Podcasts about Gene therapy

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.

Could optogenetics represent the innovative thinking needed to restore some function in retinitis pigmentosa (RP) patients? Allen Ho, MD, joins us to discuss the state of play in optogenetics, specifically regarding MCO-010 (Nanoscope Therapeutics). What have we seen in the several years since the first patients with RP were dosed with MCO-010 in the RESTORE study? And Charles Wykoff, MD, PhD, joins us to review data assessing the safety and efficacy of sura-vec (Regenxbio/AbbVie) for the treatment of non-proliferative diabetic retinopathy. What do patients look like at 2 years after a single suprachoroidal injection? Listen in to find out.  

Vidcast:  https://www.instagram.com/p/DQdnYr9jZwV/An exciting, ground-breaking clinical study now shows that children born with a defective gene coding for a vital inner ear protein can have that gene  repaired and hearing restored. This phenomenally successful preliminary clinical trial was recently published in the New England Journal of Medicine.Genetic bioengineers at New York's Regeneron Pharmaceuticals loaded a normal copy of the otoferlin gene into a dual adeno-associated virus acting as a Trojan horse.  Twelve children, born without the ability to synthesize otoferlin protein, received the gene injection, dubbed DB-OTO therapy, into their inner ears at 3 clinical centers: Harvard's Mass. Eye and Ear Infirmary, UC San Diego's Children's Hospital, and University College London.  Otoferlin is necessary for the inner ear's ability to convert sound vibrations into electrical impulses.  At 24 weeks post-injection, 9 of the 12 children, 75%, regained measurable hearing.  Three, 25%, developed near normal hearing.  The gene therapy was well-tolerated without any significant side effects.This gene therapy, with further refinement and after larger clinical trials, may be a one-and-done treatment for one common form of congenital deafness. Cochlear implants will continue to be essential therapy for other types of genetic and acquired severe hearing losses pending development of other genetic and/or chemical cochlear modifications.https://www.nejm.org/doi/full/10.1056/NEJMoa2400521#deafness #children #congenital #otoferlin #dboto 

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 ★

Most founders dream of raising millions. Few survive the 153 “no's” it takes to get there.Behind every biotech breakthrough lies exhaustion — late-night calls, failed rounds, and investors who walk away at the finish line.What separates the ones who make it isn't luck or timing — it's resilience built into process.In this episode, Jason Foster, CEO of Ori Biotech, shares how he transformed relentless rejection into a billion-dollar trajectory. From rebuilding cell-therapy manufacturing to leading global teams through economic storms, Jason reveals how founders can systematize grit, master storytelling, and survive when everything seems to fall apart.You'll learn how to navigate fundraising winters, why leadership begins with self-care, and how to build companies that endure long after the hype fades. If you've ever doubted your path as a builder, this conversation will remind you that resilience is not a trait — it's a practice.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into some of the most significant shifts and strategies shaping our industry.Novartis's acquisition of Avidity Biosciences for a staggering $12 billion marks a pivotal moment in the pharmaceutical landscape this year. With this acquisition, Novartis underscores its commitment to bolstering its neuromuscular disease pipeline. Avidity Biosciences has made a name for itself with its cutting-edge RNA therapeutic technologies, particularly its Antibody Oligonucleotide Conjugates (AOCs). This platform uniquely combines monoclonal antibodies with oligonucleotides, enhancing precision in targeting specific cell types. The integration of Avidity's technology into Novartis's research efforts could accelerate the development of new therapies, potentially transforming patient care with more effective and targeted treatment options. This move not only highlights the industry's focus on specialized therapeutic areas but also anticipates future advances in RNA therapeutics, extending beyond neuromuscular disorders to areas like oncology.In a similar vein, the FDA has shown its willingness to reconsider drugs that previously faced setbacks. GSK's Blenrep has made a return to the U.S. market after receiving approval for treating certain myeloma patients. This approval is particularly noteworthy given the drug's earlier negative advisory committee vote and postponed decision. It marks a significant rebound for GSK's oncology portfolio and reflects the FDA's dynamic approach towards drugs that show potential in specific therapeutic combinations.Meanwhile, Sanofi continues to make waves with Dupixent, achieving over €4 billion in quarterly sales due to its expanded indications. This success contrasts with a decline in Sanofi's vaccine sales, demonstrating shifting dynamics within pharmaceutical portfolios where biologics and specialty drugs are increasingly pivotal. Sanofi's recent financial report highlighted a notable 17% drop in vaccine sales due to reduced demand and pricing challenges in Europe. In response, companies must navigate fluctuating public health demands and economic pressures effectively.On the global stage, efforts to make transformative therapies like Vertex's Trikafta more accessible are gaining momentum through innovative trade-policy workarounds. A buyers club aims to introduce a lower-cost alternative produced by Bangladesh's Beximco, highlighting ongoing challenges and creative strategies in global drug accessibility.Roche's expansion through Chugai's $200 million M&A deal for an IgA nephropathy asset underscores the strategic importance of regional markets in driving growth. Similarly, Lonza's acquisition of a California biologics site aligns with its goals to meet increasing biomanufacturing demands.The industry is also adapting to technological advancements, with AI integration into life sciences commercialization being touted as a frontier for growth. Despite this potential, many organizations remain unprepared to harness AI fully. Leading companies embedding AI solutions aim for measurable outcomes that could significantly drive strategic decision-making and operational efficiencies.Eli Lilly's acquisition of Adverum Biotechnologies aligns with its strategic interests in gene therapy, focusing on promising therapeutic programs that address unmet medical needs. This acquisition centers around Ixo-vec for wet age-related macular degeneration (AMD), highlighting broader industry trends towards investing heavily in innovative therapies that address unmet needs.Conversely, Sanofi's halt on an RSV vaccine development highlights the inherent risks in vaccine development pipelines. Meanwhile, Regeneron's decision to discontinue a CAR T candidate acquired from 2seventy bio showcases ongoing reassessment witSupport the show

In this episode, we speak with Associate Professor Leszek Lisowski, Head of the Translational Vectorology Unit at the Children's Medical Research Institute (CMRI), about the cutting-edge world of gene therapy. He explains how viral vectors are engineered to deliver life-saving treatments for rare genetic diseases - many of which are so uncommon, they're only known by the name of the affected gene. Despite being often overlooked, genetic diseases impact 10% of adults and 30% of children in hospitals. Leszek sheds light on the challenges of diagnosis, the promise of emerging genetic testing, and how his work is paving the way for more accurate, effective therapies. Linkedin: Children's Medical Research Institute, Leszek Lisowski Facebook: @jeansforgenes Instagram: @jeansforgenesau

The journey to a healthier body can sometimes be a struggle. Some foods help, some don't. Certain exercises are supposed to be good but don't show everyone the same rewards. That's where our genes could unlock the path to personalized health.Kyler Ockey shares some of the breakthroughs that Shed is working on to help people individualize and personalize their health journey. Turns out our genes play an important role in how food, exercise, and our environment affect our physical body. Without knowing how to optimize our genes, we could be doing "everything right" and not get the results we want. Kyler Ockey is the Chief Strategy & Product Officer at Shed, a leader in science-backed wellness solutions. Shed created Genetic Testing Kits, designed to help individuals understand how their bodies respond to nutrition, fitness, sleep, stress, and more — and make choices that work with their biology, not against it. Available in four targeted options — Total Wellness, Nutrition + Fitness, Nutrition, and Fitness — the kits provide personalized insights and actionable recommendations for fueling smarter, training effectively, and supporting long-term health. Follow Shed on Instagram @tryshed Visit ConfidenceThroughHealth.com to find discounts to some of our favorite products.Follow me via All In Health and Wellness on Facebook or Instagram.Find my books on Amazon: No More Sugar Coating: Finding Your Happiness in a Crowded World and Confidence Through Health: Live the Healthy Lifestyle God DesignedProduction credit: Social Media Cowboys

In this week's episode of the Xtalks Life Science Podcast, host Ayesha Rashid, Senior Life Science Journalist at Xtalks.com, spoke with R. Nolan Townsend, MBA, CEO of Lexeo Therapeutics, a company developing gene therapies for cardiovascular diseases as well as CNS disorders including Alzheimer's disease. Lexeo recently announced a partnership to advance cardiac RNA therapeutics as well as $80 million in equity financing. Mr. Townsend has been CEO of Lexeo Therapeutics since 2020. He previously held senior leadership roles at Pfizer, including President of Rare Disease for both North America and International markets, and began his career in healthcare investment banking at Lehman Brothers. He currently also serves on several boards, including Arbor Biotechnologies, the Biotechnology Innovation Organization (BIO) and the Martha's Vineyard Museum. He is also a member of the New York City Economic Development Corporation's Life Sciences Advisory Council. Mr. Townsend received his MBA from the Harvard Business School and his Bachelor of Arts in Economics from the University of Pennsylvania. Tune in to hear how Lexeo is shaping the future of genetic medicines under Mr. Townsend's leadership. For more life science and medical device content, visit the Xtalks Vitals homepage. https://xtalks.com/vitals/ Follow Us on Social Media Twitter: https://twitter.com/Xtalks Instagram: https://www.instagram.com/xtalks/ Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

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

Genetic counsellors Matt and Sandra explore Huntington disease, prenatal and lab-based testing ethics, and how lab genetic counsellors act as gatekeepers. They discuss exclusion testing, the challenges of writing clear lab reports, and recent advances in gene therapy. The episode also covers the growing role of AI and automation in genetic labs, the evolving responsibilities of lab GCs, and how testing impacts life choices like career planning and family decisions.

We're live at BDC!  Host Amy Board take you straight to the exhibit hall floor, chatting with doctors like Drs. Quon, Sidonio, and Radak, plus patient advocates Collin Johnson and Matt Capsel. From man-on-the-street interviews to a touching story from Greg, this episode reminds us all to run—not walk—towards science. Join us for real conversations, patient perspectives, and insights on gene therapy, joint health, and life in the bleeding disorders community. Next up: An inspiring interview with Patty Weltin and the “Beyond the Diagnosis” art exhibit.   Don't miss this insightful, energizing episode that dives into the future of bleeding disorders care.   Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   It's a Whole New World Gene Therapy Segment brought to you by CSL Behring, which now has a first-of-its-kind hemophilia B treatment. Visit BeyondHemB.com or download B SUPPORT wherever you get your apps for more information. Show Notes:   Subscribe: The BloodStream Podcast   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter  BloodStream on Instagram BloodStream on LinkedIn BloodStream on TikTok  

Program notes:0:40 New drug for SLE1:41 Monitored for a year2:43 Only one pathway limited3:00 AI and health care4:00 How to assure compliance?5:00 Policy changes needed6:01 Pessimistic about oversight6:50 Genetic treatment for ADA deficiency7:50 Using a viral vector8:50 A curative treatment9:30 Social media and cognitive performance in adolescents10:30 Low and high increasing social media use11:30 Good enough evidence12:31 End

From CAR-T therapies to viral vectors, cell and gene treatments are redefining the boundaries of pharmacy practice—but with innovation comes complexity. Host Carolyn Liptak welcomes Dr. Mark Wiencek, Principal Microbiologist with the Technical Services Group at Contec, and Dr. Amanda Frick, Senior Clinical Manager of Market Intelligence at Vizient, to break down the challenges of compounding these advanced therapies.   Listen in as they discuss real-world risk assessments, biosafety considerations, and how hospital pharmacies can safely manage these groundbreaking yet high-risk treatments.   Guest speakers:  Mark Wiencek, PhD Principal Microbiologist, Technical Services Group Contec Amanda Frick, PharmD, BCPS Senior Clinical Manager, Market Intelligence Vizient   Host:  Carolyn Liptak, MBA, RPh  Pharmacy Executive Director  Vizient   Show Notes:  [01:02-01:51] Mark shares his background and experience in microbiology [01:52-04:04] Overview of the types of cell and gene therapies (CGT) currently used in clinical practice [04:05-05:14] Which CGT therapies are most applicable to pharmacy compounding and why [05:15-10:29] Things not on the NIOSH list and the risks [10:30-12:03] Evaluating whether viral vectors can penetrate intact skin and the true occupational exposure risks [12:04-13:18] If hazards are not defined by the NIOSH list, how should these CGT hazards be classified [13:19-15:03] Determining the safest environment for compounding CGT therapies [15:04-20:14] Best practices for decontamination, disinfection, and viral vector handling [20:15-20:59] Do you need a dedicated biosafety cabinet for CGT therapies [21:00-22:55] Recommended resources for further learning   Links | Resources:   Blind and colleagues (Nationwide): Click here Wang and colleagues (Stanford): Click here CONTEC HEALTHCARE WEBINAR Using Bugs as Drugs: Compounding Viral Vectors in Cell & Gene Therapy for Hospital Pharmacies, Mark Wiencek, May 13, 2025: Click here Blind, J.E., Ghosh, S., Niese, T.D., Gardner, J.C., Stack-Simone, S., Dean, A. and Washam, M., 2024. A comprehensive literature scoping review of infection prevention and control methods for viral-mediated gene therapies. Antimicrobial Stewardship & Healthcare Epidemiology, 4(1), p.e15. Click here Deramoudt, L., Pinturaud, M., Bouquet, P., Goffard, A., Simon, N. and Odou, P., 2024. Method for the detection and quantification of viral contamination during the preparation of gene therapy drugs in a hospital pharmacy. Occupational and Environmental Medicine, 81(12), pp.615-621. Click here Korte, J., Mienert, J., Hennigs, J.K. and Körbelin, J., 2021. Inactivation of adeno-associated viral vectors by oxidant-based disinfectants. Human Gene Therapy, 32(13-14), pp.771-781. Click here (abstract only; full article available for purchase) Martino, J.G., McConnell, K., Greathouse, L., Rosario, B.D. and Jaskowiak, J.M., 2024. Cellular therapy site-preparedness: Inpatient pharmacy implementation at a large academic medical center. Journal of Oncology Pharmacy Practice, 30(8), pp.1442-1449. Click here Penzien, C., 2023. Safe handling of BioSafety drugs and live virus vaccines. Pharm Purch Prod, 20(4), p.12. Click here Petrich, J., Marchese, D., Jenkins, C., Storey, M. and Blind, J., 2020. Gene replacement therapy: a primer for the health-system pharmacist. Journal of Pharmacy Practice, 33(6), pp.846-855. Click here Wang, A., Ngo, Z., Yu, S.J. and MacDonald, E.A., 2025. Implementing standard practices in the safe handling of gene therapy and biohazardous drugs in a health-system setting. American Journal of Health-System Pharmacy, p.zxaf026. Click here   VerifiedRx Listener Feedback Survey: We would love to hear from you - Please click here   Subscribe Today! Apple Podcasts Spotify YouTube RSS Feed

When baby KJ was diagnosed with a rare metabolic disorder, the team at Children's Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ's first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. 

his Week in Review covers 5 episodes from October 06 to October 10, featuring major developments in the pharmaceutical and biotech industries.Episodes included:1. Pharma and Biotech Daily: Novo's Acquisition, FDA Breakthroughs, and Industry Updates2. Pharma and Biotech Daily: Top Stories in the Industry from Zenas to Lilly3. Pharma and Biotech Daily: Tariff Impact, Promising Trials, and Industry Leaders4. Pharma and Biotech Daily: The Latest in Cell and Gene Therapy, M&A Activity, and Regulatory Updates5. The Essential Updates in Pharma and Biotech: Your Daily Dose of What MattersKey topics covered:- Strategic acquisitions and partnerships- Regulatory updates and FDA approvals- Clinical trial results and breakthroughs- Industry trends and market developmentsStay informed with Pharma Daily's comprehensive coverage of the pharmaceutical and biotech worldSupport the show

What if solving tomorrow's bioprocessing challenges meant questioning the very physics of chromatography and reimagining downstream processing from the ground up?For years, large biomolecules like viral vectors and exosomes have squeezed through the limitations of traditional chromatography, leaving scientists to wrestle with capacity trade-offs and slow mass transfer. But what if a fundamental shift could unlock breakthroughs for gene therapies, vaccines, and advanced biologics?In this thought-provoking episode, host David Brühlmann sits down with Alois Jungbauer, professor emeritus of downstream processing at BOKU University (Vienna) and scientific advisor at BioChromatographix International. Together, they examine the future of chromatography and downstream processing, exploring innovations that tackle the challenges of modern gene therapy, advanced therapeutics, and sustainability in manufacturing.Alois shares his perspective on anticipating industry needs, the importance of mentorship, and why the physical limits for purification of large biomolecules are yet to be reached.Episode Highlights:The difference between solving current problems and anticipating the needs of the next generation in biotech (00:00)The promise and technical details of monolithic chromatography and its application for large modalities like gene therapy vectors and exosomes (03:01)Explanation of “inverted morphology" and AXISFLOW™ in new chromatography materials (03:18)Challenges and opportunities in continuous gene therapy vector production, and why it's not widespread yet (07:40)The critical role of sustainability in downstream processing, particularly water usage and its impact on the viability and costs of distributed manufacturing (10:30)The relationship between reducing water consumption, manufacturing footprint, and cost of goods (12:16)Advice on scientific career development: listening, reading, understanding future industry needs, finding a mentor, and having persistence (13:50)Broader reflections on the value of science, societal perceptions, and science communication in today's world (17:34)Alois's key takeaway: downstream processing for gene therapy has not yet reached its physical limits—there is still significant room for improvement (20:26)If you're looking for fresh strategies to tackle the bottlenecks of large-molecule purification - and a candid perspective on where the real opportunities and societal responsibilities lie - you won't want to miss this conversation.Connect with Alois Jungbauer at leading conferences, including Bioprocessing Summit and Bioprocessing International, or find the next integrated continuous biomanufacturing event where he's co-chairing.Connect with Alois Jungbauer:LinkedIn: www.linkedin.com/in/alois-jungbauer-14984811Website : www.biochromatographix.comNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at

As an audiologist, what do you say when a patient or client asks to receive gene therapy? The headlines are certainly grabbing public interest, but what does it mean for your practice?Clinical trials and media coverage are driving interest in this novel approach to hearing loss intervention. As our guests share, so far only one gene has received clinical trials. But patients and their families may seek this treatment or ask you questions about what it means for them.To provide background and perspective, we'll hear from two people well versed in the world of gene therapy for hearing loss: Ryan McCreery of Boys Town National Research Hospital and Jenna Devare of University of Michigan Medical School.Learn More:ASHA Voices: A Promising Outlook for Gene Therapy and Hearing LossFirst Deaf Gene Therapy Recipient in U.S. Gains HearingASHA Voices: The Limits of Our GenesASHA Voices: Audiologists Address Ototoxicity in Cancer CareTranscript

Somministrare terapie geniche direttamente in utero, in modo da poter correggere, già durante la gravidanza, patologie genetiche devastanti, come le malattie mitocondriali, che fanno danni irreparabili prima ancora che il bambino nasca. Si parla in questi casi di Terapia Genica Fetale In Utero (IUFGT) e in uno studio coordinato dall’Università degli Studi di Milano e dalla Fondazione IRCCS Istituto Neurologico Carlo Besta, in collaborazione con il Policlinico di Milano e Avantea di Cremona, un gruppo di ricercatori ha descritto una procedura sperimentale per la somministrazione di questo tipo di cura, basata sull’iniezione ecoguidata transaddominale: una tecnica comprovata e sicura, già usata per l’amniocentesi e quindi ben sperimentata nella pratica clinica. I risultati dello studio, ottenuti al momento sui suini, sono pubblicati sulla rivista Gene Therapy del gruppo Nature e sono alquanto incoraggianti. Ne parliamo con Dario Brunetti, coordinatore dello studio, docente del Dipartimento di Scienze Cliniche e di Comunità dell’Università Statale di Milano e primo ricercatore presso l’Istituto Neurologico Carlo Besta.

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.

In this episode, host David Brühlmann is joined by Alois Jungbauer, Professor Emeritus at BOKU University, Vienna, and Scientific Advisor at BioChromatographix International. With over 40 years in bioprocess engineering and more than 400 published papers, Jungbauer offers a unique perspective on how downstream processing and purification technologies have evolved and where they're headed next.Alois shares his unconventional journey into the world of biotech, starting from humble beginnings on an Austrian farm, and discusses key turning points that have shaped the industry. Together, they explore the latest challenges triggered by high cell culture titers, new therapeutic modalities, and the drive for automation and process integration in manufacturing.In this episode:Why the gap between upstream and downstream processes remains a core bottleneck (03:27)How rigid and convective chromatography materials changed throughput and efficiency (06:56)The shift in purification strategies with skyrocketing titers in cell culture (08:43)Automation and integrated biomanufacturing: their role in making processes more robust and continuous (10:20)Misconceptions around resin costs in full-scale versus clinical-scale manufacturing (12:10)What automation and process monitoring mean for real-time release and factory scheduling (14:05)The resurgence and application of displacement chromatography for gene therapy vectors (15:49)Whether you're deep into process development or just curious about the next wave in biotech manufacturing, Alois Jungbauer's insights offer valuable context for the road ahead.Connect with Alois Jungbauer:LinkedIn: www.linkedin.com/in/alois-jungbauer-14984811Website : www.biochromatographix.comNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at https://scale-your-impact.notion.site/27dd9c6ba679804b80a7ce439d56c91a?pvs=105

Guest: Dr Lauren Byrne, Principal Investigator at the Huntington's Disease Centre at UCL

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Chiesi has entered a $2.1 billion collaboration with Arbor to develop a gene therapy for primary hyperoxaluria type 1. AstraZeneca continues to invest in AI partnerships, FDA lifts clinical hold on Neurizon's ALS drug, and the cell and gene therapy field faces challenges and opportunities at the Meeting on the Mesa event. CDC adopts ACIP recommendations, FDA's manufacturing initiatives progress, and Mythic cuts staff.Industry leaders in the cell and gene therapy space are gathering in Phoenix for a meeting on the mesa, where the market is experiencing mixed signals. While M&A activity and regulatory support are showing momentum, challenges with commercialization are still hindering investor interest. The focus on next-generation multiple sclerosis treatments beyond BTK inhibitors is gaining attention, with companies like Immunic Therapeutics exploring neuroprotective targets and remyelination.The future of gene therapy depends on improving safety through targeted delivery and human-relevant testing to avoid costly failures. The FDA has released new guidance to streamline regulatory pathways for cell and gene therapies for small populations, which is receiving a warmer welcome compared to previous issuances. In other news, the US government shut down, leading to the FDA closing for new applications, while Amgen presents late-stage data for Repatha in preventing heart disease.Additionally, Metsera's obesity drug shows promising efficacy in mid-stage trials. Cybersecurity is highlighted as a crucial challenge in the pharmaceutical industry, with a focus on protecting manufacturing integrity and innovation. Upcoming events include webinars on mRNA medicine and biopharma deregulation, while job opportunities in various biopharma companies are also available.Support the show

Drs. Marianeli Rodriguez and Kat Talcott join to discuss the October 2025 edition of Retinal Physician, including inherited retinal disease treatment pipeline, gene therapy, and systemic therapy for sickle cell disease and retinopathy.

Editor's Summary by Linda Brubaker, MD, and Christopher C. Muth, MD, Deputy Editors of JAMA, the Journal of the American Medical Association, for articles published from September 27-October 3, 2025.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "ECTRIMS 2025 Meeting Highlights and Clinical Takeaways," Daniel Ontaneda, MD, PhD, neurologist at Cleveland Clinic's Mellen Center for MS, shared his reflections from the 2025 ECTRIMS Congress, held September 24-26, in Barcelona, Spain. He discussed the significance of the updated MS diagnostic criteria, which generated immediate research applications and clinician discussion early in the meeting. Ontaneda also highlighted the growing emphasis on precision medicine and individualized treatment approaches, including extended-interval dosing strategies for B-cell therapies. In addition, he reviewed new therapeutic developments such as BTK inhibitors, CAR-T therapies, and remyelination research, noting both promising and disappointing data. Finally, he spoke on how ECTRIMS continues to expand beyond MS, with more presentations dedicated to NMOSD, MOGAD, and other autoimmune neurological conditions, reflecting the evolving landscape of neuroimmunology. Looking for more Multiple Sclerosis discussion? Check out the NeurologyLive® Multiple Sclerosis clinical focus page. Episode Breakdown: 1:00 – Overall impressions of ECTRIMS 2025, highlighting diagnostic updates, precision medicine, and late-breaking trial results 4:10 – Expanding focus on individualized care, especially interval-adjusted dosing strategies for B-cell therapies 7:50 – Neurology News Minute 9:50 – Insights on emerging therapeutic approaches including BTK inhibitors, CAR-T therapies, and remyelination strategies 14:35 – Growing attention toward NMOSD, MOGAD, and other autoimmune conditions within neuroimmunology discussions The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EMA Approves Semaglutide as First GLP-1 RA for Cardiovascular, Stroke-Related Benefits Del-Zota Reverses Duchenne Disease Progression in 1-Year Trial Update MDA and PPMD Release Consensus Guidelines for Safe and Equitable Use of Gene Therapy in Duchenne Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

We review the latest results from the Regenexbio sub retinal gene therapy program with Robert Avery MD, California Retina Consultants, Santa Barbara, CA

What if the future of affordable, life-changing cell and gene therapies comes down to one critical yet often overlooked factor: manufacturing efficiency?In advanced therapy medicinal products (ATMPs), groundbreaking science abounds, but the path to the clinic is still strewn with bottlenecks, especially when it comes to cost, complexity, and safety. While the promise is enormous, most therapies remain out of reach for many patients due to high cost of goods and logistical hurdles. What can actually break down these barriers and democratize access?In this episode, David Brühlmann welcomes Lucas Chan, celebrated Fellow of the Royal Society of Biology, advisor to Singapore's Ministry of Health, and a true pioneer in viral vector manufacturing. In Singapore, he founded CellVec Pte Ltd – APAC's first regulatory accredited Viral Vector specialist CDMO.Lucas's leadership spans founding ventures in emerging markets to championing game-changing innovations in gene transfer, always with an eye toward efficiency, safety, and accessibility. Having returned “back to the bench” in collaboration with the National University of Singapore while launching his consultancy, Lucas brings both visionary perspective and hands-on experience to one of biotech's toughest challenges.Here are three reasons why you need to listen to this episode:Efficiency is Everything: Lucas dissects how complex processes and outdated manufacturing paradigms are driving up the cost of cell and gene therapies and shares real-world advances, from transitioning viral vector production to stable producer cell lines to the emergence of non-viral gene transfer modalities, that are poised to rewrite the rulebook on scalability and affordability.Leadership Amid Complexity: Learn from Lucas's top leadership advice drawn from his CSO and CDMO tenure: inspire teams by connecting every task to the larger mission. In a multidisciplinary, high-stakes environment, alignment and motivation aren't just nice, they're essential for innovation.Entrepreneurship and Community: Thinking of turning your biotech expertise into a startup? Lucas underscores the value of learning from others' journeys, collaboration, and the insight that “it takes a village” when translating advanced therapies from bench to bedside, especially in emerging markets with unique affordability challenges.Want to transform your approach to cell and gene therapy manufacturing or just need inspiration to push your biotech project to the next level?Tune into this episode for practical insights, candid leadership lessons, and a renewed sense of what's possible when we challenge the status quo together.Connect with Lucas Chan:LinkedIn: www.linkedin.com/in/lucaschangtNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at https://scale-your-impact.notion.site/27dd9c6ba679804b80a7ce439d56c91a?pvs=105

Sickle cell disease was once considered an incurable disease, until recently. A wide variety of gene therapies have now become available, allowing patients with sickle cell disease to now have futures once thought unimaginable. In this podcast episode, we talk through these treatments and get expert advice on what these treatments mean for our future. 

Guest: David Jacobsohn Gene therapy is redefining the treatment landscape for sickle cell disease, with approaches like gene addition, gene editing, and fetal hemoglobin induction showing promise in reducing or even eliminating symptoms. However, while these therapies offer some advantages over allogeneic stem cell transplant, they also come with significant challenges. In a conversation with Ryan Quigley, Dr. David Jacobsohn discusses considerations for these therapeutic approaches and their potential impacts on care plans for sickle cell disease patients. Dr. Jacobsohn is the Division Chief of the Blood and Marrow Transplantation Program at Children's National Hospital and Full Professor of Pediatrics at the George Washington University in Washington, DC.

Durable vision improvements were also observed out to 18 months for previously dosed adult patients in the trial.

Viral vectors are the backbone of cutting-edge cell and gene therapies, delivering life-altering treatments to patients with genetic diseases. But making these biological delivery vehicles is a high-wire act: unpredictable, complex, and fraught with hurdles that traditional biologics manufacturers have never faced.While the world races toward next-generation therapies, the challenge of producing viral vectors at clinical and commercial scale threatens to leave patients waiting on the sidelines.Our guest on the Smart Biotech Scientist Podcast is Lucas Chan, a molecular and cell biologist with over two decades of hands-on experience in cell and gene therapy development. From his formative research at Imperial College London, where he pioneered novel viral vector cell lines, to building the UK's first GMP clinical viral vector core and later founding CellVec Pte Ltd Asia Pacific CDMO in Singapore, Lucas is the go-to architect for manufacturing viral vectors where infrastructure barely exists.Along the way, he's faced and overcome challenges few others could imagine.Here are three reasons this episode is a must-listen:Viral Vectors Demystified: Lucas breaks down a common misconception: viral vectors aren't malignant viruses, but genetically disarmed delivery vehicles that can't revert or replicate, making them safe tools for gene therapy.Manufacturing Mayhem Made Manageable: Unlike monoclonal antibodies and other biologics, viral vectors (and other cell and gene therapies) are made in living cells, leading to a level of process unpredictability and complexity where “everything, everywhere, all at once” applies. Success hinges on scientific acumen and attention to even the smallest detail, from raw materials to equipment parameters, to ensure patient safety and product quality.Pioneering in Uncharted Territory: Moving from London to Singapore, Lucas faced not just technical but regulatory and logistical challenges to founding Asia-Pacific's first CDMO dedicated to viral vectors. His advice: engage regulators early, leverage local biomanufacturing strengths, and build multidisciplinary teams to transfer and scale bioprocess know-how.Curious how viral vector manufacturing can be reimagined to unlock the full promise of cell and gene therapies? Listen to this episode and gain unique insights into the science, strategy, and sheer determination needed to bring advanced therapies out of the lab and into the clinic.Connect with Lucas Chan:LinkedIn: www.linkedin.com/in/lucaschangtNext step:Book a 20-minute call to help you get started on any questions you may have about bioprocessing analytics: https://bruehlmann-consulting.com/callPreparing for your IND? We're building a CMC Dashboard in Excel to help biotech founders track tasks, timelines, and risks in one place. Join the waitlist for early access at https://scale-your-impact.notion.site/27dd9c6ba679804b80a7ce439d56c91a?pvs=105

On "This Week in Pharmacy" for September 26th, we're talking about Trust in Healthcare, World Pharmacists Day, and Cell & Gene Therapy. Today's co-host is Dr. Melissa Smith, PharmD CPH with Florida PGx Consulting LLC. https://www.floridapgxconsulting.com/  First, #TWIRx News - the FDA approves Inluriyo imlunestrant on 9/25/2025 which is to treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, estrogen receptor-1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. (https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025)  Second, Nested-Knowledge - is going to revolutionize information access and leverage. This is a powerful tool with evidence synthesis tools for medical researchers. This will help accelerate, collaborate, automate and share the latest and personalized treatment plans. (https://nested-knowledge.com/) Next, announcing "Evidence based Podcasting", the new standard in audio learning for Providers and next level in podcast Continuing Medical Education (CME). (https://pharmacypodcast.com/2025/09/26/evidence-based-podcasting-and-the-future-of-medical-education/ ) Shout out to Nick Calla with Orsini, a leader in rare disease pharmacy solutions, has been chosen by Crinetics Pharmaceuticals, Inc., as a specialty pharmacy partner for PALSONIFY™ (paltusotine), a nonpeptide, selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist. (https://www.wvnews.com/news/around_the_web/partners/pr_newswire/subject/licensing_marketing_agreements/orsini-selected-as-specialty-pharmacy-partner-for-crinetics-palsonify-paltusotine/article_9b42bfe7-69a6-58be-98ff-5e2245145415.html) Today we have two guest Pharmacists to dig into the "Trust in Healthcare" theme of today's #TWIRx -- the godfather of Functional Pharmacy Robert Kress RPh., and Dr. Lauren Castle, PharmD, MS, AFMC - Founder & CEO of Functional Medicine Pharmacists Alliance. Who do you trust? What information is trustworthy and why?  Our special feature is updates on 'Cell & Gene Therapy' with McKesson's Head of Cell Gene Advanced Therapies, Joe DePinto at InspiroGene!  (https://inspirogene.com/)  Today's TWIRx is sponsored by 'Sykes & Company' the Pharmacy Accounting Jedi Masters and Independent Pharmacy Cooperative (IPC) and how they're revolutionzing digital health and new revenues with the iCare+ System!    Trust Matters, Cell and Gene, & World Pharmacists Day | TWIRx

Here us talk about what happened this day in history and we re-air a great conversation about Gene Therapy. We're joined by local nihilistic surf-rock band Dollar Eater and feature the track "Trash Paradise" off their new album entitled "Try Hardest".

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Uniqure's gene therapy for Huntington's disease, AMT-130, has shown promising results in a 3-year study, slowing disease progression by 75%. If approved, it would be the first genetic treatment for Huntington's. Meanwhile, Acadia Pharmaceuticals has discontinued a Prader-Willi drug after a late-stage failure. Biogen received an unexpected FDA rejection for a high dose of Spinraza, and J&J received a warning letter for violations at a Korean production plant. In the competitive bioscience industry, credentials from Biotility can help advance careers. Sanofi is investing in rare disease and neuro innovation, while Lilly is expanding manufacturing facilities. Scholar Rock faced setbacks with its spinal muscular atrophy drug.Chinese biotech has rapidly risen to become a global powerhouse, with companies making significant strides in oncology and other areas. However, despite this success, Chinese biotechs are facing financial challenges similar to those in the US. The influx of interest from big pharma has been a lifeline for these companies as they strive to prove themselves on the global stage. Akeso, a rising star in Chinese biotech, has developed potential blockbuster drugs and is valued highly in the market. However, President Trump's potential executive order restricting drug licensing deals in China could pose a threat to the progress made by Chinese biotechs. Despite these challenges, Chinese companies continue to perform well, with significant investments and partnerships driving growth in the industry. Pfizer's CEO emphasized the need for the US to focus on improving and competing with China rather than trying to slow them down. The future of Chinese biotech remains uncertain in the face of geopolitical tensions, but the industry continues to innovate and attract investment from pharmaceutical companies worldwide.

In this episode of IDEA Collider, we are joined by Alex Telford, a biotech founder, writer, and thinker based in the San Francisco Bay Area. As the co-founder of Convoke Bio, Alex discusses his journey from studying biochemistry at UCL to founding a company that develops software tools for streamlining biopharmaceutical workflows.The conversation dives into the impact of AI and language models on the biotech industry, addressing inefficiencies in drug development, and exploring the potential of synthetic biology and personalized medicine. The discussion also touches on the future of drug discovery, China's role in biotech, and the challenges of understanding neuroscience and consciousness. Don't miss this insightful discussion on the future of biopharma innovation! 00:00 Welcome to Idea Collider00:04 Introducing Alex Telford01:59 Alex's Journey from UCL to Convoke Bio05:29 The Mission and Work of Convoke Bio07:57 Challenges in Pharma Decision Making14:05 The Role of AI in Pharma18:26 Knowledge Management and AI27:58 Staying Updated in the Fast-Moving AI Field30:25 AI's Impact on Industry Economics31:38 AI in Clinical Trials and Drug Development35:56 China's Role in Drug Discovery39:24 Neuroscience and AI: Blurring the Lines46:05 Future Predictions in Pharma51:20 Addressing Cognitive Bias in Pharma53:13 Concluding Thoughts and Future Directions Keep up with Alex Telford;LinkedIn: https://www.linkedin.com/in/alexander-telford/Website: atelfo.github.ioX: https://twitter.com/atelfoSubstack: atelfo.substack.com Follow Mike Rea On;Website: https://www.ideapharma.com/X: https://x.com/ideapharmaLinkedIn: https://www.linkedin.com/in/bigidea/ Listen to more fantastic podcast episodes: https://podcast.ideapharma.com/

In this episode, Dr. Bill Andrews joins Dr. Buck Joffrey to discuss the critical role of telomeres and telomerase in aging and cellular health. He explains how telomeres shorten with each cell division, leading to aging and cellular senescence. Dr. Andrews emphasizes the importance of inducing telomerase to potentially reverse aging and shares insights from his research on telomerase inducers. He also addresses the challenges in longevity research, the relationship between telomeres and the hallmarks of aging, and the potential of gene therapy. The conversation concludes with a discussion on lifestyle factors that can influence aging and the future of aging research. Learn more about Dr. Bill Andrews: https://sierrasci.com/dr-bill-andrews/ - Download Dr. Buck Joffrey's FREE ebook, Living Longer for Busy People: https://ru01tne2.pages.infusionsoft.net/?affiliate=0 Book a FREE longevity coaching consultation with Dr. Buck Joffrey: https://coaching.longevityroadmap.com/

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Rethinking Parkinson's Care: Prevention, Equity, and Access to Treatment," Michael Okun, MD, neurologist, author of The Parkinson Plan, and medical advisor for the Parkinson's Foundation, discusses the urgent need for global access to levodopa, the gold-standard treatment for Parkinson disease. He outlines the consequences of delayed or unavailable treatment worldwide, including worsening outcomes and unnecessary disability. Okun highlights lessons from global HIV drug distribution that can inform equitable levodopa access, emphasizing the importance of tailoring strategies to local economies and healthcare systems. Additionally, he breaks down key components of The Parkinson Plan, including prevention, research priorities, amplifying patient and caregiver voices, and rethinking care models to better address the world's fastest growing neurodegenerative disorder. Finally, he explains how the book can serve as a resource not only for clinicians, but also for patients, families, and policymakers. Looking for more Movement disorders discussion? Check out the NeurologyLive® Movement disorders clinical focus page. Episode Breakdown: 1:00 – Consequences of delayed or unavailable levodopa treatment for patients worldwide 2:40 – How universal levodopa access could change care in low-resource settings 5:15 – Lessons from global HIV drug distribution for equitable levodopa delivery 7:00 – Neurology News Network 8:35 – Key pillars of The Parkinson Plan and rethinking Parkinson's care models The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EMA Approves Semaglutide as First GLP-1 RA for Cardiovascular, Stroke-Related Benefits Del-Zota Reverses Duchenne Disease Progression in 1-Year Trial Update MDA and PPMD Release Consensus Guidelines for Safe and Equitable Use of Gene Therapy in Duchenne Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Amanda Bergen, Director of Communications at the Phelan-McDermid Syndrome Foundation, steps in for Dr. Lauren Schmitt to host a special conversation with Dr. Dan Gallo, Kate Neer, and Gina Newton from Jaguar Gene Therapy. Together, they explore the origins and mission of Jaguar Gene Therapy, provide an overview of the Phelan-McDermid syndrome natural history study (NHS) and why it matters, describe key insights into their gene therapy program, and discuss details about JAG201. Check out the link to learn more about the NHS: https://pmsf.org/current-open-research/Check out our previous webinar on the NHS: https://youtu.be/pOzTf3qnNmk?feature=sharedCheck out the updated FAQs for further information about the ongoing JAG201 gene therapy trial: https://drive.google.com/file/d/1QUfuSUQQ9ozOI2eIloRPbmJJw929l-wO/view?usp=sharingLearn more about Jaguar Gene Therapy: https://jaguargenetherapy.com/

A treatment for cancer now has applications for patients with sickle cell disease. Scientists genetically modify the patient's own blood cells—transforming them into healthy ones—and then infuse them back into the body. Listen to the inspiring journey of a patient undergoing this life-changing therapy at The University of Kansas Cancer Center for a pain-free future.

With Pfizer discontinuing Beqvez and BioMarin scaling back the commercial focus of Roctavian, the curative promise of hemophilia gene therapies is tempered by significant barriers that discourage widespread adoption. In this week’s episode of “The Top Line,” Fierce Pharma’s Zoey Becker speaks with Glenn Pierce, M.D., Ph.D., vice president of medical at the World Federation of Hemophilia, about the complexities behind these innovative therapies and the multifaceted reasons for their slow uptake. Pierce discusses the competitiveness of the current hemophilia gene therapy market, why Pfizer’s product couldn’t keep up, and the patient populations that risk being left behind. To learn more about the topics in this episode: As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio BioMarin downsizes Roctavian efforts but keeps hemophilia gene therapy for 3 markets See omnystudio.com/listener for privacy information.

It seems there are news stories every week about the accelerating pace of innovation in gene therapy, but only about 50 therapies have been approved so far by the US Food and Drug Administration. Our guest today, Dr. Bobby Gaspar, leads a UK-based biotech company, Orchard Therapeutics, that developed one of those treatments using gene-modified stem cells in your blood that self-renew, so a single administration can give you potentially a lifelong effect. “Our approach is about correcting those hematopoietic stem cells and allowing them to give rise to cells that can then correct the disease,” explains Dr. Gaspar.  The therapy in focus is lenmeldy, the first approved treatment for metachromatic leukodystrophy, also known as MLD, a devastating inherited disorder that affects roughly 600 children worldwide. But Dr. Gaspar is optimistic that learnings from Orchard's work on MLD could be useful in treating much more common disorders including frontotemporal dementia, Crohn's disease and others. This highly informative conversation with host Lindsey Smith also explores the importance of newborn screening, community collaboration in advancing clinical trials for rare diseases, and a future in which each gene therapy will be used as a tool for specific applications.  “There will be many gene therapies available, some of which will become the standard of care for certain diseases, but it won't be for every disease.”Mentioned in this episode:Orchard Therapeutics If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

In this interview, we sit down with John Tobin, Vice President of Clinical Operations at Splice Bio, to explore groundbreaking gene therapy research for Stargardt's disease and its potential impact on the blind and visually impaired community. John shares insights into Splice Bio's mission, how their innovative work could transform options for people living with vision loss, and what families, professionals, and supporters should know about emerging treatments. Stay tuned until the end for information on how to get involved in Splice Bio's clinical trials. Learn more here: www.splice.bio/clinical Or send an email to: Clinicaltrials@splice.bio #SpliceBio #genetherapy #clinicaltrials

This month, as the eLife Podcast hits its century, we hear how getting frog dads to cross-foster tadpoles has revealed the way in which some frogs come by their microbiomes, the ants that do gene therapy, signs that disease causes a breakdown in nutrient exchange between the elements of the microbiome, how fungi reprogram immune cells to cause over-reactions in sepsis, and new insights into how tapeworm larvae in the brain cause seizures... Get the references and the transcripts for this programme from the Naked Scientists website

In this episode of the Gladden Longevity Podcast, Dr. Jeffrey Gladden and Steve Reiter explore various aspects of longevity, including organ age testing, the pace of aging, multi-drug gerotherapy, the role of GLP-1s, gene therapy, and the importance of lifestyle choices in optimizing health and longevity. They discuss innovative testing methods to assess organ health, the implications of aging rates, and the potential of new therapies to enhance longevity. The conversation emphasizes the significance of understanding individual health metrics and making informed lifestyle choices to achieve optimal health outcomes.   For Audience ·       Use code 'Podcast10' to get 10% OFF on any of our supplements at https://gladdenlongevityshop.com/ !    Takeaways ·       Every organ has its own age, impacting overall health. ·       Understanding organ age can help tailor longevity strategies. ·       New tests can predict health risks before they manifest. ·       Multi-drug gerotherapy offers a new approach to aging. ·       GLP-1s have benefits beyond weight loss, including heart health. ·       Gene therapy may offer new avenues for reversing aging. ·       Lifestyle factors remain crucial for longevity and health. ·       Regular sauna use significantly reduces all-cause mortality. ·       Testing and monitoring are essential for personalized health. ·       Combining therapies can enhance the effectiveness of treatments.   Chapters 00:00 Introduction to Age Hacking and Organ Age Testing 01:26 Understanding Organ Age and Proteomic Testing 07:58 The Pace of Aging and Longevity Insights 12:54 Senolytics and Multi-Drug Gerotherapy 23:53 Cleaning the System for Regenerative Therapies 25:46 Rejuvenation Factors and Supplements 26:02 Exploring GLP-1s Beyond Weight Loss 34:39 Gene Therapy and Epigenetic Reprogramming 45:36 Lifestyle Science and Hormesis   Reach out to us at:    Website: https://gladdenlongevity.com/     Facebook: https://www.facebook.com/Gladdenlongevity/    Instagram: https://www.instagram.com/gladdenlongevity/?hl=en     LinkedIn: https://www.linkedin.com/company/gladdenlongevity    YouTube: https://www.youtube.com/channel/UC5_q8nexY4K5ilgFnKm7naw    

Some people hit walls. Others knock them down, build a lab, raise millions, and call up strangers to make a genetically engineered mouse. When Terry Pirovolakis learned his young son Michael had SPG50 — a rare degenerative disease with no treatment and a grim prognosis — he didn't spiral. He mobilized. In this jaw-dropping story of parental devotion and scientific hustle, Terry recounts how he went from IT consultant to biotech trailblazer, spearheading the development of a gene therapy from scratch. From homemade mice to confronting the Prime Minister, this episode is a whirlwind of heartbreak, grit, global collaboration, and unshakable hope. It's not just about saving one child — it's about lighting a path for thousands more.Sickboy LIVE in Calgary: https://www.showpass.com/podsummityyc2025/Follow Sickboy: Instagram: https://www.instagram.com/sickboypodcastTiktok: https://www.tiktok.com/@sickboypodcastDiscord: https://discord.gg/expeUDN

My guest is Dr. Sergiu Pașca, MD, professor of psychiatry and behavioral sciences at Stanford University. We discuss the biology and genetics of autism, why autism diagnoses are increasing and recent progress in using stem cells to understand and treat profound autism and other brain disorders. Dr. Pașca explains “organoids and assembloids”—human stem cell–derived tools he pioneered to study, treat and cure complex brain diseases. We also discuss ethical and safety issues with using gene editing and stem cells in humans. Read the episode show notes at hubermanlab.com. Thank you to our sponsors AG1: https://drinkag1.com/huberman David: https://davidprotein.com/huberman Helix: https://helixsleep.com/huberman BetterHelp: https://betterhelp.com/huberman Function: https://functionhealth.com/huberman Timestamps (00:00) Sergiu Pașca (02:08) Autism Spectrum Disorder, Incidence, Genetics (07:16) Is Autism More Common in Males? (09:35) Sponsors: David & Helix Sleep (11:56) Eye Contact in Babies, Fever; Proposed Causes of Autism; Genes (18:48) Genetic or Idiopathic Autism Diagnoses, Timothy Syndrome (21:37) Rise in Autism Diagnoses (26:46) Cause, Correlation & Neurological Disease; Schizophrenia, Do Vaccines Cause Autism? (31:34) Global Increase in Autism; Gene Therapy, CRISPR, Follistatin (41:05) Sponsors: AG1 & BetterHelp (43:41) Stem Cells, Ethics, Yamanaka Factors, Human Stem Cell Models (52:03) Umbilical Stem Cells; Stem Cell Injections & Dangers, Autistic Kids (59:30) Organoids, Modeling Brain Development, Intrinsic Development Timer (1:12:22) Assembloids, Brain Cell Migration & Circuit Formation, Self-Organization (1:21:22) Four-Part Assembloid, Sensory Assembloid, Pain Conditions (1:25:45) Sponsor: Function (1:27:33) Future Medical Therapies, Cell Banking, Immortalize Tissues, Rejuvenate Cells (1:34:56) Assembloids & Ethics, Importance of Nomenclature, Science Collaboration & Self-Correction (1:45:38) Cell Transplantation & Ethics, Timing (1:55:05) Genetic Testing for Parents, Genetic Penetrance (2:02:36) Assembloids, Timothy Syndrome, Epilepsy, Schizophrenia, Dystonia (2:14:30) Scientific Career, Walking, Art, Medical School (2:20:44) Zero-Cost Support, YouTube, Spotify & Apple Follow & Reviews, Sponsors, YouTube Feedback, Protocols Book, Social Media, Neural Network Newsletter Disclaimer & Disclosures Learn more about your ad choices. Visit megaphone.fm/adchoices