Podcasts about Gene therapy

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

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।

"When you are seeing great results in your research lab, you think that everything has been done. But then when you try to humanise your drug, a new adventure starts, because the CMC process is very difficult. In my opinion, it's as difficult as demonstrating efficacy." - Mari Carmen ÁlvarezMari Carmen Álvarez PhD. is the Managing Director of Tetraneuron, a pioneering Spanish biotech developing novel gene therapies for neurodegenerative diseases. With nearly two decades in biotech leadership, she founded Valencia University's first biotech spin-off and brings expertise spanning innovation management, business development, and fundraising from seed to Series A.In the latest PharmaSource podcast episode, Mari Carmen explains why scaling gene therapy development requires more than strong science, how to navigate the complex CDMO ecosystem, and what she learned from a previous biotech failure that shaped her approach to leadership and patient impact.Full interview: https://pharmasource.global/content/podcast/from-lab-to-patient-how-tetraneurons-gene-therapy-approach-is-tackling-alzheimers-disease/

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

Send us a textThis conversation is the fourth and final segment of SurfingMASH's April discussion of drug development in memory of Stephen A. Harrison. In addition to co-hosts Jörn Schattenberg, Louise Campbell and Roger Green, panelists include hepatologist and key opinion leader Sven Francque. The discussion focuses on PPARs, genetic medicines, and other emerging drug classes while considering the idea that drug therapies can have an impact on the liver independent of their effect on fibrosis regression. It begins with Sven  discussing his experience as a lead investigator in clinical trials for the pan-PPAR agonist lanifibranor, whose Phase 3 trial is now fully recruited. Sven states that in addition to fibrosis regression, lanifibranor is likely to exhibit other pleiotropic effects, and notes that vascular changes start early in the fibrotic progression process. After Sven elaborates on these effects, Louise asks about the SCD-1 agent Aramchol. This leads to a discussion about the idea that over time, therapy will probably come to incorporate two separate modes of action, with one to treat the metabolic dysfunction and the other to treat specific effects in the liver or, as Jörn puts it, "combining weight-neutral and weight-reducing drugs." This evolves into a discussion of what Louise terms "personal-centric" medicine, or what patient advocate Mike Betel has previously described on SurfingMASH as "tailored medicine."The rest of the conversation predominantly lists other classes of drugs, "safe" mitochondrial uncouplers, genetic medicines, and others. Roger inquires about the FASN inhibitors, which are entering Phase 3 clinical trials. Jörn says that the data appears positive and unique. That said, he and Sven agree we need more data.Louise sounds the closing note for this roundtable by discussing our co-founder, Stephen Harrison, and the energy and enthusiasm he brought to the entire drug development process. The group agrees that Stephen's impact continues to be felt through the MASLD community, even as he is missed by us all.

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.

Send us a text00:00 - Surf's Up, Season 6 Episode 8The conversation addresses three issues that are distinct, yet each is pivotal to the future of MASLD and MASH therapies. The first, from the Global Think-Tank on Steatotic Liver Disease, considers how personal and digital approaches can be combined to form the most effective strategy for patient management. In the second, Global Liver Institute President and CEO Larry Holden joins Roger Green to discuss the new challenges in Washington stemming from the Trump Administration and RFK Jr. The final section is the conclusion of our recent drug development roundtable, examining drug classes we did not previously discuss, along with a thought on where combination therapy is likely to lead. 00:04:57 - Global Think-Tank on SLD Roundtable, Part 2Behavior consultant Dr. Kristina Curtis joins Jörn Schattenberg, Louise Campbell and Roger Green to discuss issues related to patient-centered care. This discussion focuses on the elements of successful behavior change and the importance of real-time, actionable feedback. The group discusses the interplay of diagnostic test feedback, real-time personal exchanges and AI-based algorithms in what Kristina terms a "hybrid therapy."00:18:39 - Newsmaker: Larry HoldenGLI President and CEO Larry Holden addresses two issues related to current goings-on in Washington. First, he acknowledges that we are in for "dark times" under the current administration, and describes some of the decisions and challenges we face. Second, he suggests what individuals and organizations can do to create the best possible situation -- and even some "wins" -- for people living with liver disease. These suggestions reflect his experiences during a 30-year career on Capitol Hill, prior to his joining GLI.00:46:15 - Drug Development Roundtable, Part 4Sven Francque joins Jörn, Louise and Roger to share an up-to-date look at drug development. This discussion focuses on PPARs, genetic medicines, and other emerging drug classes. Sven uses the example of the pan-PPAR lanifibranor to explore the idea that drug therapies can have an impact on the liver independent of their effect on fibrosis regression. The group proceeds to discuss other emerging drugs in development and their modes of action. One theme: over time, we may see prescribers consider using different therapies to address metabolic vs. liver-specific effects, often in combination.01:00:47 - Business ReportThanks to our listeners, Jörn's vacation, Welcome Regeneron

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. The FDA has initiated a voucher program to address lower drug prices and boost domestic investment, with up to five companies being selected based on national priorities. Roche has halted shipments of Sarepta's Elevidys gene therapy after reports of patient deaths. Sarepta is under FDA scrutiny following a third patient death, while the agency welcomes a new top drug regulator and rejects multiple new drugs from entering the market. Insights from women in biopharma highlight the importance of resilience. Zhen Li, an engineer, is playing a crucial role in shaping the field of siRNA. Research suggests that GLP-1s may offer protection against neurodegenerative diseases. Dispatch Bio enters the scene with a substantial $216 million investment for universal CAR T therapy. Sarepta complies with an FDA request to suspend US shipments of Elevidys, raising concerns about transparency and impacting the company's reputation. Replimune experiences a significant drop in stock value following an FDA rejection, while AstraZeneca makes a bold $50 billion investment in US manufacturing to prepare for potential tariffs. Thank you for tuning in to Pharma and Biotech daily for the latest updates in the industry.

We are going to become a review podcast now. It is what i love to talk about and it will be mine. Video games, Movies, TV Shows, Comic Books, Albums of Heavy Metal mostly. Things i enjoy and love to talk about. Join Us Now!

Last week's public disclosure that a gene therapy from Sarepta had caused a third death led FDA to ask the company to stop distributing its DMD gene therapy Elevidys, a move the biotech has resisted. The deaths, and disputes between FDA and Sarepta, raise questions about the future of AAV gene therapies, as well as the future of FDA's platform technology designation. On the latest BioCentury This Week podcast, BioCentury's analysts unpack the events surrounding Sarepta's gene therapies and discuss how FDA, industry and patient groups should come together to learn the lessons from the tragic, avoidable deaths.BioCentury's analysts also assess Monday's appointment of Stanford professor and biotech executive George Tidmarsh to lead FDA's Center for Drug Evaluation and Research, and check in on the latest trends in venture financings. This episode of BioCentury This Week is sponsored by IQVIA Biotech.View full story: https://www.biocentury.com/article/656537#biotech #biopharma #pharma #lifescience #GeneTherapy #AAVTherapy #Sarepta #Elevidys00:01 - Sponsor Message: IQVIA Biotech02:03 - Gene Therapy17:59 - Leading CDER27:00 - Venture ReportTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.George Tidmarsh, a biopharma veteran and adjunct professor at Stanford's medical school, has been appointed as the new head of the FDA's Center for Drug Evaluation and Research (CDER). His extensive industry experience will be valuable in his new government position. Data secrecy among cell and gene therapy developers is hindering progress in the field, causing fragmentation, stalling innovation, and delaying access to treatments. Meanwhile, Sarepta is facing challenges with its elevidys shipments and has lost platform designation for its technology. At the same time, Roche's phase III trial in COPD has failed, impacting the market path for astegolimab. Layoffs are happening at companies like GSK, Sail, and BioNTech. Experts are exploring new ways to overcome barriers in cell therapy production.Data secrecy among cell and gene therapy developers continues to hinder progress and access to treatments. Acadia has introduced a new team and pipeline with ambitious goals. Patients are fighting for access to Brainstorm's ALS drug after promising real-world data. Moderna's withdrawal of its flu vaccine has left combination flu/COVID-19 vaccines in limbo. In other news, Sarepta is facing challenges with its DMD gene therapy, Ultragenyx's gene therapy for Sanfilippo syndrome is rejected by the FDA, GSK's Blenrep loses an adcomm vote, and BMS' anemia drug Reblozyl fails a Phase III trial. The FDA is experiencing layoffs and employee turnover amid an overhaul. Vinay Prasad overruled reviewers on Moderna's COVID-19 shot for kids. Upcoming events include a webinar on AI for real-world research and job opportunities in the biopharma industry. Readers are encouraged to provide feedback and suggest topics for future coverage.

Laurent Servais, MD, PhD - Eligible for Gene Therapy? The Complexity and Therapeutic Implications of Anti-AAV Antibodies

We love to hear from our listeners. Send us a message.On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Ralf Schmid, Ph.D., Associate Director of Preclinical Research at Novartis Biomedical Research, about the evolving use of large animal models in gene therapy development. Dr. Schmid discusses the current reliance on non-human primates (NHPs), their growing logistical and ethical challenges, and the emerging interest in alternatives like genetically engineered pigs and sheep. He outlines key considerations around safety, biodistribution, and immunogenicity that still necessitate large-animal testin —particularly for CNS-targeted AAV therapies — and emphasizes the need for thoughtful study design, responsible sourcing, and diversification in model systems. Dr. Schmid also previews his participation in the upcoming Next Generation Gene Therapy Vectors Summit and reflects on the future of preclinical safety testing in a landscape aiming to balance innovation, rigor, and compassion.Subscribe to the podcast!Apple | Spotify | YouTube

One of the challenges for developing gene therapies for inherited eye diseases is that a large number of individual mutations to a gene can drive the same disease. That makes conventional gene replacement therapy a difficult approach to treat a large number of patients. Ocugen is developing gene therapies that target master regulators of genetic networks and can work across different mutations. We spoke to Arun Upadhyay, chief scientific officer and head of research and development at Ocugen, about inherited retinal diseases, the company's platform technology that can work across different genetic mutations, and its potential applications beyond the eye. One note before we begin. Since recording this podcast, Ocugen announced that Carisma Therapeutics and Ocugen's subsidiary OrthoCellix, entered into a definitive merger agreement to create a Nasdaq-listed, late clinical stage regenerative cell therapy company focused on orthopedic diseases. That transaction is not discussed in this interview.

This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years. In this episode, Dr. Glorioso will discuss the reviews, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Beverly Davidson, PhD, The Children’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine. If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

For this exciting conversation, I am joined by NanoPalm CEO and co-founder Dr. Ali Alhasan and by Charles River Executive Director Ramin Baghirzade, who joins us to talk about Charles River's incubator program for startups like NanoPalm. Alhasan and his co-founder, Dr. Samar Alsudir, began NanoPalm to help reach rare disease patients who could benefit from advanced gene therapies. NanoPalm is the first company to conduct a gene therapy clinical trial in Saudi Arabia, with plans for many more treatments in the works. Listen now to learn about the trial, Charles River's incubator program, and about the current state of biotech startup in Saudi Arabia.

Astellas is doubling down on gene therapy while others retreat. Richard Wilson, SVP at Astellas Gene Therapies, joins In Vivo to discuss why the company believes AAV technology will transform medicine despite current industry headwinds.

Imagine a future where diseases like leukemia, sickle cell anemia and multiple sclerosis are no longer life sentences, but treatable and even curable. That is the promise of gene therapy.In this episode, Namritha Ravinder, director of cell biology at Thermo Fisher Scientific, explains the groundbreaking technologies that correct the genetic errors in our cells that lead to disease. These gene therapies may offer permanent solutions with single-dose treatments. Namritha also provides an expert's view on what's to come from more than 2,000 gene therapies in the pipeline, and the innovation needed to make these new treatments accessible to patients.

In this episode of Sg2 Perspectives, Jayme Zage, PhD speaks with leaders from the Children's Hospital of Philadelphia (CHOP), Katherine Helbig, MS, LCGC, AVP of Research Strategy and Accelerator Programs, and Dan Fields, JD, SVP and Chief Strategy Officer, about the evolving landscape of cell and gene therapy. They share compelling patient stories and outline the operational and strategic infrastructure needed to deliver these groundbreaking treatments. From CAR T-cell therapies to life-changing gene therapies for chronic conditions, CHOP's commitment to innovation and sustainability offers insights for organizations looking to enter this dynamic space.   We are always excited to get ideas and feedback from our listeners. You can reach us at sg2perspectives@sg2.com, or visit the Sg2 company page on LinkedIn.

The expanded Phase 1/2/3 shortens time to potential FDA approval.

Joe DePinto, MBA, Head of Cell, Gene & Advanced Therapies at McKesson, joins the show to discuss InspiroGene, McKesson's end-to-end platform built to address the complex delivery, coordination, and commercialization needs of cell and gene therapies. He breaks down how InspiroGene integrates logistics, specialty pharmacy, patient support services, and real-time data tracking to close critical gaps in the CGT ecosystem—especially around why many eligible patients are still not being identified, referred, or treated. He also explores the broader vision: enabling scalable access to these therapies as they move from rare diseases to larger populations, and what it will take—from providers, payers, and pharma—to meet that future. Check out Chadi's website for all Healthcare Unfiltered episodes and other content. www.chadinabhan.com/ Watch all Healthcare Unfiltered episodes on YouTube. www.youtube.com/channel/UCjiJPTpIJdIiukcq0UaMFsA

Synopsis: When an introverted engineer becomes the President & CEO of REGENXBIO, transformation follows. Curran Simpson joins host Rahul Chaturvedi to unpack his unlikely journey from biotech operations to the C-suite—and how that hands-on experience is reshaping gene therapy's future. They dive into the evolution of REGENXBIO's pipeline, tackling ultra-rare diseases like MPS II, ambitious plans for Duchenne Muscular Dystrophy, and commercial partnerships with giants like AbbVie. Curran offers hard-earned leadership lessons, honest reflections on scaling science, and insights into how one-time gene therapies could revolutionize treatment in both rare and common diseases. From clinical nuance to strategic boldness, this is a masterclass in biotech leadership, platform focus, and staying patient-first—no matter how complex the science or market. Biography: Curran M. Simpson is the President and Chief Executive Officer and member of the Board of Directors at REGENXBIO. Mr. Simpson previously served as the Company's Chief Operating Officer. In that role, he led key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operations. Mr. Simpson joined REGENXBIO in 2015 with extensive leadership experience across biopharmaceutical operations and served as the Company's Chief Technology and Operations Officer before becoming COO. Prior to joining REGENXBIO, he was the Regional Supply Chain Head for North America and Interim Chief Operating Officer at GlaxoSmithKline (GSK). Mr. Simpson earlier served as interim CEO of Human Genome Sciences (HGS), where he led the integration of HGS into GSK, and as Senior Vice President of Operations and Vice President of Manufacturing Operations at HGS. Prior to HGS, Mr. Simpson was Director of Manufacturing Sciences at Biogen. Earlier in his career, Mr. Simpson served in an overseas assignment at Novo-Nordisk Biochem in Denmark and in various senior development and engineer roles at Genentech, working on Herceptin and Avastin, among other roles. Mr. Simpson has an M.S. in surface and colloid science from Clarkson University and a B.S. in chemistry from the Clarkson College of Technology.

We love to hear from our listeners. Send us a message.In this episode of Cell & Gene: The Podcast, George Magrath, CEO of Opus Genetics, shares with Host, Erin Harris, how the company is advancing gene therapies for inherited retinal diseases (IRDs), with a focus on LCA5, a rare and severe form of childhood blindness. Built on the foundational science of Dr. Jean Bennett and leveraging the legacy of Luxturna, Magrath shares how Opus is developing subretinal AAV-based therapies targeting seven distinct mutations. He also explains why gene therapy is particularly well-suited to IRDs, shares compelling early clinical data, and discusses the promise of early intervention in preserving vision and enabling proper neural development. Subscribe to the podcast!Apple | Spotify | YouTube

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. A judge has suspended mass layoffs at the Department of Health and Human Services, ruling that the staff cuts are likely unlawful as the agency does not have the authority to make such changes without Congress. In other news, private equity firms have recently acquired Bluebird Bio, a gene therapy biotech company, raising questions about their motivations. Additionally, RFK Jr. is advocating for changes to the vaccine injury compensation program to include COVID-19 vaccines, while the FDA is considering regulatory action on Argenx's Vyvgart Hytrulo due to safety concerns. The article also discusses Gilead's plans for dominance in the HIV market with a new drug, and other updates in the pharmaceutical industry.Private equity firms are increasingly targeting biotech companies, with Bluebird Bio recently being acquired by two firms. The first half of the year saw a surge in M&A activity in the pharmaceutical industry, with big pharmas like Eli Lilly and Novartis making multiple deals. Deals related to Alzheimer's disease also saw a significant increase following the approval of new treatments. Gilead is expanding its presence in the HIV market with a new drug, while GSK is also vying for market dominance. Other notable deals include AbbVie's acquisition of Capstan and Novartis' partnership for cardiovascular disease targets. Overall, the industry is seeing increased activity and interest from private equity firms and big pharma companies.

Welcome to ohmTown. The Non Sequitur News Show is held live via Twitch and Youtube every day. We, Mayor Watt and the AI that runs ohmTown, cover a selection of aggregated news articles and discuss them briefly with a perspective merging Science, Technology, and Society. You can visit https://www.youtube.com/ohmtown for the complete history since 2022.Articles Discussed:Lost in Spaaaaace Satellitehttps://www.ohmtown.com/groups/mobble/f/d/bezos-backed-methane-tracking-satellite-lost-in-space/Drone Based Reforestationhttps://www.ohmtown.com/groups/mobble/f/d/canada-turns-to-drones-for-reforestation-after-wildfires/Gene Therapy Repaired Hearinghttps://www.ohmtown.com/groups/wanted/f/d/deaf-teenager-and-24-year-old-gain-ability-to-hear-after-experimental-gene-therapy/The Seven Year Itchy Shutdownhttps://www.ohmtown.com/groups/hatchideas/f/d/heathrow-shutdown-caused-by-problem-found-seven-years-ago/Starting Skills at a Young Agehttps://www.ohmtown.com/groups/four-wheel-tech/f/d/12-year-old-allegedly-sells-stolen-cars-for-as-little-as-30/A Plane Went for a Divehttps://www.ohmtown.com/groups/four-wheel-tech/f/d/plane-dives-from-36000-to-10000-feet-in-just-10-minutes/Patient Gaminghttps://www.ohmtown.com/groups/warcrafters/f/d/patient-gaming-only-gets-more-attractive-when-70-games-get-heavy-steam-sale-discounts-just-months-after-launch/Linking to Rival Search Platforms in the UKhttps://www.ohmtown.com/groups/technologytoday/f/d/google-may-be-forced-to-link-to-rival-search-platforms-in-the-uk-2/Redesign the Bank of England Banknoteshttps://www.ohmtown.com/groups/hatchideas/f/d/bank-of-england-to-redesign-banknotes-and-wants-your-help/Not My Left Turnhttps://www.ohmtown.com/groups/mobble/f/d/banning-left-turns-could-save-your-commute-your-gas-mileage-and-maybe-your-life/

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.The CDC's new vaccine advisors are meeting with an unexpected agenda today, including discussions on topics such as Sanofi and Gilead's protein degrader deals, the FDA's review of Sarepta's gene therapy, and a contentious hearing with RFK Jr. over vaccines. The meeting marks the beginning of a new era for the influential vaccine committee. The FDA has named a psychedelic proponent as CDER deputy director, while Nektar has declared a Phase IIb win for eczema treatment. Experts say conflicts of interest among axed ACIP members are a "red herring," and the pharma industry is facing turmoil in failed immuno-oncology projects. Prescription drug sales are projected to hit $1.75 trillion by 2030, thanks to GLP-1s. Transitioning to the next news, a major pharmaceutical company has announced a breakthrough in cancer research, potentially changing the landscape of treatment options for patients worldwide. This development comes at a time when the industry is seeing significant advancements in personalized medicine and targeted therapies. Moving on to regulatory updates, the FDA has recently approved a new drug for a rare disease, providing hope for patients who previously had limited treatment options. This decision showcases the agency's commitment to expediting the approval process for innovative therapies that address unmet medical needs. In other news, a biotech startup has secured funding for its groundbreaking technology that aims to revolutionize drug delivery methods. This investment highlights the growing interest in novel approaches to drug development and underscores the importance of innovation in the industry.Wrapping up today's episode, we take a look at the latest trends in digital health, with companies leveraging artificial intelligence and big data analytics to improve patient outcomes and streamline healthcare delivery. These technological advancements have the potential to transform the way healthcare is delivered and pave the way for a more efficient and patient-centric system. That's all for today's episode of Pharma and Biotech daily. Stay tuned for more updates on the latest developments in the pharmaceutical and biotechnology sectors.

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.

In this week's episode, we'll learn more about social determinants of health that impact access to allogeneic hematopoietic cell transplantation in patients with acute myeloid leukemia, or AML; use of megakaryocyte growth factor receptor-based stem cell depletion as part of pretransplant conditioning in ex vivo autologous gene therapy; and identification of an eight-protein risk signature as well as a novel single protein biomarker, soluble oncostatin M receptor, for risk stratification in AML.Featured Articles:Social Determinants of Health and Access to Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid LeukemiacMPL-Based Purification and Depletion of Human Hematopoietic Stem Cells: Implications for Pretransplant ConditioningBlood-Based Proteomic Profiling Identifies OSMR as a Novel Biomarker of AML Outcomes

One of the challenges of delivering gene therapies to the eye is that once a subretinal injection is made, the therapy's distribution is confined to the margins of the pocket of fluid that is created, known as a bleb. Atsena, which is developing gene therapies for X-linked retinoschisis and Leber congenital amaurosis 1, uses its AAV.SPR technology that allows the gene therapy to spread laterally after injection. We spoke to Patrick Ritschel, CEO of Atsena Therapeutics, about the challenges of gene therapies for inherited retinal diseases, how the company's unique vector technology addresses this, and how it allows for safer and more effective delivery of gene therapies to the retina.

From baby blood to breakthrough cures — we explore how unlocking fetal hemoglobin through gene editing (exa-cel/Casgevy) is transforming treatment for sickle cell disease and β-thalassemia.

The tragic death of a second non-ambulatory DMD patient treated with Sarepta's Elevidys gene therapy marks a turning point for the field and should drive all stakeholders to come together to figure out how to safely treat patients with this new modality. On the latest BioCentury This Week podcast, BioCentury's analysts discuss the opportunity FDA, companies and patient advocates have to share data and identify a safe path forward for the gene therapy field, both in DMD and beyond.The analysts also review promising early data for amylin agonists to treat obesity from Metsera and Eli Lilly, and discuss the vision for FDA's future laid out by Commissioner Marty Makary and CBER director Vinay Prasad. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656214#biotech #biopharma #pharma #lifescience #obesity #FDA #DMD00:01 - Sponsor Message: ICON Biotech01:19 - Gene Therapy Safety11:55 - Amylin in Obesity18:17 - FDA's FutureTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Gene therapies are the most expensive drugs in the world. And that is because producing a viral vector for "in vivo" gene therapy is arguably the most complicated process ever attempted at scale in biopharmaceuticals. In a prior video, I talked about the Chinese hamster's revolutionary impact on scaling antibody manufacturing. Today, let's double-click on that (hate this phrase). In this video, we look at the history and complex manufacturing issues of viral vector gene therapy.

Gene therapies are the most expensive drugs in the world. And that is because producing a viral vector for "in vivo" gene therapy is arguably the most complicated process ever attempted at scale in biopharmaceuticals. In a prior video, I talked about the Chinese hamster's revolutionary impact on scaling antibody manufacturing. Today, let's double-click on that (hate this phrase). In this video, we look at the history and complex manufacturing issues of viral vector gene therapy.

In this episode of BioTalk, Amy C. Hay, Chief Business and Strategy Officer at the Cell Therapy Manufacturing Center (CTMC), joins the conversation to explore the evolving landscape of cell and gene therapy. Amy shares insights from her extensive career in oncology care and innovation, highlighting the role CTMC—a joint venture between National Resilience and MD Anderson Cancer Center—is playing in accelerating the transition from discovery to commercialization. She discusses the current state of the industry, what disruption really means in this context, and how new business models can drive stability and impact for early-stage biotech companies. Amy also offers her perspective on how manufacturing must evolve to meet clinical demand, and how CTMC is positioned to lead in this next era of therapeutic development.   Editing and post-production work for this episode was provided by The Podcast Consultant.   Amy C. Hay is the Chief Business and Strategy Officer at the Cell Therapy Manufacturing Center (CTMC), a joint venture between National Resilience and MD Anderson Cancer Center. She brings decades of experience in oncology care, strategic growth, and healthcare innovation to her role, where she leads business strategy, partnerships, and long-term growth initiatives. Prior to CTMC, Amy held leadership roles at Varian (a Siemens Healthineers company), MD Anderson Cancer Center, and several global consulting efforts focused on advancing cancer care. Her career spans work across the U.S. and internationally, with a focus on driving innovation, commercialization, and patient access in complex health systems.

Superpowers for Good should not be considered investment advice. Seek counsel before making investment decisions. When you purchase an item, launch a campaign or create an investment account after clicking a link here, we may earn a fee. Engage to support our work.Watch the show on television by downloading the e360tv channel app to your Roku, AppleTV or AmazonFireTV. You can also see it on YouTube.Devin: What is your superpower?Nicole: Willingness to take bold leaps and embrace the unknown.Imagine a world where cancer is as treatable as the flu. That's the vision Nicole Paulk, the founder and CEO of Siren Biotechnology, is working tirelessly to bring to life. Driven by groundbreaking science, Nicole's company is on the brink of launching clinical trials for a revolutionary cancer treatment.Nicole's work leverages engineered viruses—those that don't make you sick—to deliver genetic medicines directly to cancer cells. These “good viruses” act as a delivery system for anti-tumor drugs, targeting difficult-to-treat cancers like recurrent high-grade gliomas, a fatal brain cancer. “We started to see data that looked really promising in this space,” Nicole explained, adding, “We felt a moral obligation to go after this cancer where there's just this huge unmet need.”Siren Biotechnology's approach is designed to have broad applications. Unlike treatments that target specific genetic mutations, their method has the potential to work on various tumor types. Nicole describes it as using viruses like a “little FedEx delivery truck” that can be engineered to deliver medicine precisely where it's needed in the body.This innovation didn't happen overnight. Nicole, a former professor of virology at UCSF, took the bold step of leaving academia to launch Siren Biotechnology. “I decided to resign from my faculty position and go be the founder and CEO just because I was so excited about the data and what we had so far,” she shared.In addition to traditional venture capital funding, Siren Biotechnology is inviting the public to invest through a regulated investment crowdfunding campaign. This unique opportunity allows patients, families, and supporters touched by cancer to be part of their journey. “We wanted to bring patients and their families onto our cap table,” Nicole said. “It's a way to involve them in the conversation much earlier.”Siren Biotechnology's work could redefine cancer care and inspire a new model for patient-centered innovation. With clinical trials just months away, the future looks brighter for those battling cancer—and for all of us who dream of a world where cancer is no longer a death sentence.tl;dr:Nicole Paulk's Siren Biotechnology transforms viruses into targeted cancer therapies with universal potential.The company focuses on recurrent high-grade gliomas, a fatal brain cancer with no standard treatment.Nicole left academia to lead Siren, leveraging her groundbreaking virology research to launch the company.Siren's unique crowdfunding campaign lets patients and families invest in the fight against cancer.Nicole's superpower, fearless innovation, drives her bold mission to revolutionize cancer care.How to Develop Fearless Innovation As a SuperpowerNicole's superpower is her willingness to take bold leaps and embrace the unknown. After spending nearly two decades building her academic career, Nicole left her position as a virology professor at UCSF to start Siren Biotechnology. She explained, “I feel like my superpower is just kind of being willing to jump off the cliff without a parachute and be like, we're going to figure it out. We're going to make it work.”Nicole exemplified fearless innovation when her research led to a groundbreaking discovery about the biology of a virus, one that required rewriting existing textbooks. Initially met with skepticism, her work was later validated and became a foundation for Siren Biotechnology's cancer treatment. This pivotal moment highlights Nicole's courage to challenge conventional knowledge and pursue transformative ideas.Tips for Developing This Superpower:Be willing to question conventional wisdom and explore new paths.Take calculated risks, even if it means stepping away from a well-defined career path.Embrace discomfort and uncertainty as a necessary part of innovation.Engage others by communicating your vision in relatable and accessible ways.By following Nicole's example and advice, you can make fearless innovation a skill. With practice and effort, you could make it a superpower that enables you to do more good in the world.Remember, however, that research into success suggests that building on your own superpowers is more important than creating new ones or overcoming weaknesses. You do you!Guest ProfileNicole Paulk (she/her):CEO, Founder, President, Siren BiotechnologyAbout Siren Biotechnology: We are combining two transformative therapeutic technologies – AAV gene therapy and cytokine immunotherapy – into a single, reimagined modality that overcomes key challenges and redefines how we destroy tumor cells and elicit anti-tumor immunity.This is the first AAV gene therapy drug product that can be made once and used in numerous indications – a huge leap forward for the field. This drastically reduces clinical development times, manufacturing timelines, and capital needs for each clinical trial. Most importantly, ‘universal' means countless solid tumor cancer patients – regardless of tumor type or mutations – may benefit from this breakthrough approach.Website: sirenbiotechnology.comX/Twitter Handle: @SirenBioCompany Facebook Page: facebook.com/SirenBiotechnologyOther URL: wefunder.com/siren.biotechnologyBiographical Information: Dr. Nicole Paulk is the CEO, Founder, and President of Siren Biotechnology and has dedicated her career to advancing the field of gene therapy. With nearly two decades of expertise, Nicole has been at the forefront of developing cutting-edge advances to propel the field of gene therapy forward for a wide range of diseases.Before founding Siren, Nicole held various leadership positions in academia and industry and most notably was an Assistant Adj Professor of AAV Gene Therapy in the UCSF Department of Biochemistry & Biophysics before leaving to found Siren. Nicole has a B.S. in Medical Microbiology, a Ph.D. in Viral Gene Therapy and Regenerative Medicine from OHSU, and completed her Postdoctoral Fellowship and Instructorship in Human Gene Therapy at Stanford University prior to starting her lab at UCSF. Nicole is a pioneer in the development of next-generation AAV platforms for gene repair, gene transfer and gene editing, directed evolution for novel engineered capsid evolution, and comparative multi-omic approaches to interrogate translational AAV biology.Nicole is a renowned expert in gene therapy and has consulted extensively for big pharma, written draft CMC guidance for the FDA, and sits on the Scientific Advisory Boards for Sarepta, Astellas, Metagenomi, Dyno Therapeutics, CEVEC, GRO Biosciences, Excision BioTherapeutics, WhiteLab Genomics, Johns Hopkins Gene Therapy Initiative, the Gene Therapy for Rare Disorders Searchlight Program, and several stealth startups. She has been quoted in The Wall Street Journal, The Economist, The Boston Globe, Endpoints, STAT, Phacilitate, GEN, BioPharma Dive, Evaluate Vantage, SF Business Times, WIRED, Drug Discovery World, MIT Tech Review, C&EN, and more. She sits on the Scientific Editorial Boards of the journals Gene Therapy, Human Gene Therapy, and Biopharma International Gene Therapy. She is the Chair of the American Society of Gene and Cell Therapy (ASGCT) Translational Science Committee, and a member of the ASGCT Cancer Cell and Gene Therapy Committee and the Biocom California Cell and Gene Therapy Committee. She has invented numerous AAV gene therapy technologies that have been shared or licensed to dozens of gene therapy companies and nonprofit groups working in rare diseases.Outside of work, you can find Nicole adventure traveling (think whitewater rafting meets backcountry trekking), snowboarding, planning elaborate Halloween parties complete with animatronics and ghoulish menus, tending her vegetable garden, and obsessing over the latest wearable gadgets. If you're trying to track her down at a conference and can't find her, it's because she snuck off to an oyster bar.X/Twitter Handle: @Nicole_PaulkLinkedin: linkedin.com/in/nicolepaulkInstagram Handle: @sirenbioSupport Our SponsorsOur generous sponsors make our work possible, serving impact investors, social entrepreneurs, community builders and diverse founders. Today's advertisers include FundingHope, Kingscrowd and Crowdfunding Made Simple. Learn more about advertising with us here.Max-Impact MembersThe following Max-Impact Members provide valuable financial support:Carol Fineagan, Independent Consultant | Lory Moore, Lory Moore Law | Marcia Brinton, High Desert Gear | Paul Lovejoy, Stakeholder Enterprise | Pearl Wright, Global Changemaker | Ralf Mandt, Next Pitch | Scott Thorpe, Philanthropist | Matthew Mead, Hempitecture | Michael Pratt, Qnetic | Sharon Samjitsingh, Health Care Originals | Add Your Name HereUpcoming SuperCrowd Event CalendarIf a location is not noted, the events below are virtual.Impact Cherub Club Meeting hosted by The Super Crowd, Inc., a public benefit corporation, on June 17, 2025, at 1:00 PM Eastern. Each month, the Club meets to review new offerings for investment consideration and to conduct due diligence on previously screened deals. To join the Impact Cherub Club, become an Impact Member of the SuperCrowd.SuperCrowdHour, June 18, 2025, at 12:00 PM Eastern. Jason Fishman, Co-Founder and CEO of Digital Niche Agency (DNA), will lead a session on "How to Spin $1 of Advertising into $10!" He'll reveal proven strategies and marketing insights drawn from years of experience helping successful crowdfunding campaigns. Whether you're a founder planning a raise or a supporter of innovative startups, you'll gain actionable tips to boost visibility, drive engagement, and hit your funding goals. Don't miss it!Join us on June 25, 2025, at 8:00 PM Eastern for the Superpowers for Good Live Pitch—streaming on e360tv, where purpose-driven founders take the virtual stage to present their active Regulation Crowdfunding campaigns to a national audience of investors and changemakers. Selected startups are chosen for their commitment to community, alignment with NC3's Community Capital Principles, and their drive to create real-world impact. Thanks to sponsors DNA and DealMaker, this event is free to watch and amplifies the voices of underrepresented and mission-aligned entrepreneurs. Don't miss this inspiring evening where capital meets purpose—tune in to discover and support the next wave of impact-driven innovation.SuperCrowd25, August 21st and 22nd: This two-day virtual event is an annual tradition but with big upgrades for 2025! We'll be streaming live across the web and on TV via e360tv. Soon, we'll open a process for nominating speakers. Check back!Community Event CalendarSuccessful Funding with Karl Dakin, Tuesdays at 10:00 AM ET - Click on Events.Devin Thorpe is featured in a free virtual masterclass series hosted by Irina Portnova titled Break Free, Elevate Your Money Mindset & Call In Overflow, focused on transforming your relationship with money through personal stories and practical insights. June 8-21, 2025.Join Dorian Dickinson, founder & CEO of FundingHope, for Startup.com's monthly crowdfunding workshop, where he'll dive into strategies for successfully raising capital through investment crowdfunding. June 24 at noon Eastern. Regulated Investment Crowdfunding Summit 2025, Crowdfunding Professional Association, Washington DC, October 21-22, 2025.Call for community action:Please show your support for a tax credit for investments made via Regulation Crowdfunding, benefiting both the investors and the small businesses that receive the investments. Learn more here.If you would like to submit an event for us to share with the 9,000+ changemakers, investors and entrepreneurs who are members of the SuperCrowd, click here.We use AI to help us write compelling recaps of each episode. Get full access to Superpowers for Good at www.superpowers4good.com/subscribe

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The American Medical Association is calling for a Senate probe into RFK Jr. after he removed all 17 members of the CDC's vaccine advisory board. They are also urging an immediate reversal of the HHS Secretary's decision. In other news, FDA leaders promise support for gene therapy as it faces challenges, Novo Nordisk invests over $800 million in discovering oral obesity drugs, and a new company, InVitro Cell Research, focuses on interventions to slow aging and prevent age-related diseases. FDA Commissioner Marty Makary and CBER Director Vinay Prasad published an article outlining the FDA's priorities, including accelerating cures and deploying artificial intelligence quickly. Other news includes Lilly's muscle-preserving treatment pact, Vertex laying off staff after axing a diabetes asset, and Genentech reshuffling in South San Francisco. Recursion cuts workforce by 20%.FDA leaders have pledged support to gene therapy makers as the industry faces challenges in commercialization. Despite huge valuations, gene therapies are struggling to reach the market. Poor communication about the value of groundbreaking treatments is hindering their prospects. Right of first negotiation deals do not always lead to mergers and acquisitions, as found by Jefferies in their analysis of licensing deals. Trilink's new comprehensive IVT kit simplifies the production of mRNAs. In other news, Lilly strikes a $650 million deal for a muscle-preserving treatment, Odyssey ends its quest for Nasdaq, BMS expands its radiopharma presence, and SpliceBio secures $135 million in funding for protein splicing medicines. Novo ups its obesity efforts with an $800 million pact, while Cullinan makes a $700 million deal for autoimmune T cell engager in China.Thank you for listening to Pharma and Biotech Daily.

In honor of Molecular Therapy’s 25th anniversary, this episode is the second in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years. In this episode, Dr. Glorioso will discuss the reviews, AAV vector development, back to the future, with author Sheila Mikhail; M34, Inc.; and The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy; with author Dr. Mark Kay, Stanford University. If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

This episode focuses on the operational considerations for the use of in vivo and viral based gene therapies in health-system pharmacies and the appropriate stakeholders. CE for this episode expires on May 31, 2027.   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.

This episode is brought to you by State & Liberty, Blokes & Joi, Fatty15 and FLYKITT. Bbiochemist and biomolecular therapy expert Jay Spall joins us to unravel his transformative journey from spinal injury to becoming a leading figure in the world of biohacking and longevity. Jay shares groundbreaking insights into follistatin therapy, detailing its impressive effects on endurance, body composition, and overall well-being. His personal narrative, combined with scientific research and anecdotes, paints a vivid picture of how cutting-edge therapies can enhance human performance and extend the quality of life. Follow MiniCircle @minicircledna Follow Chase @chase_chewning ----- In this episode we discuss... (00:01) Introduction to Biochemical Therapy (10:07) Reclaiming Our Health Through (17:30) Hormone Optimization Therapies (32:09) Modern Progressive Interventions (42:28) Taking Healthcare Power Back by Our Choices (54:49) Neurological Therapy (01:07:45) Gene Therapy for Health and Longevity (01:14:55) How to Actually Measure Longevity (01:22:14) Maximizing Healthspan and Lifespan With Gene Therapy (01:31:09) Why Community Matters (01:36:05) Connecting With MiniCircle ----- Episode resources: Save 15% on the best-fitting men's clothes with code CHASE at https://www.StateAndLiberty.com  Save 10% on any diagnostic labs with code CHASE at https://www.JoiAndBlokes.com  Save an additional 15% on the 90-day starter kit of C15:0 essential fatty acid with code EVERFORWARD at https://www.Fatty15.com/everforward  Never get jet lag again and save 15% with code CHASE at https://www.FLYKITT.com  Watch and subscribe on YouTube Learn more at MiniCircle.io