Podcasts about Gene therapy

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. 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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

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.

A gene therapy clinical trial for Danon disease run by Rocket Pharmaceuticals has been suspended following the death of a patient. MIT and Recursion have released Boltz-2, an open-source tool that predicts molecular binding affinity at newfound speed and accuracy, aiming to democratize commercial drug discovery. Intellia Therapeutics saw its stock nosedive late last week after a patient in its Phase III trial of the CRISPR-based therapy experienced non-lethal liver toxicity. In other news, a therapeutic plasma exchange regimen combined with intravenous immunoglobulin reduced some biomarkers of biological age in humans. Sanofi has agreed to acquire Blueprint Medicines for up to $9.5 billion, and a universal CAR T cell therapy was produced by applying CRISPR gene editing to cells donated by healthy individuals. Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.  Listed below are links to the GEN stories referenced in this episode of Touching Base Danon Disease Patient Dies in Rocket Gene Therapy Trial By Alex Philippidis, GEN, May 27, 2025 Boltz-2 Released to Democratize AI Molecular Modeling for Drug Discovery By Fay Lin, PhD, GENEdge, June 6, 2025 StockWatch: Intellia Stumbles on News of Patient's Severe Liver Toxicity By Alex Philippidis, GEN, May 30, 2025 The State of CRISPR Summit Registration https://bit.ly/CRISPR25 Plasma Exchange in Humans Reduces Biological Age by Over Two Years GEN, June 1, 2025 Sanofi to Acquire Blueprint Medicines for Up to $9.5B By Alex Philippidis, GEN Edge, June 2, 2025 "Universal" CAR-T Immunotherapy Targets Relapsed/Refractory Blood Cancers GEN, May 30, 2025 Hosted on Acast. See acast.com/privacy for more information.

During the 2025 Duke AVS Course, Duke faculty sat down with several key lecturers to discuss their presentations. Avni P. Finn, MD, MBA, discussed her experiences delivering subretinal gene therapy, David Zacks, MD, PhD, explored important factors in retinal detachment repair, Xi Chen, MD, PhD, highlighted the various surgical approaches for young patients who present without a posterior vitreous detachment, and Yannek I. Leiderman, MD, PhD, provided pearls for scleral fixation of secondary IOLs. 

In this episode of SCW for Pharma host Evren Ozkaya sits down with Audrey Greenberg, CEO and Founder of AG Capital Advisors, to explore the evolving landscape of CDMOs in the cell and gene therapy space.Audrey, who brings a finance background and deep industry insight, outlines what it takes to build and scale a CDMO business—from managing high fixed costs and staffing GMP operations to raising capital and building trust. She explains why earning the first million dollars in this space is often the hardest, and how their end-to-end manufacturing model, housed under one roof, simplifies pharma supply chains and reduces complexity for clients.Evren and Audrey explore the headwinds facing the industry, including geopolitical uncertainty, tariffs and capital scarcity. Audrey also highlights that, for gene and cell therapies, supply is currently outpacing demand. Despite the current uncertainties, she remains optimistic about company valuations and expects a rebound in the second half of 2025. In the meantime, she argues, digital transformation is not optional — it is the foundation for resilience and future valuation.They dive into how data, automation, and AI improve efficiency, reduce errors, and increase revenue. In a high-skill, high-cost environment—where PhDs often run the production floor—robotics and standardization become essential. Audrey also makes the case that digital maturity is more than an operational upgrade: it enhances customer trust, boosts EBITDA, and improves a CDMO's standing in an M&A-driven market.The conversation also touches on reshoring and the role of digitalization in helping the U.S. expand capacity without spiking drug prices. Audrey notes that reshoring began before COVID-19 but has gained urgency with recent tariff policies.The episode wraps with career advice for rising professionals: explore broadly, stay flexible, and understand both the operational and commercial sides of the business. Adaptability, Audrey says, is the most valuable skill in today's pharma world.

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.

Despite the long holiday weekend, news in biopharma never slows down. In this week’s episode of "The Top Line," the Fierce team breaks down some of the biggest stories from the past week. On the pharma side, Eric Sagonowsky and Kevin Dunleavy examine Big Pharma’s first-quarter 2025 performance. While most of the top 25 companies are still delivering strong sales growth despite emerging uncertainties in Washington, D.C., a few major players are starting to see a slowdown. Each company tells a different story, and Sagonowsky and Dunleavy dive into the nuances of the current commercial landscape. Later, Fierce Biotech's Gabrielle Masson and Darren Incorvaia highlight key data from the American Society of Gene & Cell Therapy conference and preview what the team is watching at this week’s American Society of Clinical Oncology annual meeting. To learn more about the topics in this episode: Seven top pharmas posted revenue declines in Q1. The common thread? All are US firms Atsena eye disease gene therapy hits safety goals, closes retinal splits in phase 1/2 ASGCT: Analysts see Rocket gene therapy setting 'a new bar' for efficacy in heart condition Rocket crashes as gene therapy patient dies, FDA imposes hold This episode is sponsored by Cencora. See omnystudio.com/listener for privacy information.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.## Intellia reported an adverse event in its gene therapy trial, causing its stock to drop, adding to existing safety concerns in gene therapy. Analysts downplayed the event, but patient deaths in other trials have raised red flags.## Moderna lost a $760 million bird flu vaccine contract amid uncertainties surrounding its COVID-19 vaccine. ## Other news includes Iteos closing down after a failed trial, Merck and Daiichi Sankyo pulling a lung cancer filing, and Novo opposing pharma tariffs.## AGC Biologics will be at Bio International to discuss their capabilities in drug production. ## Additional stories cover Rocket's gene therapy hold, Biogen's strategy shift, and AbbVie's staff cuts.

In this latest episode of the BroadEye podcast, hosts Shawn Maloney and Dr. Bruno Fernandes sit down with Dr. Arshad M. Khanani, MD, MA, FASRS, a pioneer in vitreoretinal gene-therapy trials and Managing Partner at Sierra Eye Associates. Dr. Khanani shares front-line insights into how genetic medicines are reshaping treatment for inherited and degenerative retinal diseases, highlighting both the promise and the practical challenges of bringing one-time therapies to patients.   Why Gene Therapy Matters Today Recent years have seen the first US approvals of in-vivo retinal gene therapies, beginning with voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophy in December 2017. These one-off treatments offer durable vision improvements where none existed before, but they also raise questions of long-term safety, vector delivery, and equitable access.   Dr. Khanani's Journey With over a decade leading surgical trials in sustained-delivery and viral-vector therapies, Dr. Khanani has overseen early human work on next-generation agents—including subretinal injections of AAV vectors and novel capsid designs. He describes lessons learned from Phase 1 safety studies through global registrational trials, emphasizing iterative “vector evolution” to boost efficacy and minimize inflammation.   Key Takeaways Vector Optimization: Directed-evolution platforms are accelerating discovery of capsids that target photoreceptors more precisely, reducing off-target toxicity. Durability vs. Dose: Striking the right balance between transgene expression and immune activation remains central—higher doses don't always equal better outcomes. Regulatory Pathways: While Luxturna set a precedent, streamlined FDA and EMA guidance on trial endpoints and real-world evidence will shape the next wave of approvals. Cost & Access: Innovative payment models (e.g., outcomes-based installment plans) are emerging to address the multi-hundred-thousand-dollar price tags of these therapies.   About the Guest Dr. Arshad M. Khanani, MD, MA, FASRS, is a vitreoretinal surgeon, researcher, and Managing Partner at Sierra Eye Associates in Reno, Nevada. He holds dual degrees in medicine and medical humanities from the University of Nevada, Reno, and is a fellow of the American Society of Retina Specialists. Dr. Khanani has authored over 100 peer-reviewed articles on retinal disease and served as principal investigator on more than 30 clinical trials of gene and cell therapies for inherited and age-related retinal disorders. He also lectures internationally on surgical innovation, vector design, and the economics of advanced ocular treatments.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Former Chinook board member, Rouzbeh Haghighat, has been indicted for insider trading related to Novartis' $3.2 billion acquisition of the company. Despite this scandal, pharma R&D spending increased in 2024, climbing 1.5% across the global pharmaceutical sector. The acquisition of SiteOne by Lilly follows Vertex into the non-opioid pain space, providing diversification for Lilly, which has been focusing on obesity and diabetes treatments. Meanwhile, AbbVie's Allergan cuts over 200 staff after a botched marketing campaign, and Inflarx axes a rare skin disease study due to disappointing late-stage data.AGC Biologics will be at Bio International in Boston to showcase their global capabilities in drug production. Vaccine overhaul, rocket grounding, and drug price transparency are also highlighted in the latest news. Biogen's strategy for Zurzuvae shifts as obstetricians/gynecologists rise to the front lines. Drug price transparency in the US is discussed as being easier said than done. Additionally, Rocket's gene therapy for Danon disease is on hold after a patient death, and four biotechs are facing uncertainty in the COVID-19 vaccine landscape.Global pharmaceutical companies are increasing their research and development spending despite political and economic challenges. Biogen is shifting its strategy for the drug Zurzuvae as obstetricians and gynecologists become more involved. Drug price transparency in the US is still a challenge, despite efforts to increase transparency. Trilink has introduced a new poly(A) tail modification to enhance protein expression.In other news, a former Chinook board member has been indicted for insider trading, Trump has appointed Dr. Oz to lead drug pricing negotiations, and Lilly is following Vertex into non-opioid pain treatment with a SiteOne acquisition. Sanofi has purchased Vigil for $470 million to reignite an Alzheimer's target.

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Fixing Gene Therapy Access With AI | How Genoplex Is Helping Patients Get Treated Faster #genetherapy #biotech #healthcareaiWhat if gene therapy could cure your disease, but you never made it to the treatment center in time? In this episode of Biotech Bytes, we dive into that real and urgent problem with Christopher Leidli, CEO of Genoplex.ai. Chris shares how his company uses AI to match patients with advanced therapies and treatment centers faster than ever before. Please visit our website to get more information: https://swangroup.net/ We talk about the logistics, the outdated systems, and the patient pain points that make accessing cutting-edge gene and cell therapies so difficult. From working at biotech giants like Amgen and J&J to launching a startup to bridge the access gap, Chris has seen every side of this challenge.You'll learn the difference between gene therapy, cell therapy, and T-cell therapy, the current FDA-approved treatments, and why health equity is still a major barrier. We also get into how Genoplex's platform works and why AI could be the key to helping patients get lifesaving care when it matters most.

Welcome back to another eye-opening episode of Ditch the Lab Coat with Dr. Mark Bonta! While we usually dig into the science behind medicine's biggest questions, on this episode, Dr. Mark is joined by the extraordinary Dr. Erwin Loh — a powerhouse at the intersection of medicine, law, healthcare leadership, and medical futurology. Dr. Loh is not only the President of the Royal Australasian College of Medical Administrators and National Director of Medical Services for Calvary Healthcare in Australia, but he's also a trusted voice on LinkedIn, where he untangles medical breakthroughs and ethical dilemmas for his global audience on the daily (sometimes 5 to 10 times a day!)—all while juggling life as an executive and dad.Together, Mark and Erwin tackle some of the most pressing—and headline-worthy—topics in medicine today. They dig into Long Covid: what it is, why it's not just "all in your head," and why the recognition of this condition is also reshaping how we think about elusive illnesses like chronic fatigue syndrome and fibromyalgia. If you've wondered what's really happening with the mysterious gut microbiome, the resurgence of old viruses, or the risks of misinformation in today's media chaos, you'll find answers grounded in the latest evidence, not just the loudest opinions.But that's not all—they venture into the weird and wild wonders of scientific progress, from the looming issue of plastics in our cells to CRISPR gene editing and the promises (and perils) of AI in healthcare. Along the way, you'll hear why microplastics might be the “asbestos of our age,” how mixing vaccines can make you a lightning rod for online trolls, and why being cautiously optimistic about humanity's next steps—despite climate change, pandemics, and deepfake bioweapons—is not just reasonable, it's necessary.Whether you're a science junkie, a future-watcher, or simply want a hopeful, no-nonsense take on where medicine is headed (and how it affects your life), this episode delivers smart, ethical, and accessible conversation. Pour yourself something strong, turn your curiosity up to eleven, and get ready for a tour de force of myth-busting, insight, and inspiring optimism from two voices at the cutting edge of healthcare.Tune in for the kind of honest, evidence-based, and thought-provoking discussion you won't find anywhere else—right here on Ditch the Lab Coat!Dr Loh's Links( https://www.linkedin.com/in/erwinloh/ )( https://x.com/erwinloh )Episode HighlightsLong Covid: Real and Varied Long Covid is a legitimate, multifaceted syndrome. Most cases improve within two years, but symptoms and underlying mechanisms differ widely.Post-Viral Syndromes Aren't New Long-term illness after viral infections, like after influenza or Epstein Barr, has long existed—Covid just spotlighted this issue.Chronic Illness Recognition Grows Long Covid research is giving more credibility to conditions like chronic fatigue syndrome and fibromyalgia within the medical community.Silent Illnesses Challenge Healthcare Illnesses without definitive biomarkers, such as Long Covid, are harder to diagnose and treat, often leading to patient stigma.Microbiome's Expanding Influence Our gut, skin, and oral microbiomes profoundly affect physical and mental health, though much remains to be discovered about exactly how.Viruses and Chronic Disease Links Viruses may contribute to diseases like multiple sclerosis, cancers, and possibly Alzheimer's, highlighting new frontiers in research.Gene Editing: Transformative Power CRISPR technology lets us precisely edit human genes, promising cures for some diseases but raising major ethical and safety concerns.AI: Double-Edged Healthcare Tool AI accelerates medical discovery and innovation but can also enable harmful outcomes, including creation of bioweapons or misinformation.Plastics: Ubiquitous Unknown Threat Microplastics are everywhere, even in our cells. The true health risks are not fully understood but raise serious environmental and biological questions.Episode Timestamps4:22 – Long Covid: Global Challenge and Uncertainty9:31 – Recognizing Long Covid's Impact10:47 – Exploring Long Covid Treatments16:39 – Unknown Frontiers in Science18:22 – Understanding Microbiome and Disease Dynamics21:04 – COVID Vaccination Journey and Hybrid Immunity24:42 – LinkedIn: Fewer Trolls, Richer Conversations29:04 – Gene Therapy via Phage Infections31:58 – Genetic Correction: Hope and Ethics36:54 – AI and Humanity's Existential Challenges41:14 – Future of Microplastics and Society44:24 – AI, Cognition, and Future Governance46:28 – "Medical Futurology with Irwin Lowe48:31 – "AI in Medicine: Trust MattersDISCLAMER >>>>>>    The Ditch Lab Coat podcast serves solely for general informational purposes and does not serve as a substitute for professional medical services such as medicine or nursing. It does not establish a doctor/patient relationship, and the use of information from the podcast or linked materials is at the user's own risk. The content does not aim to replace professional medical advice, diagnosis, or treatment, and users should promptly seek guidance from healthcare professionals for any medical conditions.   >>>>>> The expressed opinions belong solely to the hosts and guests, and they do not necessarily reflect the views or opinions of the Hospitals, Clinics, Universities, or any other organization associated with the host or guests.    Disclosures: Ditch The Lab Coat podcast is produced by (Podkind.co) and is independent of Dr. Bonta's teaching and research roles at McMaster University, Temerty Faculty of Medicine and Queens University. 

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.A patient death has led to the halt of Rocket's gene therapy trial for Danon disease, due to complications from an acute systemic infection. This incident highlights the unpredictable nature of gene therapies and may pose challenges for investment in the field. On the other hand, mRNA technology has been both helped and hindered by the COVID-19 pandemic. While the pandemic has increased interest and funding in mRNA research, vocal skeptics of COVID-19 vaccinations have negatively impacted the reputation of mRNA technology. Despite this, some see promise in mRNA for therapeutics in cancer and rare diseases.In other news, Prothena is considering business options after a failed phase III trial, and a new biotech company has been launched to focus on neuro diseases. Additionally, AGC Biologics will be at Bio International to showcase their capabilities in protein biologics and advanced therapies.mRNA technology played a significant role in combating COVID-19, but is now facing scrutiny due to vaccine skeptics and cuts in government funding for research. Despite this, the pandemic accelerated advancements in mRNA therapeutics for cancer and rare diseases. The lack of FDA-approved treatments for rare diseases, particularly in children, highlights a critical need for innovation in this area. The American Association for Cancer Research's annual conference will unveil data that could impact cancer treatment, while the future of vaccine development faces challenges amidst regulatory changes and funding issues. Additionally, advancements in cell and gene therapy are on the horizon. Suggestions for coverage topics in the biopharma industry are welcomed.

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My first solo episode, i talk about mostly Lilo and Stitch and i did not want to spoil most of thunderbolts so i kept it short and sweet. Gave my reviews and that is really all for this video. Follow @genetherapypod on Instagram.

Headlines have announced that sickle cell disease can be cured with gene therapy. Now there's a better way developed at Johns Hopkins and other centers around the world, using what are called ‘half-matched donors,' to give new bone marrow to … A new technique is better than gene therapy for curing sickle cell disease, Elizabeth Tracey reports Read More »

Gene therapy for sickle cell disease has a breathtaking price tag of 2-3 million dollars per person, and for some people who receive it, is doesn't actually cure the condition. That's according to Richard Brodsky, a sickle cell expert at … Gene therapy for sickle cell disease often isn't a cure, Elizabeth Tracey reports Read More »

This episode reviews the viral replication process, common viral vectors for in vivo gene therapy, and Food and Drug Administration-approved therapies. The episode will also highlight novel in vivo gene therapies in the development pipeline. CE for this episode expires on May 25, 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.

In this episode of the VJHemOnc podcast you will hear from Akshay Sharma, MBBS, MSc, St. Jude Children's Research Hospital,... The post Gene therapy in sickle cell disease: patient selection, potential complications, & more appeared first on VJHemOnc.

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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 

A baby born with a rare, life-threatening genetic disease is now thriving after receiving an experimental gene-editing treatment. - 米国のKJ（ケイジェイ）・マルドゥーンちゃんは、CPS1欠損症という命にかかわる稀な遺伝性疾患を持って生まれてきました。緩和ケアを含む今後が話されていた折、最新技術を使った遺伝子編集（gene-editing）治療を行うことになり希望が見えてきました。

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.

Baby KJ Muldoon was born with a rare genetic condition that is often fatal, but doctors used custom CRISPR gene therapy to target the exact mutation in his DNA. His family shares their emotional journey in their first TV interview with CBS News. Singer Cassie Ventura faced intense cross-examination in Sean "Diddy" Combs' sex crimes trial, with defense attorneys asking her to read aloud past text messages. Jericka Duncan reports on the disturbing details.In this week's "Kindness 101," Steve Hartman and his children revisit the story of a teenage athlete who started giving back to his community after a small suggestion inspired a profound shift in his outlook on life.In honor of Mental Health Awareness Month, Amazon Books Editorial Director Sarah Gelman shares titles focused on self-care, emotional wellness and navigating complex relationships. To learn more about listener data and our privacy practices visit: https://www.audacyinc.com/privacy-policy Learn more about your ad choices. Visit https://podcastchoices.com/adchoices

Despite missing the primary endpoint, the treatment improved vision for some patients.

At-home cervical cancer screening device gains clearance; the FDA indicates it wants to remove pediatric fluoride products from the market; the chikungunya vaccine is put on-pause for some individuals; a subcutaneous autoinjector is approved to treat migraines; and the FDA will review a gene therapy for Hunter syndrome.

Part B of the trial will enroll nine adult patients as well as three pediatric patients.

The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.

In this episode of the Pain and Performance Podcast, I sit down with Mac Davis, the founder of Mini Circle, a cutting-edge company revolutionizing gene therapy with mini-circle plasmids. If you've ever wondered how science is pushing the boundaries of longevity, muscle growth, and inflammation control; this conversation is a must-listen. "Health feels better than convenient."What Makes Mini-Circle Plasmids Different?Most gene therapy sounds like sci-fi, but Mini Circle's approach is different; safer, cleaner, and non-invasive to your DNA. Unlike traditional methods that can alter your genetic code, mini-circle plasmids work alongside your body's natural systems without changing your original blueprint.No DNA modification – Just temporary instructions that help your body heal and optimize itself.Delivered into fat tissue – Simple injections (no scary viral vectors).Focused on real-world results – From reducing chronic pain to boosting muscle growth.The Power of Folistatin: More Than Just MuscleFolistatin isn't some synthetic lab chemical; it's a natural human hormone that regulates muscle growth, fat metabolism, and even bone health. But its real superpower? Fighting chronic inflammation, one of the biggest hidden killers in modern health.Patients with stubborn joint pain and injuries report dramatic improvements after Folistatin therapy.It's not just for athletes; anyone dealing with age-related muscle loss or inflammation could benefit.Clotho: The "Magic" Longevity GeneIf you haven't heard of Clotho, pay attention. This gene therapy is showing insane potential for:Cognitive function – Patients describe "brain fog" lifting within weeks.Kidney health – A major factor in how well we age.Overall vitality – Some say they feel "like their younger selves" again.One of the most striking takeaways? A patient who said:"This is how I was before. This is me."Stacking Therapies for Maximum ImpactWhy stop at one therapy when you can combine them? Gene therapy stacking (like using Folistatin + Clotho) could be the next big leap in health optimization. Think of it like building a personalized longevity protocol—tailored to your body's needs.People in their 50s and 60s regaining muscle and energy levels they hadn't seen in decades.Individuals prioritizing quality of life over just adding years.As Mac puts it: "People deserve to be healthy."Links: DERRICKTikTok:https://www.tiktok.com/@drderrickInstagram:https://www.instagram.com/derrickbhines/Youtube:https://www.youtube.com/@DrDerrickMac Davis:Website: https://minicircle.io/ Instagram:https://www.instagram.com/minicircledna/

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novo Nordisk predicts a brighter future for Wegovy with the end of the semaglutide shortage, but analysts remain skeptical as Eli Lilly's Zepbound gains ground in sales. The new HHS vaccine requirement has raised questions about its true intentions, with leading vaccine physician Paul Offit criticizing the lack of clarity. Meanwhile, pharmaceutical imports from Ireland are on the rise, biotech stocks fell after Vinay Prasad was named to succeed Marks at CBER, and Lotte Biologics' ADC facility in Syracuse offers end-to-end services for antibody manufacturing. CRISPR's Casgevy is gaining traction with more gene therapy proof of concept expected in 2025, impacting M&A and IPOs in the biotech industry. Summit Pharmaceuticals is nearing the first global phase III data for Keytruda, set to lose exclusivity in 2028 and potentially face competition from biosimilars. A report suggests that low-price drug nations are benefiting from US innovation, undervaluing innovative medicines by 90%. VC financing in biopharma declined by 20% in Q1, but megarounds kept the median deal size high. M&A and IPOs faced challenges due to policy issues, leading to an increase in licensing deals. Novo vows to improve market access for Wegovy, while Trump orders FDA to ease US plant expansion and increase inspections of foreign facilities. BMS pledges a $40 billion investment in the US, Novartis makes M&A moves, and Lilly remains unfazed by CVS's decision to side with Novo in the obesity market battle. Some drugmakers are stockpiling products in the US amid Trump's trade war. NGM terminates half of its staff as its lead asset moves through a registrational study. Job opportunities in clinical quality assurance audit, RBQM central statistical monitoring, and field medical capabilities are available.

We review grog, we took some breaks and never addressed them hence the weird cuts sometimes. We talk older video games, i make funny and gross jokes she does not appreciate. Its all fun and games.

Drug development activities focused on the rare, neuromuscular condition Duchenne muscular dystrophy have translated into functional improvements and an extension of life expectancy. Regenxbio is among several companies pursuing a gene therapy to treat Duchenne. Regenxbio believes both its microdystrophin—a truncated form of the dystrophin gene­ small enough to fit in a vector­—and the vector it is using, give it a competitive advantage over other efforts. We spoke Curran Simpson, CEO of Regenxbio, about the company's platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.

In this episode Gene and his Girlfriend talk about so much stuff that i forgot it all and i think you should just listen to the podcast maaannn, check it out.

Jake Lesnik, Co-Founder and CEO of DM Therapeutics, joins EisnerAmper's TechTalk host Janina Teoxon to discuss how his gene therapy company developed a novel, non-viral platform designed to improve the efficacy, safety, and accessibility of gene therapies for inherited diseases. In this episode, discover how its non-viral Dark Matter DNA™ technology overcomes the limitations of viral vector-based gene therapies by providing long-lasting therapeutic effects and the capacity to deliver larger, more complex genes. Tune in to learn more about the company's mission to turn its breakthrough into new medicines and curative therapies that could expand the range of treatable inherited diseases.

This week's expert, Hepatologist and Key Opinion Leader Scott Friedman, joins Roger to discuss advances in acceptance of gene therapy and knowledge in other areas of basic liver science. When discussing science, he pays particular attention to findings on the diversity of stellate cells and his interest in CAR-T as a therapy for liver disease.This conversation starts with Scott discussing gene therapy. Specifically, he applauds the idea that gene therapy is  becoming accepted in many diseases after a faulty start years ago, due to an unfortunate patient death in a badly controlled trial.  He comments that this acceptance has unique benefits in liver disease because the liver can regenerate so much faster and more efficiently than other organs. He mentions some of the rare liver diseases in which patients are benefiting from gene therapy, and notes that we now have gene therapies and early-stage trials to target PNPLA3 and  other genes associated with MASH and MASH cirrhosis. Next, Scott discusses stellate cells, which he has discussed in earlier episodes of SurfingMASH. Science is increasingly demonstrating how many different types of heterogeneous stellate cells exist. As Scott puts it, these cells "come in many flavors," each of which plays a different role in cell generation or cell death. In fact, the specific therapeutic challenges that present themselves may vary as a patient moves along the pathway from F1 to F2 to F3 to F4. Further, we are learning that there may be several different forms of MASH to present differently at a cellular level. This makes tremendous sense, given that no one drug has proven successful in even a significant majority of patients yet. As the conversation winds down, Scott shares what he describes as a "sobering note" about the state of research funding in America in 2025. As he notes, there are certain kinds of applied and developmental research that private companies do well, but other kinds of basic research that only occur when funded in public and not-for-profit sectors. As a specific example, he cites CRISPR, initially funded publicly and now in the hands of biotech companies, which is used to treat a variety of diseases more effectively than they could have been treated before, if at all. He also comments that a poor early commercial decision slowed the development of statins. 

In this episode of Tales From the Heart host Lisa Salberg speaks with Dr. Steve Ommen about HCM: Family Screenings, Genetics + Gene Therapy.   This conversation was recorded Apr. 18, 2025.

Are new solutions for pediatric hearing loss finally within earshot? In this episode of the Back Table ENT Podcast, we talk with Dr. Daniela Carvalho, pediatric otolaryngologist and director of the Cochlear Implant Program at Rady Children's Hospital in San Diego. --- SYNPOSIS Dr. Carvalho discusses the latest advancements and practices in the field of pediatric hearing loss, including newer indications for cochlear implants and innovative gene therapies. They delve into the genetic causes of congenital hearing loss, current screening practices, and various gene therapies being tested for hearing deficiencies, such as those involving the OTOF gene and its application through viral vectors. The conversation also touches on the potential for future treatments, including completely implantable cochlear devices and the role of vaccines in preventing CMV-related hearing loss. --- TIMESTAMPS 00:00 - Introduction 01:54 - Understanding Congenital Hearing Loss and Screening 05:41 - Challenges with Older Children and Hearing Loss 08:48 - Cochlear Implants: Indications and Advances 15:59 - Exploring Gene Therapy for Hearing Loss 23:54 - Challenges and Considerations in Gene Therapy 25:23 - Counseling Families on Hearing Loss 27:04 - Future of Genetic Therapy and Cochlear Implants 32:46 - Addressing Congenital CMV 40:59 - Final Thoughts and Future Research --- RESOURCES Dr. Daniela Carvalho https://www.rchsd.org/doctors/daniela-carvalho-md-mmm/

Mac Davis never planned on revolutionizing human health through gene therapy. A serial biotechnology entrepreneur, he founded Minicircle after developing a groundbreaking transient genetic enhancement platform. In this episode, Davis shares his journey from scientific researcher to innovative startup founder, discussing the potential of hormones like statin to optimize muscle health, reduce inflammation, and potentially slow aging. He explores the challenges of building a gene therapy company, early investor support from Sam Altman and Peter Thiel, and his vision of making personalized health interventions as accessible as a routine medical treatment.

Asthik Biswas, Spyros Batzios, and Kshitij Mankad expand on their recent letter to the editor to explain why ushering in the new era of gene therapy treatments requires not just clinical but also imaging readiness. Imaging readiness in the gene therapy era-exploring standardized protocols for response assessment Asthik Biswas, et al https://doi.org/10.1002/jimd.12828

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##New vaccine shows promising results in clinical trialsA new vaccine for a common virus has shown promising results in clinical trials, with a high efficacy rate and minimal side effects. This development could potentially revolutionize the way we prevent and treat this virus in the future.##FDA approves groundbreaking gene therapy for rare genetic disorderThe FDA has approved a groundbreaking gene therapy for a rare genetic disorder, marking a significant milestone in the field of genetic medicine. This therapy has the potential to transform the lives of patients suffering from this debilitating condition.##Big pharma company announces major mergerA major pharmaceutical company has announced a merger with another big player in the industry, creating a powerhouse in the global pharmaceutical market. This merger is expected to have far-reaching implications for the industry as a whole.##Biotech startup secures record-breaking funding roundA biotech startup has secured a record-breaking funding round, signaling strong investor confidence in their innovative approach to drug development. This funding will allow the startup to advance their research and bring potentially life-saving treatments to market.##Researchers discover new mechanism of action for cancer drugResearchers have discovered a new mechanism of action for a commonly used cancer drug, shedding light on how it effectively targets cancer cells. This discovery could lead to the development of more targeted and effective cancer treatments in the future.##FDA issues warning about potential side effects of popular arthritis medicationThe FDA has issued a warning about potential serious side effects associated with a popular arthritis medication, urging patients to consult their healthcare providers before starting or continuing treatment. This warning highlights the importance of monitoring and reporting adverse reactions to medications.##Biopharmaceutical company announces breakthrough in Alzheimer's researchA biopharmaceutical company has announced a major breakthrough in Alzheimer's research, identifying a promising new drug candidate that could potentially slow or even reverse the progression of the disease. This development offers hope for millions of patients and their families affected by Alzheimer's.##Pharma giant partners with leading research institute to accelerate drug discoveryA pharmaceutical giant has partnered with a leading research institute to accelerate drug discovery efforts and bring innovative therapies to market faster. This collaboration aims to leverage the strengths of both organizations to address unmet medical needs and improve patient outcomes.##Health authorities approve new treatment for rare autoimmune diseaseHealth authorities have approved a new treatment for a rare autoimmune disease, providing much-needed hope for patients who have struggled to find effective therapies. This approval represents a significant milestone in the field of autoimmune medicine.##Biotech company receives grant to develop novel antibioticA biotech company has received a grant to develop a novel antibiotic targeting drug-resistant bacteria, addressing a critical need in the fight against antibiotic resistance. This funding will support the company's research efforts and help bring this potentially life-saving treatment to market.##Innovative technology platform revolutionizes drug deliveryAn innovative technology platform has revolutionized drug delivery, enabling more precise targeting of drugs to specific cells or tissues in the body. This technology has the potential to improve treatment outcomes and reduce side effects for patients across various therapeutic areas.#End of podcast episode

00:00:00 - Surf's Up: Season 6 Episode 5Host Roger Green briefly describes this episode's three sections and introduces Roundtable guests. The Roundtable panel shares groundbreakers. 00:10:39 - Roundtable: A Deep Dive Into Drug Development, Part OneThe opening portion of this month's roundtable centers around two issues: exciting data for FGF-21s and, more generally, treating patients with cirrhosis. Naim Alkhouri sets the tone in his opening comments, which start by focusing on the exciting SYMMETRY data from efruxifermin and then hones in on FGF-21s and resmetirom in cirrhosis. The rest of the conversation features Jörh Schattenberg, Sven Francque and Naim discussing therapies in development for compensated and decompensating cirrhosis.00;24:44 - Newsmaker: Naga Chalasani on Real-World Experience Prescribing ResmetiromNaga joins Roger to discuss the paper Early Experience with resmetirom to treat Metabolic Dysfunction-Associated Steatohepatitis With Fibrosis in a Real-World Setting from his group at Indiana University, which his group authored and Hepatology Communications recently posted. The paper, based on IU Health's experience with its first 113 resmetirom patients, shares the group's practical experience developing processes to work closely with the specialty pharmacies dispensing resmetirom and, finally, concludes that a more engaged patient management strategy might reduce drug discontinuation to a level comparable with clinical trials.  00:47:21 - Expert: Scott Friedman on Gene Therapy, Diversity of Stellate Cell Types, Other Basic Liver ScienceScott and Roger cover a range of basis science topics in a fast-moving 19-minute discussion. It starts with Scott discussing the increasing acceptance that gene therapy is an acceptable way to treat a range of liver diseases, many of which are orphan or ultra-orphan but, in fact, include potential gene therapies for non-cirrhotic MASH and MASH cirrhosis. He notes that in addition to classic gene therapy, which introduces protective gene variants into the systems of patients with the risky variants, gene therapy is now looking to introduce FGF-21 into patients through genetic modification. From there, the conversation covers CAR-T therapy, the increasing ability to identify many different types of stellate cells and the idea that the most effective therapy for eary fibrosis, advanced fibrosis and cirrhosis might require fundamentally different kinds of interventions. The two final elements are the idea that what we now call "MASH" may be several diseases with different etiologies with similar manifestations and a passionate call for all of us to support maintaining NIH funding in whatever ways we can.01:06:45 - Business ReportAs Roger copes with his laryngitis, AI voices deliver an abbreviated business report 

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Trump has signaled support for removing the IRA's 'pill penalty', with analysts cautiously optimistic about the executive order. Tariffs are in focus as Q1 earnings get underway, with EU and US pharmas making demands of the European Commission. Former FDA officials warn of potential implications of workforce cuts at the FDA. J&J sets the tariff tone as Q1 earnings begin to roll in. Experts offer advice on optimizing process development and validation steps for cell and gene therapies. In other news, bluebird's second suitor, Ayrmid, fails to make an offer, and the top 6 highest-paid pharma CEOs in 2024 are revealed.The top 6 highest paid pharma CEOs in 2024 have been revealed, with Johnson & Johnson's Joaquin Duato no longer holding the top spot. Duato has urged for a tax fix rather than tariffs to drive US pharma manufacturing. Viking Therapeutics saw a share rally after rival Pfizer discontinued an obesity candidate. Johnson & Johnson's Q1 earnings beat analyst estimates, thanks to Tremfya and Carvykti. Novartis has pledged a $23 billion boost to US operations amid tariff threats. Trump has signaled support for removing IRA's pill penalty and opened a national security probe on pharma imports. The industry is facing uncertainty due to ongoing tariff drama. Trilink's grna for gene editing has been successful, offering high-purity custom guide RNAs for research purposes. Trump's tariff pause sparked a late-day rally for pharma stocks. Some companies, like Glycomine, Merck, and Boehringer Ingelheim, have received significant funding or made deals in the biopharma sector.Senior Editor Annalee Armstrong encourages readers to suggest topics for future coverage in the biopharma industry.

In the final episode of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss the abstract she presented at the AAN Annual Meeting and details on the upcoming gene therapy trial.  Show reference: https://www.neurology.org/doi/10.1212/WNL.0000000000208987 

In part two of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss current treatment options for Leber hereditary optic neuropathy (LHON). Show reference:   https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html   

In part two of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss current treatment options for Leber hereditary optic neuropathy (LHON). Show reference:   https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html  

Guest Whitney Fallon, NP shares how your body works on an Individual basis with your own genetic processes understanding Neuro divergence with ADHD and Autism. How or why, you can or can't lose weight, insulin resistance, the best detox or nutrient absorption. Whitney Fallon Links: https://linktr.ee/woodedlakewellness https://Wellnessrenovation.com Feel the reassurance of a Psychiatric Service Dog—Train your own dog if they recognize and reduce any psychological symptoms, to fly, be in 'no-pet' housing, with no pet fees and access public places under ADA law. Training from Joanne S. Williams, LCSW. A 30-second free guide to see if you qualify at ServiceDogPro.com!   Free 30 minute focus call with Joanne to talk about what would work best for you for your emotional health. Free Cultivate patience worksheet to make the world a more patient place.  

In part one of this four-part series, Dr. Justin Abbatemarco and Dr. Nancy Newman discuss how Leber hereditary optic neuropathy (LHON) typically presents and outline the steps for diagnosing LHON in a clinical setting. Show reference:  https://index.mirasmart.com/AAN2025/PDFfiles/AAN2025-002206.html 

This week's episode of Trambles is brought to you by pollen. Tim putzes around while he waits to speak with KISS bassist and cofounder Gene Simmons.

HFA Symposium is this weekend, and we have CEO Dan Kelsey on the podcast to tell us all about it! Plus, a gene therapy segment with the fantastic Brendan Hayes about what she's hearing in the community about gene therapy for hemophilia B.    Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Show Notes: Subscribe: The BloodStream Podcast   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. Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter