cancer. By understanding the specific mutations driving tumor growth and leveraging the body's immune response, these combinations are setting new standards for cancer care.In another significant development, researchers have unveiled promising results from an innovative mRNA vaccine trial aimed at combating infectious diseases beyond COVID-19. The technology, which gained prominence during the pandemic, is now being applied to other viral threats like influenza and RSV. Early-phase clinical trials have shown robust immune responses and favorable safety profiles, suggesting mRNA vaccines could soon revolutionize how we approach vaccination for a variety of pathogens. This versatility and rapid development timeline make mRNA platforms particularly attractive for addressing emerging infectious diseases swiftly.Meanwhile, a new study has raised awareness about antibiotic resistance—a growing concern globally. Scientists have developed an advanced diagnostic tool capable of rapidly identifying bacterial infections and their resistance profiles within hours instead of days. This innovation can significantly impact how clinicians prescribe antibiotics, ensuring targeted treatments that minimize resistance development. By providing real-time insights into bacterial genetics and resistance mechanisms, this tool empowers healthcare providers to make informed decisions that preserve antibiotic efficacy for future generations.Lastly, let's toSupport the show
Happy Thanksgiving… Thursday, November 27, 2025. Week 48. Continued from #S10e189… And the AAV Paper (https://pubmed.ncbi.nlm.nih.gov/40988338/) from #S10e187… https://curesyngap1.org/podcasts/syngap10/clinical-research-ai-dx-nl47-survey-autism-press-6-days-to-register-for-syngap1conf-s10e187/ https://curesyngap1.org/blog/ Issac's story, Transmitter reprint, Scramble 4 write up and JK on #Autism, #MustRead https://curesyngap1.org/resources/webinars/ 119 - 112 Register for livestream of the conference, AAV from Allen Inst., dos en espanol, Missense, Unlock and Rare-X for ProMMiS. https://curesyngap1.org/podcasts/syngap1-stories/ A gold mine have you listened to #38, the Virginie Pod, really must listen, she is our leader. https://www.linkedin.com/posts/graglia_syngap1stories-syngap1-syngap1storiesty-activity-7387203351907708928-liNL CLINICAL TRIAL & GENETIC MEDICINE CORNER Example of Ultragenyx FAST Angelman follow on trial to look at other ages and genotypes, key message, never give up. https://www.linkedin.com/posts/cureangelman_the-global-aurora-study-will-enroll-approximately-activity-7389647402690957312-Bihi Congrats to Novartis on approval of the first Gene Therapy to Cure SMA! https://www.linkedin.com/posts/graglia_sma-fdaapproval-rarediseaseinnovation-activity-7398939783005347840-Ocd_ Remember Spinraza was approved in December 2019. TODOS Sign up for Citizen Health: https://www.citizen.health/partners/srf USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/ SOCIAL MATTERS 4,468 LinkedIn. https://www.linkedin.com/company/curesyngap1/ 1,480 YouTube. https://www.youtube.com/@CureSYNGAP1 11.2k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1/ $CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ Episode 190 of #Syngap10 #CureSYNGAP1
This week, a world first gene therapy treats rare Hunter syndrome. Could these personalised medicines be used more widely? We speak to Claire Booth, professor in Gene Therapy at Great Ormond Street Hospital. And high in the Chilean desert, the last bit of 13 billion year old light has hit the mirror of the Atacama Cosmology Telescope for the last time. Dr Jenifer Millard, a science communicator and host of the Awesome Astronomy podcast, tells us what it's been up to for the past 20 years.And Penny Sarchet, managing editor at New Scientist brings her pick of the latest new discoveries.Think you know space? Head to bbc.co.uk, search for BBC Inside Science, and follow the links to the Open University to try The Open University Space Quiz. Presenter: Tom Whipple Producers: Alex Mansfield, Ella Hubber, Jonathan Blackwell, Tim Dodd and Clare Salisbury Editor: Martin Smith Production Co-ordinator: Jana Bennett-Holesworth
Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve deep into a series of transformative events that underscore the dynamic nature of our industry, where scientific innovation meets regulatory evolution and market adaptation.We begin with significant regulatory news from Medicare, which recently announced price reductions for 15 prescription drugs, including Novo Nordisk's semaglutide products, Ozempic and Wegovy. This initiative is part of the Inflation Reduction Act aimed at making essential medications more affordable. By potentially increasing accessibility to these treatments, this move highlights a growing trend towards cost containment in drug pricing within the U.S. healthcare system. It reflects a broader effort to ensure that life-saving treatments remain within reach for more patients, emphasizing the need for balance between innovation and affordability.Turning to approvals, Otsuka has secured FDA clearance for Voyxact, a first-in-class treatment targeting IgA nephropathy (IgAN). This positions Otsuka in an increasingly competitive market space populated by major players like Novartis and Vertex. The entry of Voyxact could pave the way for innovative therapeutics in kidney diseases, offering new hope to patients who have had limited treatment options until now.On the other side of the Atlantic, French authorities have conducted a raid on Sanofi's headquarters as part of a tax fraud investigation. This development sheds light on ongoing scrutiny in the pharmaceutical sector regarding financial practices and regulatory compliance. Such investigations can have far-reaching implications on corporate governance and transparency, reminding us of the importance of ethical practices in maintaining industry trust.Novo Nordisk has strategically used its FDA national priority voucher to expedite the review process for a high-dose formulation of Wegovy. This move underscores the importance of regulatory incentives in accelerating drug development timelines, allowing for quicker patient access to potentially life-changing therapies. It's a testament to how strategic navigation through regulatory pathways can significantly impact drug availability.In clinical trials, Sarepta Therapeutics received FDA clearance to conduct a study combining its gene therapy Elevidys with sirolimus in patients with Duchenne muscular dystrophy. The study aims to address liver safety issues associated with Elevidys, which had led to previous label restrictions. This reflects the industry's commitment to enhancing therapeutic safety profiles while expanding treatment indications.In oncology advancements, AstraZeneca's Imfinzi received FDA approval for use in early-stage stomach cancer, marking its third perioperative indication. This approval underscores the expanding role of immunotherapy across various cancer types and stages, offering new treatment paradigms that could improve surgical outcomes and long-term patient survival.Despite these advances, there is skepticism regarding artificial intelligence's role in regulatory compliance submissions among pharmaceutical professionals. A survey reveals that 65% express distrust towards AI-generated outputs, highlighting challenges that AI technologies face in gaining acceptance within highly regulated environments such as pharmaceuticals. However, federal recommendations to revamp U.S. biotechnology research emphasize incorporating AI into scientific processes to maintain global competitiveness. This call reflects concerns over potential declines in innovation leadership and underscores the need for strategic investment in research infrastructure.In antitrust news, the Federal Trade Commission (FTC) outlined its case agaiSupport the show
Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a landscape rich with scientific innovation, regulatory scrutiny, and strategic business decisions shaping the future of healthcare.Let's begin with Novartis, which has achieved a significant milestone by securing FDA approval for Itvisma. This is an intrathecal formulation of its gene therapy Zolgensma, designed to treat older patients with spinal muscular atrophy (SMA). Priced at $2.59 million, this approval is a critical advancement in gene therapy for SMA, broadening the treatment horizon for a wider patient demographic. Gene therapies are increasingly crucial in addressing rare genetic disorders, offering transformative potential in patient care. Novartis's SMA market expansion post-FDA approval of Itvisma not only enhances its SMA portfolio but also signifies growing acceptance towards gene therapies as viable treatment options for genetic disorders. Meanwhile, Novartis is strategically restructuring, planning to cut 550 jobs at a Swiss plant by 2027 while expanding its workforce in North Carolina. This move reflects broader industry trends toward optimizing global operations and investing in regions with strategic manufacturing capabilities.In the sphere of regulatory scrutiny, lawmakers are questioning the FDA's National Priority Voucher Program amid concerns about corruption and expedited reviews. This situation highlights ongoing challenges within regulatory frameworks to balance innovation speed with rigorous safety assessments. An investigation into the FDA's new priority review voucher program has been initiated due to concerns over corruption and expedited review processes potentially compromising drug safety. This inquiry could influence future regulatory frameworks and underscores balancing accelerated drug approvals with rigorous safety standards. Richard Pazdur expressed concerns about expedited drug approval programs' safety and legality as he takes on his new role as director of the FDA's Center for Drug Evaluation and Research. These initiatives aim to accelerate drug reviews but spark debate over patient safety implications—underscoring an ongoing tension between innovation speed and regulatory diligence.Turning to Novo Nordisk, their expansion of the Amycretin program demonstrates a commitment to tackling chronic conditions like diabetes. Following promising Phase 2 data showcasing dual agonist capabilities, Novo Nordisk is advancing pivotal trials focused on obesity. This strategic pivot aligns with market needs and scientific discoveries that could significantly enhance diabetes management options. Further emphasizing Novo Nordisk's commitment to diabetes management, their expansion of the amycretin program after promising Phase 2 results demonstrates the efficacy of a dual agonist originally focused on obesity. This underscores a trend toward multifunctional biologics addressing metabolic disorders by targeting multiple pathways—indicative of broader industry shifts towards integrated therapeutic approaches. Novo Nordisk's recent mid-stage clinical trial results for Amycretin—a weight loss treatment—are noteworthy as they demonstrated sustained efficacy over 36 weeks in type 2 diabetes patients without a plateau in weight loss. Analysts highlight its potential as a superior therapeutic option in the burgeoning weight loss market due to its durable solution for weight management.On a contrasting note, SK Life Science encounters regulatory hurdles as the FDA scrutinizes advertising practices related to its antiseizure medication Xcopri. This scenario underscores the complex interplay between marketing strategies and regulatory compliance within the pharmaceuticSupport the show
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Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a host of transformative events reshaping the landscape, from strategic acquisitions and funding infusions to regulatory maneuvers and scientific breakthroughs.Johnson & Johnson has taken a decisive step in its oncology strategy with the $3 billion acquisition of Halda's cell death technology. This acquisition, focusing on the "hold and kill" bifunctional small molecule platform, is poised to enhance J&J's prostate cancer pipeline significantly. It underscores J&J's commitment to expanding its oncology portfolio through innovative platforms designed to improve therapeutic outcomes. The move highlights a broader industry trend toward personalized medicine and targeted cancer therapies, which are becoming pivotal in improving patient care.In another domain of cancer treatment, Nuvalent has unveiled promising Phase 1/2 data for its candidate neladalkib, which could position the company as a formidable competitor to Pfizer's established lung cancer drug, Lorbrena. The promising data might expedite regulatory discussions with the FDA, potentially leading to an accelerated approval process. This development illustrates the competitive landscape in oncology, where firms strive to introduce novel therapies with improved efficacy and safety profiles.The field of antibody-drug conjugates (ADCs) is also experiencing significant advancements. A San Diego-based biotech has secured $120 million in funding to develop a best-in-class ADC formula, with support from Merck & Co. This initiative aims to refine the precision and efficacy of ADCs by delivering cytotoxic agents directly to cancer cells while minimizing collateral damage to healthy tissues. Such innovations are crucial as they represent a new frontier in targeted cancer therapy.In terms of financial activities, Artios Pharma's successful $115 million Series D funding round is set to bolster its clinical efforts in exploring DNA damage response inhibitors for cancer treatment. These inhibitors target cancer cells' ability to repair DNA damage, holding potential for more effective therapies against resistant cancer types. Meanwhile, Sofinnova Partners' €650 million raise for biotech and medtech investments amid a volatile economic environment underscores continued investor confidence in life sciences despite market uncertainties.Bayer is making strategic moves in China by opening an incubator in Beijing. This facility will host local biopharma companies such as Suzhou Puhe Biopharma and Beijing Youngen Technology, fostering innovation and collaboration within China's burgeoning biotech landscape. Such initiatives reflect global efforts to leverage regional strengths and foster cross-border collaborations.On the operational side, Nxera Pharma is restructuring its workforce by laying off 15% of its staff as part of a strategic pivot towards profitability. This decision mirrors broader industry trends where companies refocus resources on core projects to streamline operations and enhance financial stability.A recent study has highlighted the impact of NIH grant cuts on clinical trials across the United States. Over 383 trials involving more than 74,000 patients have been disrupted due to funding terminations under the current administration. This situation raises concerns about the sustainability of clinical research funding and its implications for ongoing medical advancements.Jazz Pharmaceuticals has reported practice-changing Phase 3 results for its HER2-targeted drug Ziihera for gastroesophageal adenocarcinoma. These findings reaffirm Jazz's confidence in positioning Ziihera as a preferred first-line treatment option for HER2-positive cancers, poSupport the show
In this episode of the Parkinson's Podcast, learn about gene therapy for Parkinson's, including common misunderstandings and ongoing research about it. This episode contains some technical terms and abbreviations. For your convenience, here is a short list of definitions that might be helpful. AADC – Aromatic L-Amino Acid Decarboxylase: An enzyme that helps make dopamine (not a rock and roll band) AAV – Adeno-Associated Virus: A harmless virus used to deliver gene therapy CDNF – Cerebral Dopamine Neurotrophic Factor: A protein that helps keep dopamine-producing cells healthy DBS – Deep Brain Stimulation: A surgical treatment that uses electrical signals to help control symptoms GAD – Glutamic Acid Decarboxylase: An enzyme that helps rebalance brain circuits. GBA – A gene linked to Parkinson's risk GCH1 – GTP Cyclohydrolase 1: An enzyme that supports dopamine production. GDNF – Glial Cell Line-Derived Neurotrophic Factor: A protein that helps protect brain cells LRRK2 – A gene linked to Parkinson's risk PARK2 – A gene linked to Parkinson's risk PET – Positron Emission Tomography: A brain scan that shows chemical activity PINK1 – A gene linked to Parkinson's risk SNCA – A gene linked to Parkinson's risk More information about Gene Therapy for Parkinson's is available here: https://davisphinneyfoundation.org/blog/gene-therapy-parkinsons/ https://youtu.be/n2KNtRK3SIk https://youtu.be/v9mJiO4EEag Sign up for updates on webinars, events, and resources for the Parkinson's community: https://dpf.org/newsletter-signup
The Foundation funded preclinical studies for the approach and launched Opus.
Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]
Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]
Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]
Clinical trials turn scientific discoveries into real options for patients and volunteers, advancing care while safeguarding participants. Sheldon Morris, M.D., M.P.H., explains how independent oversight, core ethical principles, and stepwise phases evaluate safety and benefit, and clarifies participants' rights and responsibilities. Sandip Patel, M.D., F.A.S.C.O., highlights how carefully designed studies open access to promising cell and gene-based approaches while balancing risks and benefits. Robert A.J. Signer, Ph.D., underscores why participation—including consenting to share samples and data—helps researchers understand disease and accelerate better treatments. Morris, Patel, and Signer point audiences to practical ways to locate studies through registries and national databases, discuss what costs are typically covered, and advise asking questions about study design and safety. They also caution against unproven “stem cell tourism” and emphasize informed decisions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40438]
We discuss the early results of gene therapy of neovascular AMD by intravitreal administration of the vector with Dr. Glenn Yiu, Professor of Ophthalmology, University of California, Davis.
In this episode Rafael sits down with Michelle Chen, President and CEO of Form Bio, a techbio company using AI to advance genome engineering and the development of genetic medicines. Michelle shares her zigzag journey from scientist to executive, how her team is tackling the biggest bottlenecks in gene therapy, and why AI has become essential in designing safer, more effective treatments. TPM E51 highlights >Episode 51 links:Michelle Chen on LinkedInForm Bio
Host Matt Burgess speaks with Assistant Professor Barbara Harrison of Howard University about APOL1-related kidney disease, sickle cell disease, and the role of cultural awareness in genetic counselling. They discuss health inequities, community trust around new therapies like gene editing, and efforts to increase diversity in the profession through the GOLDEN mentorship program.
For the first time in human history, we can diagnose thousands of genetic diseases—often for under $1,000—but we still can't treat most of them. The problem isn't understanding what's broken; it's delivering the fix to the right cells.Eric Kelsic, CEO of Dyno Therapeutics, joins a16z's Jorge Conde to explain how AI-designed protein shells are solving gene therapy's delivery crisis. They explore why Huntington's patients can now get 15 extra years of healthy life, how Dyno inverted the liver-to-brain delivery ratio by 1000x, and why capsids evolved by nature are now being designed by machine learning models trained on millions of variants.Eric introduces the concept of genetic agency—humanity's first-ever ability to take action at the DNA level—and details why solving delivery for common diseases will make ultra-rare disease treatments economically viable. Plus: what happens when gene therapy requires neurosurgery today but could be a simple injection tomorrow, why recent deaths in clinical trials prove we need better technology now, and how genetic medicine could become as routine as surgery within our lifetimes. Resources:Follow Eric on X: https://x.com/ekelsicFollow Jorge on X: https://x.com/JorgeCondeBioLearn more about GATC 2025: https://www.dynotx.com/gatc2025 Stay Updated:If you enjoyed this episode, be sure to like, subscribe, and share with your friends!Find a16z on X: https://x.com/a16zFind a16z on LinkedIn: https://www.linkedin.com/company/a16zListen to the a16z Podcast on Spotify: https://open.spotify.com/show/5bC65RDvs3oxnLyqqvkUYXListen to the a16z Podcast on Apple Podcasts: https://podcasts.apple.com/us/podcast/a16z-podcast/id842818711Follow our host: https://x.com/eriktorenbergPlease note that the content here is for informational purposes only; should NOT be taken as legal, business, tax, or investment advice or be used to evaluate any investment or security; and is not directed at any investors or potential investors in any a16z fund. a16z and its affiliates may maintain investments in the companies discussed. For more details please see a16z.com/disclosures. Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.
Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations. Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients." "To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon." "I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future." #ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials advi.com Download the transcript here
Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations. Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients." "To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon." "I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future." #ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials advi.com Listen to the podcast here
From childhood curiosity and early passions to her son's life-changing diagnosis, Liz shares the deeply personal journey that turned her into one of the most daring figures in longevity science — even becoming the first human to test her own gene therapy.Together, Len and Liz dive into:The truth about telomeres, stem cells, and the hallmarks of agingHow AAV gene therapy could rewrite the human lifespanThe clash between innovation, regulation, and Big PharmaWhy mental health, music, and creativity are essential to longevityAnd what the future of aging gracefully — or not at all — might look likeBy the end, you'll be questioning what it really means to live a long life… and whether immortality is closer than we think. EndoDNA: Where Genetic Science Meets Actionable Patient CareEndoDNA bridges the gap between complex genomics and patient wellness. Our patented DNA analysis platforms and AI technology provide genetic insights that support and enhance your clinical expertise.Click here to check out to take control over your Personal Health & Wellness Connect with EndoDNA on SOCIAL: IG | X | YOUTUBE | FBConnect with host, Len May, on IG Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.
How far has gene therapy and genetics medicine advanced? How accessible is that healthcare to Montanans? Click on the podcast to learn more about how Shodair Children’s Hospital‘s world-class genetics program has served Montana families for 50 years, and how […] The post Making Gene Therapy Accessible in Montana first appeared on Voices of Montana.
Gene therapy isn't science fiction anymore, it's becoming one of the most exciting frontiers in diabetes care. In this episode, Dr. Jeremy Pettus and Dr. Steve Edelman sit down with Dr. Fraser Wright, career gene therapist and Co-Founder/Chief Gene Therapy Officer at Kriya Therapeutics, to explore how gene therapy could one day help the body make its own insulin.Together, they unpack what gene therapy actually is, Dr. P's involvement as a medical consultant for Kriya Therapeutics, how AAV (adeno-associated virus) vectors work, and what makes this “one-and-done” approach so different from traditional treatments. From success stories in blindness and hemophilia to emerging type 1 diabetes studies, the trio breaks down the science, the safety, and the hope behind this revolutionary research.You'll learn how gene therapy has moved from rare diseases to more common ones, why the first human trials in diabetes are on the horizon, and what and what this could mean for long-term blood sugar control and independence from injections.Key Topics:Gene Therapy 101: How gene therapy differs from protein-based drugs like insulin, and why AAV vectors act as safe, engineered delivery vehicles rather than infectious viruses.Why AAV, Why Now: Seven FDA-approved AAV-based therapies have proven the potential of long-term, single-dose treatments.From Eyes to Endocrine: Lessons learned from retinal gene therapy are now guiding approaches to metabolic conditions like diabetes.The Type 1 Diabetes Approach: A muscle-targeted program aims to help the body naturally produce insulin and stabilize blood sugar levels.Control and Safety: Built-in glucose sensing and the ability to turn off gene expression ensure precision and reversibility.Real-World Considerations: How exercise, treatment site, and existing technologies like pumps or CGMs could work alongside this therapy.0:00 – Intro: Jeremy and Steve introduce gene therapy and why this topic is a game-changer2:15 – What gene therapy is and how it's different from standard protein-based treatments4:54 – Understanding AAV: safety, engineering, and why it's the preferred delivery method8:50 – Real-world success: seven FDA-approved AAV therapies for genetic diseases12:20 – Lessons learned from treating blindness and how they apply to diabetes17:40 – The Type 1 Diabetes model: using muscle tissue to produce insulin22:15 – Managing control, glucose sensing, and preventing hypoglycemia25:50 – Safety measures and how treatment can be reversed locally if needed28:30 – Exercise, durability, and what animal studies reveal about real-life performance31:10 – Timelines, trials, and what's next for gene therapy in diabetes. What's Ahead: Clinical trials expected to begin around 2026, offering cautious but real optimism for the future of diabetes treatment.Visit TCOYD's Website for more diabetes edutainment for people living with diabetes: tcoyd.org**Tune in for two new episodes each month! Like what you hear and want to help us grow? Please rate and review this podcast so we can reach more people living with diabetes!**Follow our social media channels to empower yourself with the essential areas of diabetes knowledge led by two endocrinologists living with type 1 diabetes: Facebook | Instagram | YouTube ★ Support this podcast ★
What if your skin cells could help you have a baby? Could the future of fertility mean having genetically related children even if a woman is in menopause or lost fertility due to cancer treatments? In this fascinating episode of Brave & Curious, Dr. Lora Shahine sits down with Dr. Paula Amato, reproductive endocrinologist and researcher at Oregon Health & Science University, to unpack the groundbreaking study that made headlines around the world — creating human embryos from skin cells. Dr. Amato explains the science behind this breakthrough, known as in vitro gametogenesis (IVG), and how her team combined genetic material from skin cells and eggs to explore what could one day help people with infertility. She describes the painstaking process of somatic cell nuclear transfer, the ethical oversight behind this research, and the incredible collaboration that led to this first step forward. Together, Dr. Shahine and Dr. Amato break down complex genetics, discuss the difference between this and “three-parent IVF,” and reflect on what it all means for the future of reproductive medicine. In this episode you'll hear: [2:06] Ripped from the headlines: Human Embryos Made From Skin Cells [4:15] A refresher on chromosomes [7:55] Step-by-step explanation of the science [11:27] Introducing IVG (in vitro gametogenesis ) [15:24] IVG vs IVF [19:38] Who does this benefit? [22:20] How might it change the future? [25:37] Ethical and societal questions [28:03] Balancing progress with responsibility [33:45] What is next in fertility science? Resources mentioned: Research Study in Nature Communications OHSU Center for Embryonic Cell and Gene Therapy @paula-amato on LinkedIn Dr. Shahine's Weekly Newsletter on Fertility News and Recommendations Follow @drlorashahine Instagram | YouTube | Tiktok | Her Books
Ladies, it's Breast Cancer Awareness Month, so go get your boobies checked! We're also talking family lineage, growing up on a farm, and the groundbreaking treatment for Huntington's disease.00:00:00 - Introduction & Getting Started 00:02:04 - Fall Season & Halloween Decorations Debate 00:05:27 - Big Tea & Do Dog (Parent Nicknames) 00:15:27 - Big Family Reflections 00:20:08 - Family Genealogy & Lineage00:24:11 - Pop Culture & Science Discussion00:30:16 - Gene Therapy & Medical Research 00:33:07 - Eric Dane & ALS Awareness 00:37:11 - mRNA Vaccine Technology 00:38:36 - Wrapping Up & Superpower TalkMORE PODCAST EPISODES: https://youtube.com/playlist?list=PLTGuNbPgq2EartAwwgs_H-LVho3FvWnXpJUST LISTEN TO THE PODCAST: https://link.chtbl.com/imomsohardSEE US ON TOUR: (FALL DATES COMING SOON)https://www.imomsohard.com/WATCH OUR AMAZON PRIME SPECIAL: https://www.amazon.com/IMomSoHard-Live/dp/B07VBJ34DTIf you are interested in advertising on this podcast email ussales@acast.comTo request #IMOMSOHARD to be on your Podcast, Radio Show, or TV Show, reach out to talent@pionairepodcasting.comFOLLOW US: Facebook: https://www.facebook.com/imomsohardInstagram: https://www.instagram.com/imomsohard/Twitter: https://twitter.com/imomsohardGet our sponsor DISCOUNT CODES here!https://linktr.ee/imshpodcastABOUT US Female comedy duo Kristin Hensley and Jen Smedley have been performing, teaching, and writing comedy internationally for a combined 40+ years. They have been moms for one quarter of that time and it shows. How do they cope? They laugh about all of the craziness that comes with being a mom and they want you to laugh about it too! From snot to stretchmarks to sleepless nights, Kristin and Jen know firsthand that parenting is a hard job and they invite you to join them in taking it all a little less seriously (even if for a few short minutes a day). After all, Jen currently has four days of dry shampoo in her hair and Kristin's keys are still in her front door. They try, they fail, they support each other, and they mom as hard as they can.Disclaimer: This podcast is for entertainment purposes only. Views expressed on this podcast solely reflect those of the host and do not reflect the views of Pionaire. Hosted on Acast. See acast.com/privacy for more information.
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