Podcasts about Gene therapy

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.

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.

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. 

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.

Welcome all to IS PHARMACOLOGY DIFFICULT Podcast! I am Dr Radhika VijayThis is my lecture/presentation for the conference = Global GoaCon 2025In this episode, I will talk about- "Biopharmaceuticals and Gene Therapy"It will be a great, insightful discussion!Its for all- doctor, pharmacologist, med student, pharmacist and laymen interested in science of Pharmacology, drugs and medicinesMy podcast is featured in "BEST SCIENCE PODCASTS"- Check the link here:https://podcasts.feedspot.com/india_science_podcasts/My podcast is featured in "BEST INDIAN MEDICAL PODCASTS". Check the link here:https://podcasts.feedspot.com/india_medical_podcasts/?feedid=5503395For all the updates and latest episodes of my podcast, please visit www.ispharmacologydifficult.com where you can also sign up for a free monthly newsletter of mine."Pharmacology Further" E-Newsletter and Podcast:The links for these are at all my websites and specifically:Link for E-Newsletter: https://pharmacologyfurther.substack.com/Link for the E-Newsletter Podcast: https://www.pharmacologyfurther.comIt actually contains lot of updates about the medical sciences, drug information and my podcast updates also.You can follow me on different social media handles like twitter, insta, facebook and linkedin. They all are with same name "IS PHARMACOLOGY DIFFICULT". If you are listening for the first time, do follow me here, whatever platform you are consuming this episode, stay tuned, do rate and review on ITunes, Apple podcasts, stay safe, stay happy, stay enlightened, Thank you!!Please leave Review on Apple podcasts!My E-Newsletter sign up at Substack!Connect on Twitter & Instagram!My books on Amazon & Goodreads!

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 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.

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.

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.  

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]

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]

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

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.

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

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

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

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