Podcasts about Gene therapy

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

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 a fascinating and insightful discussion, I sat down with Dr. Jon Brudvig, a medical scientist and expert in neuroscience and drug development. Together, we explored the science of aging, the critical role of cellular recycling, and the practical steps anyone can take to improve their healthspan. Jon Brudvig shared his journey from rare disease research to longevity science, offering actionable insights into how our daily choices impact the aging process. Such a good conversation, I have split it into two parts. What You'll Learn: The Cellular Foundation of Aging:Discover how Dr. Brudvig’s research into lysosomal storage diseases led him to understand the universal decline in cellular recycling (autophagy and lysosomal function) as a key driver of aging. Autophagy Explained:Learn why autophagy—the body’s process of breaking down and recycling damaged cellular components—is essential for health, and how modern lifestyles can disrupt this natural cycle. The Fundamentals of Longevity:Understand why exercise, diet, sleep, vitamin D, omega-3s, and real-life social connections are the true building blocks of a long and healthy life. Mouse Models and Human Longevity:Gain perspective on the limitations of animal studies in longevity research, and why findings in mice don’t always translate directly to humans. The mTOR Pathway and Protein Intake:Explore the debate around the mTOR pathway, protein consumption, and how balancing periods of growth and rest may be the key to optimal aging. Healthspan vs. Lifespan:Reflect on the difference between living longer and living better, and why increasing your years of good health (healthspan) is a more realistic and valuable goal than chasing extreme longevity. Key Takeaways: Aging is cellular: The decline in lysosomal and autophagy function is a core driver of aging and disease. Autophagy matters: Fasting, exercise, and periodic stressors help trigger the body’s natural recycling processes. Master the basics: Exercise, a balanced diet, quality sleep, and strong social ties are the most effective longevity interventions. Be cautious with quick fixes: Not all supplements or drugs that work in mice will work in humans—focus on proven fundamentals first. Balance is key: Intermittent activation of growth pathways (like mTOR) through meals and exercise, balanced with periods of rest, supports healthy aging. Protein quality counts: Mix your protein sources and prioritise whole foods for optimal health outcomes. Connect with Dr. Jon Brudvig for more insights into longevity and health: LinkedIn: Jon Brudvig Substack: https://jonbrudvig.substack.com/ 00:53 Dr. Brudvig’s Background and PhD Focus01:22 Gene Therapy and Lysosomal Storage Diseases02:45 Connecting Rare Diseases to Aging03:10 Lysosomal Function and Aging03:40 Professional Work in Drug Development04:01 The Link Between Professional and Personal Interests04:22 The Role of Lysosomes in Autophagy04:40 What is Autophagy?04:54 Why Autophagy Matters for Cellular Health05:07 The Recycling Analogy: Cells as Master Recyclers05:26 Evolutionary Importance of Autophagy06:14 Autophagy, Fasting, and Modern Lifestyles07:07 The Impact of Modern Comfort on Health07:28 Social Media, Longevity, and Biohacks08:00 The Interventions Testing Program Explained08:42 Mouse Studies and Their Limitations09:32 The Importance of Fundamentals: Exercise, Diet, Sleep10:22 Omega-3s and Social Networks for Longevity11:00 The Hallmarks of Aging11:40 Homeostasis and the Aging Process12:20 Healthspan vs. Lifespan: Which Matters More?13:07 The Two Camps in Longevity Science13:51 Promising Interventions: SGLT2 Inhibitors and Rapamycin14:45 Rapamycin: Hype, Dosing, and Human Application15:53 mTOR Pathway, Protein Intake, and Longevity17:00 Balancing mTOR Activation and Autophagy18:00 Practical Protein Advice: Whey, Collagen, and Plant-Based Sources18:45 Final Thoughts: Eat Real Food and Mix Protein Sources See omnystudio.com/listener for privacy information.

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, "Overviewing Consensus Guidelines to Standardize Gene Therapy Care in Duchenne," Barry Byrne, MD, chief medical advisor at the Muscular Dystrophy Association and director of the Powell Gene Therapy Center at the University of Florida, discusses newly published consensus guidelines from the MDA and Parent Project Muscular Dystrophy on the safe delivery and monitoring of gene therapy in Duchenne muscular dystrophy (DMD). Byrne explains the rationale behind convening a global panel of experts, emphasizing the need for standardized practices in patient selection, administration, and follow-up care as gene therapy becomes more widely available. He highlights the importance of expanded multidisciplinary teams—including hematology, cardiology, nephrology, and immunology—in managing immune-related safety concerns, with particular attention to monitoring liver inflammation and emerging strategies such as rapamycin use. In addition, Byrne outlines how these guidelines address real-world challenges around access, including geographic barriers, language considerations, and financial constraints, while underscoring their role in shaping future gene therapy approaches as additional therapies move through development. Looking for more Neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:00 – Why consensus guidelines were needed for gene therapy in DMD 2:10 – Top-line clinical considerations from the published recommendations 4:30 – Protocols for monitoring and managing adverse events, especially liver toxicity 6:30 – Neurology News Network 8:30 – Addressing health equity, language access, and financial barriers in gene therapy care 12:00 – How these recommendations may shape the future of DMD treatment 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: Efgartigimod Aims to Become First Therapy for Seronegative Generalized Myasthenia Gravis Following Positive Phase 3 Data FDA Approves Lecanemab Autoinjector, Marking First At-Home Treatment for Alzheimer Disease Eisai Submits sBLA for Weekly Subcutaneous Lecanemab as Starting Dose 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.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novartis has increased its commitment to its partnership with Argo BioPharma by an additional $5.2 billion, focusing on RNAi agreements targeting cardiovascular diseases. This highlights the ongoing advancements and challenges in the biopharmaceutical industry. Biotechs are turning to special purpose acquisition companies (SPACs) as a way to go public amid the IPO freeze. Gene therapy, with its potential to cure deadly diseases, is still facing challenges in terms of insurance coverage in the U.S. The industry is seeing a shift with some of the biggest biotech SPACs from the 2021 bubble no longer on the market. Meanwhile, Cytokinetics' cardiac myosin inhibitor, aficamten, has shown promising results in a phase III study for patients with obstructive hypertrophic cardiomyopathy. RFK Jr. has announced plans to reorganize chronic disease programs in the US to address high COVID-19 death rates. Companies like Novartis and Arrowhead are making significant commitments to various programs, while Trump's efforts to shore up the pharma supply chain with U.S. API are being questioned. Novartis continues its cutting spree with layoffs in New Jersey.These developments shed light on the evolving landscape of the biopharmaceutical industry.

The upcoming BioPharmaChem Ireland Budget 2026 submission places skills and innovation at its core, emphasising the need for public investment and strategic collaboration across the ecosystem to unlock the sector's full potential.In the latest episode of Ibec Responds, Dr. Sinead Keogh, Director of Biopharmachem Ireland and Head of Ibec Sectors, is in conversation with Darrin Morrissey, CEO of NIBRT.Together, they discuss how the institute is helping to realise Ireland's ambition in cell and gene therapy. From cutting-edge research and world-class talent development to bold initiatives like the Research Ireland Impact Centre proposal, discover how Ireland is positioning itself as a global leader in therapeutics innovation.Thank you for listening. To explore all of Ibec's podcast offering, visit here. Make sure to follow Ibec Podcasts to stay up to date with new episodes.

Gene therapy is no longer reserved for rare diseases or billionaire tech founders. In this episode, I sit down with Jay Spall, senior executive at MiniCircle, to discuss how gene therapy has entered a new era—one where optimizing your health, building muscle, and extending your lifespan can be done safely, affordably, and effectively.Jay shares how MiniCircle's first breakthrough product, Follistatin, uses plasmid-based delivery to boost muscle growth, reduce fat, and improve metabolic health—all from a single injection. We break down what gene therapy really is, how it compares to CRISPR, how long the effects last, and what to expect in terms of results and safety. Plus, Jay offers a look into the company's future therapies and his powerful personal journey from chronic illness to radical transformation.We cover the science, the stories, and the surprising benefits you might not expect.Interested in receiving Follistatin gene therapy? Contact our clinic, Chara Health, at 818-356-8232 or email info@chara-health.comKey Takeaways:Introduction (00:00)Jay's personal health journey and introduction to gene therapy (02:24)Minicircle's gene therapy technology and initial success (08:29)Follistatin's benefits and real-world applications (11:14)Clinical study results and broader health benefits (13:52)Muscle gain, fat loss, and strength benefits (19:08)How long does Follistatin's benefit last (22:48)How involved is Follistatin treatment (28:01)Follistatin side effects (33:08)Challenges and future directions of gene therapy (42:24)Additional Resources:If you are interested in receiving Follistatin gene therapy, please contact our clinic Chara Health at 818-356-8232, or email info@chara-health.com.✨ Connect with Jay SpallWebsite: https://minicircle.io/our-therapies/follistatin/Instagram: https://www.instagram.com/minicircledna/—✨ Learn more about how to live a long and pain-free life: https://joykongmd.com/ ✨ Follow me on Facebook: https://www.facebook.com/stemcelldrjoy/ ✨ Follow me on Instagram: https://www.instagram.com/dr_joy_kong/ —Dr. Joy Kong is a regenerative medicine and anti-aging expert. Her podcast is part of her mission to reduce suffering and elevate happiness. Join us every week for the latest holistic health insights that will help you live a long and pain-free life.

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    

What information do we receive from the sound around us? Hear thoughts from Matt Goupell (University of Maryland) and Brian Monson (University of Illinois, Urbana-Champaign), who join the podcast to discuss their research, along with findings on speech in noisy environments and spatial awareness.Goupell and Monson are presenting at the 2025 ASHA Research Symposium on Hearing at the ASHA Convention. As part of ASHA Voices' annual coverage of that symposium, they join the podcast to discuss their presentations and research.First, Monson shares what we can learn from extended high frequencies—those frequencies higher than 8,000 Hz. He shares thoughts on why these quieter frequencies may be the first to go. Then, Goupell tells us what bilateral cochlear implants are doing well, and how he hopes to see them improve in the future.Learn More:Research Symposium on HearingASHA Voices: A Promising Outlook for Gene Therapy and Hearing LossASHA Voices: The Community Link to Hearing Care AccessTranscript

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Regeneron's experimental drug for myasthenia gravis, cemdisiran, has shown promising results in a Phase III trial and the company is aiming to file for FDA approval by 2026. Pharmaceutical spending in China has reached record levels, with more than $48 billion spent in the first half of the year. Former FDA Commissioner Scott Gottlieb has raised concerns about the US losing its edge in biopharma innovation to China. Additionally, a drug program for a rare neurodegenerative disorder has been discontinued by Amylyx, while pharma companies are increasingly focusing on scientific research and partnerships. Lilly's obesity pill is heading to the FDA for approval, AbbVie is investing in psychedelics, and HHS has announced more changes in the industry. The pharmaceutical landscape is rapidly evolving with new developments and challenges arising.Pharma CEOs are facing increasing pressure amid political turmoil and public distrust over drug pricing. The industry spent over $48 billion in China in the first half of the year, with expectations of increased deal volume in the future. Legal experts are working to determine ownership rights of AI creations in biotech. Obesity treatments are being compared for effectiveness, while biotech companies are making strides in AI-driven manufacturing. Job opportunities in gene therapy and biostatistics are available.

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

In this episode of Moving Medicine Forward, host Jeremy Schrand welcomes Dr. Rich Curry to discuss how CTI is helping redefine what's possible in clinical research. From the complexities of cell and gene therapy to the human stories behind the science, Dr. Curry shares how CTI's expertise in rare diseases and regenerative medicine is accelerating innovation and improving patient outcomes. Discover how CTI's patient-first approach and global regulatory insight are shaping the future of advanced therapies.01:03 Dr. Curry explains how his background in neuropsychology and oncology research led him to clinical trials and CTI. 03:08 Dr. Curry highlights why cell and gene therapy excites him, emphasizing its potential to transform treatment approaches. 04:20 He explains how CTI's full-service research model supports complex, high-risk cell and gene therapy trials. 05:39 Dr. Curry describes the critical safety considerations in cell and gene therapy trials, particularly around immune system risks. 09:31 He explains how CTI approaches long-term follow-up and risk mitigation, sometimes monitoring patients for up to 15 years. 10:51 Dr. Curry shares how CTI stays ahead of global regulatory expectations through its dedicated regulatory and scientific affairs team. 12:03 He gives an example of how CTI helped a sponsor overcome regulatory challenges by identifying risk factors to ensure trial continuation. 13:16 Dr. Curry explains how CTI's focus on regenerative medicine and rare disease expertise sets it apart in gene therapy support. 14:01 He discusses how CTI's patient-first philosophy shapes trial design to improve patient experience and trial success. 14:54 Dr. Curry shares excitement for future innovations, especially in treating solid tumors, autoimmune diseases, and neurodegenerative disorders. 17:00 He explains how CTI prepares for the next wave of therapies by building strong sponsor partnerships and providing excellent service.  

Subscribe to our channel: https://www.youtube.com/@optispanGet Our Newsletter (It's Free): https://www.optispan.life/Meet the founders (Ivan Morgunov, Anna Vakhrusheva) of Unlimited Bio, a biotech company developing gene therapies in special economic zones, operating outside of traditional FDA regulation. This episode explores their model, the science behind their therapies targeting the extracellular matrix, and the future of accessible anti-aging treatments.0:00 - The Mission: Fighting Overregulation in Longevity9:31 - The Science: Why the Extracellular Matrix is Key30:16 - The Offshore Model: How It Works & The Controversies54:04 - Their First Therapy: VEGF Gene Therapy Explained55:51 - The Quality Debate: GMP vs. A New Approach1:24:16 - The Future Pipeline: Follistatin, Klotho & Combination Therapies1:48:27 - The Vision: A New Regulatory System for the USProduction: Nicholas Arapis, https://videocastproductions.comDISCLAIMER: The information provided on the Optispan podcast is intended solely for general educational purposes and is not meant to be, nor should it be construed as, personalized medical advice. No doctor-patient relationship is established by your use of this channel. The information and materials presented are for informational purposes only and are not a substitute for professional medical advice, diagnosis, or treatment. We strongly advise that you consult with a licensed healthcare professional for all matters concerning your health, especially before undertaking any changes based on content provided by this channel. The hosts and guests on this channel are not liable for any direct, indirect, or other damages or adverse effects that may arise from the application of the information discussed. Medical knowledge is constantly evolving; therefore, the information provided should be verified against current medical standards and practices.More places to find us:Twitter: https://twitter.com/optispanpodcastTwitter: https://twitter.com/optispanTwitter: https://twitter.com/mkaeberleinLinkedin: https://www.linkedin.com/company/optispanInstagram: https://www.instagram.com/optispanpodcast/TikTok: https://www.tiktok.com/@optispanhttps://www.optispan.life/Hi, I'm Matt Kaeberlein. I spent the first few decades of my career doing scientific research into the biology of aging, trying to understand the finer details of how humans age in order to facilitate translational interventions that promote healthspan and improve quality of life. Now I want to take some of that knowledge out of the lab and into the hands of people who can really use it.On this podcast I talk about all things aging and healthspan, from supplements and nutrition to the latest discoveries in longevity research. My goal is to lift the veil on the geroscience and longevity world and help you apply what we know to your own personal health trajectory. I care about quality science and will always be honest about what I don't know. I hope you'll find these episodes helpful!

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Do you ever wonder who's actually getting gene therapy around the world? So have we! Dr. Hank Greely from Stanford and Simon Fletcher, a nurse and PhD Candidate from Oxford, break it down in a way that's eye-opening and real. Then stick around for Dr. Radek Kaczmarek's update on the latest game-changing research. Two fascinating chats, one episode, this is BloodStream you don't want to miss!   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  

The Foundation funded earlier, proof-of-concept studies for the Best1 gene therapy and later launched Opus to develop several IRD gene therapies.

Earlier this year, baby KJ became the first patient in the world to receive a personalized gene editing therapy, which was developed by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine. In June, KJ went home after spending more than 300 days at CHOP. On this episode of Breaking Through, Madeline speaks to KJ's parents, Kyle and Nicole, about the treatment that changed KJ's life – and how he's doing today.

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

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

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Novo Nordisk's drug Wegovy has received approval for label expansion, potentially boosting its market performance. Tonix has also secured approval for a new fibromyalgia medication. Additionally, Novo has teamed up with GoodRx to provide lower prices for its drugs. Merck KGaA has made a significant investment of $2 billion in RNA-targeting technology through a collaboration with Skyhawk Therapeutics. On the sterilization front, Fedegari now offers tailored solutions for the pharmaceutical sector. In other updates, Pfizer's sickle cell therapy did not succeed in Phase III trials, while gene therapy company Kriya has raised an impressive $313 million. Kennedy has shown support for mRNA vaccines in cancer treatment. Bausch Health has closed a facility, Precigen has received FDA approval for an immunotherapy drug, and Eli Lilly has partnered with Superluminal in the obesity pipeline. Stay tuned for more industry news and developments.

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

About this episode:  Sickle cell disease affects an estimated 100,000 people in the United States. Recent advancements in gene therapies and medicines like hydroxyurea are diminishing extreme pain, reducing strokes, and extending survival times for those afflicted by the disease. In this episode: leading sickle cell disease expert Dr. Mark Gladwin explains how revolutionary new treatments work and discusses the challenges to access to life-saving care. Guest: Dr. Mark Gladwin is a physician-scientist and the Dean of the University of Maryland School of Medicine and Vice President for Medical Affairs at the University of Maryland, Baltimore. His research focuses include sickle cell disease and hypertension. Host: Dr. Josh Sharfstein is vice dean for public health practice and community engagement at the Johns Hopkins Bloomberg School of Public Health, a faculty member in health policy, a pediatrician, and former secretary of Maryland's Health Department. Show links and related content: New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle—CNBC Gene Therapy: What You Need to Know—Sickle Cell Disease Association of American No More Pain: Breakthrough Sickle Cell Treatment from Johns Hopkins Offers Curative Potential—Johns Hopkins School of Medicine Transcript information: Looking for episode transcripts? Open our podcast on the Apple Podcasts app (desktop or mobile) or the Spotify mobile app to access an auto-generated transcript of any episode. Closed captioning is also available for every episode on our YouTube channel. Contact us: Have a question about something you heard? Looking for a transcript? Want to suggest a topic or guest? Contact us via email or visit our website. Follow us: @‌PublicHealthPod on Bluesky @‌JohnsHopkinsSPH on Instagram @‌JohnsHopkinsSPH on Facebook @‌PublicHealthOnCall on YouTube Here's our RSS feed Note: These podcasts are a conversation between the participants, and do not represent the position of Johns Hopkins University.

Sam Altman is backing Merge Labs, a new brain-computer interface venture exploring a gene therapy approach to connect human brains with AI. The company is considering genetically modifying brain cells to respond to ultrasound, enabling a less invasive implant system. Merge is currently raising $250 million at an $850 million valuation, with major backing expected from OpenAI's venture arm. This move positions Altman in direct competition with Elon Musk's Neuralink, which uses electrode-based implants. Merge's approach combines sonogenetics and embedded ultrasound hardware, aiming for high-bandwidth communication between thought and AI systems. Altman is co-founding the project but won't take a day-to-day role or personally invest.

"...and by the time they figure out what went wrong, we'll be sitting on a beach, earning twenty percent." This is part 2 of our podcast series covering the important factors in successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be applied to positively impact gene therapy in FSHD. In part 2, we focus on viral dosing and how it affects therapeutic biodistribution and expression. In addition, we reveal what is known about variable durability of transgene expression and what to look for to predict a durable, or non-durable, therapeutic. Check out the MyFSHD Youtube for the music, as well.

In this episode, host Jonathan Sackier is joined by Emanuele Angelucci, Director of Hematology and Cellular Therapies and Director of the Stem Cell Transplant and Cellular Therapies Program at the Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Ospedale Policlinico San Martino Hospital in Genoa, Italy.   Timestamps   00:00 – Introduction 02:14 – The future of stem cell transplantation 03:15 – What continues to drive Emanuele in his work 05:40 – Donation of haematopoietic stem cells 10:00 – Gene therapy for haemoglobinopathies 11:40 – Will stem cell transplantation remain central in treating thalassaemia and sickle cell disease? 14:14 – Emanuele's work on iron overload and toxicity 16:53 – Current landscape for haemoglobinopathies 22:11 – Key recommendations in the most recent clinical guidelines for haemoglobinopathies  25:00 – Promising developments on the horizon for patients with haemoglobinopathies  28:00 – Emanuele's key takeaways 

"My friends are toys. I make them. It's a hobby. I'm a genetic designer." This is a two part podcast covering the important factors is successful gene therapy and introduces the knowledge gained from other neuromuscular diseases and how it can be used to positively impact gene therapy in FSHD. In part 1, we focus on AAV serotypes, transgene constructs, immune responses, and delivery. We look into DMD gene therapy and how that can impact the current FSHD gene therapy programs. Check us out on the MyFSHD Youtube channel as well.

A single gene that reverses ageing by over 13 years without activating pluripotency or cancer risk? Shift Bioscience may have cracked the code with SB000, an AI-discovered gene that could revolutionise how we approach ageing, rejuvenation, and gene therapy. In this detailed breakdown, Lisa Tamati, longevity coach, biohacker, and host of the Longevity & Lifestyle podcast Pushing The Limits, explores how SB000 was discovered using cutting-edge AI and transcriptomic ageing clocks, what it actually does at the cellular level, and what this means for the future of safe, targeted anti-aging interventions. What you'll learn in this episode: How cell age reversal has become a real possibility Why Yamanaka factors (OSKM) carry cancer risk How Shift Bioscience used AI to simulate 562 million gene combinations What makes SB000 unique as a “transcriptomic rejuvenator” The therapeutic potential of SB000 in reversing ageing safely Why this matters for the future of biohacking, longevity science, and gene therapy Visit Shift Biosciences to learn more about this study: https://www.shiftbioscience.com/

We love to hear from our listeners. Send us a message.In Episode 109 of Cell & Gene: The Podcast, Host Erin Harris talks to Will Chou, M.D., CEO of Passage Bio about the promise and challenges of developing gene therapies for frontotemporal dementia (FTD), a neurodegenerative disease with strong genetic underpinnings. Dr. Chou discusses the biological basis of FTD and the complex scientific, manufacturing, and regulatory hurdles that come with targeting the brain. They discuss the latest interim results from the upliFT-D study of Passage Bio's PBFT02, including safety and tolerability findings, dose cohort comparisons, and the evolving strategies around immunosuppression. Dr. Chou also highlights upcoming milestones for PBFT02 and what they could mean for advancing gene therapies in neurodegenerative diseases.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Subscribe to our channel: https://www.youtube.com/@optispanGet Our Newsletter (It's Free): https://www.optispan.life/Join longevity pioneers Dr. Aubrey de Grey, Dr. Brian Kennedy, and Dr. Matt Kaeberlein for a rare, unfiltered discussion on the future of aging. They debate the timeline for longevity escape velocity, the promise of new damage-repair therapies, and offer their candid thoughts on today's experimental treatments.0:00 - Intro1:50 - Longevity Escape Velocity: Are We Close?7:20 - The Big Debate: Damage Repair vs. Slowing Growth15:36 - Aubrey's Groundbreaking Combination Study in Mice21:17 - The Stacking Problem: Why Don't Combinations Always Work?33:42 - The Clinical Frontier: What's Real vs. "Sketchy"?47:03 - Gene Therapy & TPE: Would These Experts Do It?1:00:13 - The Future: What Are They Most Excited About?Production: Nicholas Arapis, https://videocastproductions.comDISCLAIMER: The information provided on the Optispan podcast is intended solely for general educational purposes and is not meant to be, nor should it be construed as, personalized medical advice. No doctor-patient relationship is established by your use of this channel. The information and materials presented are for informational purposes only and are not a substitute for professional medical advice, diagnosis, or treatment. We strongly advise that you consult with a licensed healthcare professional for all matters concerning your health, especially before undertaking any changes based on content provided by this channel. The hosts and guests on this channel are not liable for any direct, indirect, or other damages or adverse effects that may arise from the application of the information discussed. Medical knowledge is constantly evolving; therefore, the information provided should be verified against current medical standards and practices.More places to find us:Twitter: https://twitter.com/optispanpodcastTwitter: https://twitter.com/optispanTwitter: https://twitter.com/mkaeberleinLinkedin: https://www.linkedin.com/company/optispanInstagram: https://www.instagram.com/optispanpodcast/TikTok: https://www.tiktok.com/@optispanhttps://www.optispan.life/Hi, I'm Matt Kaeberlein. I spent the first few decades of my career doing scientific research into the biology of aging, trying to understand the finer details of how humans age in order to facilitate translational interventions that promote healthspan and improve quality of life. Now I want to take some of that knowledge out of the lab and into the hands of people who can really use it.On this podcast I talk about all things aging and healthspan, from supplements and nutrition to the latest discoveries in longevity research. My goal is to lift the veil on the geroscience and longevity world and help you apply what we know to your own personal health trajectory. I care about quality science and will always be honest about what I don't know. I hope you'll find these episodes helpful!

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta has released new safety data for their gene therapy Elevidys, showing zero ambulatory deaths in patients with Duchenne muscular dystrophy. This data confirms the therapy's positive risk/benefit profile. Meanwhile, Novo Nordisk's rapid rise and fall was driven by GLP-1s like Ozempic and Wegovy, leading to a plummet in stock value. Insmed has received FDA approval for the first bronchiectasis drug and DPP1 blocker, Brinsupri. Texas has accused Eli Lilly of "bribing" prescribers to push their drugs Mounjaro and Zepbound, leading to a new lawsuit. Genscript is entering a new era of innovation and trust, offering exclusive promos and events. Additionally, Tang Capital is on a buyout binge in the biotech industry, while a top ARPA-H official has departed in protest of cuts to mRNA funding. The biotech industry continues to see contractions with companies like AbSci, Fate, and Oric downsizing. In career advice, loyalty is seen as a flawed strategy that can cost individuals opportunities.Arrowhead, a biotech company, has emerged as a savior in the RNA interference (RNAi) pipeline after Sarepta faced troubles unrelated to Arrowhead's assets. Concentra Biosciences, a biotech shell company, has been on a buying spree, acquiring four biotechs in the past month and seven so far this year. President Trump's most favored nation drug pricing policy is seen as unfavorable for all parties involved. Novo Nordisk experienced a rapid rise and fall due to the success of ozempic and wegovy. Lilly is facing drug pricing pressure while defending its injectable GLP-1 empire. Novartis is rumored to be considering acquiring RNA specialist Avidity. Despite safety concerns, Sarepta beat Q2 estimates, but not due to sales of its product Elevidys. Various biotech companies are downsizing while others are receiving significant investments.

Today, I'm joined by the trailblazing Liz Parrish, a true pioneer in the world of longevity science and regenerative medicine. Liz doesn't just talk about cutting-edge gene therapies—she's the first patient to actually undergo many of them herself. In our conversation, Liz shares how her journey from stem cell advocacy to biotech innovation was fueled by a deeply personal mission: her son's diagnosis with type 1 diabetes.   What we discuss: How Liz Parrish got into genetics and stem cells ... 00:06:45 Transition from childhood disease to treating aging ... 00:09:35 Difference between stem cells, gene therapy, and exosomes ... 00:14:20 Challenges in gene therapy delivery and cell reprogramming ... 00:19:22 Liz Parrish as patient zero for gene therapies ... 00:22:01 Results and lessons from Parrish's self-experimentation ... 00:23:16 Senescent cells, telomeres, and aging interventions ... 00:29:22 Evolution and progress in gene therapy ... 00:34:14 Barriers and ethics of gene therapy for rare diseases and children ... 00:36:53 Public perception and future accessibility ... 00:44:55 Affordability, scaling, and redosing gene therapies ... 00:55:00 Practical longevity habits and hormones ... 01:03:54   Our Amazing Sponsors: OraltidePro™️by Profound Health - A unique mouthwash that: Promotes growth of shrinking gums, Speeds healing of mouth & tongue, Prevents oral infections (such as gingivitis), helps with enamel remineralization, reduces bacteria growth and etching and fills slots in damaged enamel. Check out OraltidePro™️at profound-health.com and use code NAT15 for 15% off your first order. NMN+G Rx by Wizard Sciences -  A scientifically formulated blend of NMN, ginsenosides from Panax ginseng, and apigenin. Together, they enhance mitochondrial function, boost NAD+ levels, and support cellular repair. Go to wizardsciences.com and look for NMN+G. Use code NAT15 at checkout to get 15% off your purchase. Fresh-Pressed Olive Oil Club: Get a full-size $39 bottle of one of the world's finest artisanal olive oils — fresh from the new harvest—for just $1. No commitment to buy anything, now or ever. Go to GetFreshLongevity.com for a free bottle and pay just $1.   More from Nat:  YouTube Channel Join My Membership Community Sign up for My Newsletter  Instagram  Facebook Group

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Vinay Prasad has returned to his position as director of the Center for Biologics Evaluation and Research at the FDA after a sudden exit less than two weeks ago. The FDA has restricted the patient pool for bluebird's Skysona gene therapy due to safety concerns, and Sarepta's AAV gene therapy platform has come under scrutiny following patient deaths from liver injuries. Genscript is celebrating a new era of innovation and trust.The text discusses the scrutiny faced by AAV gene therapy technology following patient deaths related to Sarepta Therapeutics' platform. There is a push for improvement in the technology, while some are looking to move on to other options. The article also highlights the importance of mRNA technology in cancer research and the need for continued investment in this area. Additionally, it mentions the FDA's decision on Biogen and Eisai's Alzheimer's drug, as well as five oral obesity drugs that could rival Lilly's Orforglipron. Other topics covered include cancer drug developments, gene therapy news, and upcoming industry events.Stay tuned for more updates on the latest news in the pharmaceutical and biotechnology industries.

Send us a text00:00 - Surf's Up 6.10.1This conversation covers three topics. Jörn Schattenberg discusses two papers that suggest an exciting role NITs might play in future prescribing decisions, two executives from LiverRight describe the U.S.'s first virtual liver clinic, and Tom Jobson of Predictive Health Intelligence updates us on how simple analyses of large data bases can identify and motivate high-risk liver patients to visit their doctors. 00:17:30 - Roundtable: NITs might help physicians determine the value of metabolic vs. specific anti-fibrotic effects for individual patientsThe overall theme of this roundtable is "Major Stories and Events of the First Half, 2025." Jörn's contribution is to discuss two papers that use proteomic analyses to determine the degree to which a patient's MASH is driven by metabolic issues as compared to direct fibrotic challenges. Today, when we have only a few drugs, limited NITs and no proteomic tests available for use in practice, these findings point to directions for future research and test development. Over time, providers may be able to prescribe based on the knowledge of h0w much benefit metabolic agents might provide as compared to anti-fibrotics. This may point to step therapy or multi-agent first line therapy, but it is an exciting idea.00:29:50 - Newsmakers: LiverRight opens America's first virtual liver clinic LIverRIght CEO Brandon Tudor and Chief Medical Officer Alexander Lalos join Roger Green to discuss the launch of America's first virtual hepatology clinic. Brandon shares his personal history to explain why providing fast access is so important to him. Alex describes his original motivation to go into Transplant Hepatology and how, over time, his focus has shifted from healing the sick to preventing disease in the first place. They describe how LiverRight works and their successes to date in reducing patients' time from first contact to visit, often from months to days. NOTE: Roger Green is a paid advisor to LiverRight.00:56:44 - Expert: Tim Jobson describes how Hepatoscope is helping the NHS identify untreated high-risk liver patients and bring them to the office for screening. Tim describes hepatoSIGHT, a tool that "allows clinicians to get their hands on the data and to find patients both for treatment and for clinical trials."  He describes it as standard in Somerset, UK, now, and proceeds to share new data about the patient experience. This is a unique program in that providers reach out to tell individual patients they should visit the physician based on information found in their medical records. Interestingly, patient response is overwhelmingly positive. Six in ten respondents rated their satisfaction with the process, giving it a mean of 4.8 on a 5-point scale. Tim also shared some preliminary modeling suggesting that sustained use of hepatoSIGHT could increase clinical trial participation as much as 50-fold if trial sites had the capacity to take all these patients. In all, this is a warming, affirming look at whether and how patients know they benefit from what we ask them to do and share.   01:29:58 - ConclusionThe business report discusses the next few episodes and asks who would like to meet Roger at Paris MASH.

Gene therapy for sickle cell disease—patient perspective by TRANSFUSION's Monthly Podcast

In this episode of the Brainstorm, Sam, Nick, and Multiomics Research Analyst, Shea Wihlborg, dive into the complexities of the FDA's recent decisions and their implications for gene therapy. They discuss the controversial halt of a Duchenne muscular dystrophy treatment, the role of AI in drug discovery, and the exciting future of genomics. If you know ARK, then you probably know about our long-term research projections, like estimating where we will be 5-10 years from now! But just because we are long-term investors, doesn't mean we don't have strong views and opinions on breaking news. In fact, we discuss and debate this every day. So now we're sharing some of these internal discussions with you in our new video series, “The Brainstorm”, a co-production from ARK and Public.com. Tune in every week as we react to the latest in innovation. Here and there we'll be joined by special guests, but ultimately this is our chance to join the conversation and share ARK's quick takes on what's going on in tech today.Key Points From This Episode:Discussion on the FDA's recent halt of a Duchenne muscular dystrophy treatment due to safety concerns.Exploration of the role of AI in improving drug discovery and clinical trials.Insights into the future of gene therapies for both rare and common diseases.The potential impact of regulatory changes on innovation in the biotech industry.For more updates on Public.com:Website: https://public.com/YouTube: @publicinvestX: https://twitter.com/public

In this podcast, Dr. Zoe Ngo and Dr. Austin Wang discuss the AJHP Case Study "Implementing standard practices in the safe handling of gene therapy and biohazardous drugs in a health-system setting” with host and AJHP Editor in Chief Dr. Daniel Cobaugh. The information presented during the podcast reflects solely the opinions of the presenter. The information and materials are not, and are not intended as, a comprehensive source of drug information on this topic. The contents of the podcast have not been reviewed by ASHP, and should neither be interpreted as the official policies of ASHP, nor an endorsement of any product(s), nor should they be considered as a substitute for the professional judgment of the pharmacist or physician.

Subscribe to our channel: https://www.youtube.com/@optispanGet Our Newsletter (It's Free): https://www.optispan.life/Following a breaking news story about hospitalizations at a longevity conference, scientist Dr. Matt Kaeberlein shares his firsthand experience from the event. He reveals the dangerous mix of legitimate science, unregulated therapies, and outright pseudoscience, offering critical advice on how to separate hope from hype.0:00 - A Tale of Three Conferences5:35 - The Longevity 'Religious Revival'10:09 - The Peptide Incident: What Happened?21:36 - Red Flags: How to Spot a Charlatan24:35 - Gene Therapy, TPE & More: Hope or Hype?36:25 - Reacting to the News Story1:02:32 - The Story You Won't Believe ('Feeling Your Energy')Production: Nicholas Arapis, https://videocastproductions.comDISCLAIMER: The information provided on the Optispan podcast is intended solely for general educational purposes and is not meant to be, nor should it be construed as, personalized medical advice. No doctor-patient relationship is established by your use of this channel. The information and materials presented are for informational purposes only and are not a substitute for professional medical advice, diagnosis, or treatment. We strongly advise that you consult with a licensed healthcare professional for all matters concerning your health, especially before undertaking any changes based on content provided by this channel. The hosts and guests on this channel are not liable for any direct, indirect, or other damages or adverse effects that may arise from the application of the information discussed. Medical knowledge is constantly evolving; therefore, the information provided should be verified against current medical standards and practices.More places to find us:Twitter: https://twitter.com/optispanpodcastTwitter: https://twitter.com/optispanTwitter: https://twitter.com/mkaeberleinLinkedin: https://www.linkedin.com/company/optispanInstagram: https://www.instagram.com/optispanpodcast/TikTok: https://www.tiktok.com/@optispanhttps://www.optispan.life/Hi, I'm Matt Kaeberlein. I spent the first few decades of my career doing scientific research into the biology of aging, trying to understand the finer details of how humans age in order to facilitate translational interventions that promote healthspan and improve quality of life. Now I want to take some of that knowledge out of the lab and into the hands of people who can really use it.On this podcast I talk about all things aging and healthspan, from supplements and nutrition to the latest discoveries in longevity research. My goal is to lift the veil on the geroscience and longevity world and help you apply what we know to your own personal health trajectory. I care about quality science and will always be honest about what I don't know. I hope you'll find these episodes hel

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy Elevidys and growing scrutiny over the company’s transparency following multiple patient deaths. In this week’s episode of "The Top Line," we explore Sarepta’s brief standoff with the FDA over Elevidys, a short-lived marketing pause, and the regulator’s surprise reversal on the treatment. Fierce Pharma’s Fraiser Kansteiner and Angus Liu sit down with Fierce Biotech’s Gabrielle Masson to recap the story and discuss the broader implications the Elevidys saga may have for gene therapy, the FDA and the pharmaceutical industry. To learn more about the topics in this episode: Sarepta pivots to siRNA and lays off 500 staffers as Elevidys gets box warning Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe See omnystudio.com/listener for privacy information.

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. He's one of the first to be successfully treated with a bespoke therapy targeting a tiny but deadly error in his DNA. His doctors hope the technology could one day help the estimated 350 million people worldwide with rare diseases. - एक दुर्लभ आनुवंशिक रोगसहित जन्मिएको शिशुले व्यक्तिगत जीन सम्पादन उपचारबाट उल्लेखनीय सुधार देखाएको छ। डाक्टरहरू विश्वास गर्छन् कि यो प्रविधिबाट भविष्यमा लाखौं दुर्लभ रोगीहरूलाई उपचार सम्भव हुनेछ।

A top official at the Food and Drug Administration steps down after the agency changes course on a treatment for Duchenne muscular dystrophy. What does this episode say about the Trump Administration's approach toward drug development and innovation? Plus, Robert F. Kennedy Jr. says he plans to overhaul the National Vaccine Injury Compensation Program. Learn more about your ad choices. Visit megaphone.fm/adchoices

Anti-aging expert Dr Rhonda Patrick reveals how magnesium, HIIT workouts, creatine, and vitamin D can prevent disease, slow aging, and boost brain power - starting today.  Dr. Rhonda Patrick is a PhD biomedical scientist, expert in aging, nutrition, and metabolism, and founder of FoundMyFitness. She produces a monthly series of in-depth, science-focused podcasts exploring actionable strategies for enhancing longevity, cognitive performance, and physical health. She explains: Why cardiovascular fitness is the #1 predictor of lifespan The groundbreaking science behind rejuvenating your heart by 20 years How small daily habits can cut your risk of Alzheimer's and cancer The hidden link between mitochondrial health, aging, and chronic disease 00:00 Intro 02:17 My Mission to Improve People's Health 03:50 What Impact Will Rhonda's Research Have on People? 05:22 The Role of Genetics in Aging vs. Lifestyle 07:36 Lifestyle and Life Expectancy 10:06 The Future of Aging, Longevity, and Gene Therapy 15:28 Death-Related Risks of Being Sedentary 22:30 How to Improve Your Cardiorespiratory Fitness 25:45 Best Workout Routine to Improve Cardio Health 30:14 Norwegian 4x4 Training Explained 32:41 How the Body Generates Energy and Exercise Intensity 38:28 Why We Can't Drink Lactate and the Effects of Vigorous Training 42:33 How to Reduce Cognitive Decline 47:11 What Causes Dementia and Alzheimer's 53:09 Do Multivitamins Improve Cognitive Performance? 01:00:26 70% of the U.S. Population Is Vitamin D Deficient 01:03:12 Vitamin D Deficiency and Increased Risk of Dementia 01:14:41 How the Keto Diet Affects Life Expectancy and Healthspan 01:19:43 Exogenous Ketones and Cognitive Repair 01:24:06 Recommended Superfoods 01:26:46 Omega-3: Effects on Mental Health, Depression, and Longevity 01:32:21 Is Omega-3 Supplementation the Same as a High Omega-3 Diet? 01:41:28 Effects of Creatine on Cognitive Function 01:49:18 Link Between Cancer Outcomes and Creatine 01:50:09 Does Creatine Cause Hair Loss? 01:59:06 Fasting Windows to Achieve Autophagy 02:01:01 Intermittent Fasting: Do's and Don'ts 02:02:19 Effects of Fasting on Sleep 02:04:49 How Soon After Training Should You Take Protein? 02:08:29 What Is Insulin Resistance? 02:10:28 Benefits of Red Light Therapy 02:11:36 Infrared vs. Traditional Saunas 02:18:41 Sauna Benefits: Reducing Stress and Improving Mood 02:22:12 What Are Microplastics and Are They Harmful? 02:27:48 The Role of Fiber in Eliminating Microplastics 02:31:43 Are There Risks to Living Near a Golf Course? 02:42:18 What Is Choline? Follow Dr Rhonda: Instagram - https://bit.ly/3TYAEUD X - https://bit.ly/3IFvCu1  Website - https://bit.ly/4o75U1F  Weekly health newsletter - https://bit.ly/4kYsR4f  You can subscribe to Rhonda's monthly series on YouTube and Spotify: YouTube: https://bit.ly/4l1FZW9  Spotify: https://bit.ly/45l77Lc  You can download Rhonda's free protocol guides, here: The Cognitive Enhancement Blueprint - https://bit.ly/44QYF6t  The Omega-3 Supplementation Guide- https://bit.ly/44Pi1sE  How to Train According to the Experts - https://bit.ly/4mbtzMm  Research Document: https://stevenbartlett.com/wp-content/uploads/2025/07/DOAC-Dr-Rhonda-Patrick-Independent-Research-further-reading.pdf The Diary Of A CEO: ⬜️Join DOAC circle here - https://doaccircle.com/  ⬜️Buy The Diary Of A CEO book here - https://smarturl.it/DOACbook  ⬜️The 1% Diary is back - limited time only: https://bit.ly/3YFbJbt  ⬜️The Diary Of A CEO Conversation Cards (Second Edition): https://g2ul0.app.link/f31dsUttKKb  ⬜️Follow Steven - https://g2ul0.app.link/gnGqL4IsKKb  Sponsors: Intuit - https://www.intuit.com/expert-careers/?cid=aud_nativly_reach_us_expertnetwork-fy25_aw_hostread-diaryofceo-na-60s_broad_audio_1x1_intuit-gtm_na_na   KetoneIQ - Visit https://ketone.com/STEVEN for 30% off your subscription order Learn more about your ad choices. Visit megaphone.fm/adchoices

Drs. Armstrong and Patel discuss current and emerging therapies for diabetes-related macular edema and wet age-related macular degeneration including anti-VEGF agents, the need for durability and reduced treatment burden, alternative delivery strategies, novel molecules, and gene-therapy.

Last month, scientists reported a historic first: they gave the first personalized gene-editing treatment to a baby who was born with a rare life-threatening genetic disorder. Before the treatment, his prognosis was grim. But after three doses, the baby's health improved. So how does it work? What are the risks? And what could this breakthrough mean for the 30 million people in the US who have a rare genetic disease with no available treatments?To help get some answers, Host Flora Lichtman is joined by the physician-scientists who led this research: geneticist Dr. Kiran Musunuru and pediatrician Dr. Rebecca Ahrens-Nicklas.Guests: Dr. Rebecca Ahrens-Nicklas is an assistant professor of pediatrics and genetics at the Children's Hospital of Philadelphia and the University of Pennsylvania.Dr. Kiran Musunuru is a professor of translational research at the University of Pennsylvania.Transcripts for each episode are available within 1-3 days at sciencefriday.com. Subscribe to this podcast. Plus, to stay updated on all things science, sign up for Science Friday's newsletters.