Podcasts about Gene therapy

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.

This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year's BIO Conference in Boston. The half-team discusses this week's biggest news: the death of another patient who took Sarepta's Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta's report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac's early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC's entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.  

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.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Metsera's new long-acting amylin injection, met-233i, has shown promising weight loss results over eight months, leading to a rise in shares. Meanwhile, CDC vaccine advisors are either being pushed out or leaving their positions. Other top stories include Sirna's expansion beyond the liver, Keros returning $375 million to investors, and ACIP members receiving termination notices. In vitro cell research is focused on slowing aging and preventing age-related diseases. Updates on Merck's oral PCSK9 inhibitor, Sanofi and Regeneron's Dupixent effectiveness, and Avidity's muscular dystrophy drug are also highlighted.The expansion of RNA therapeutics is discussed, with multiple companies aiming to target small interfering RNA to various organs by 2030. Uniqure's regulatory progress in developing a gene therapy for Huntington's disease has sparked optimism, although past disappointments for patients are noted. Perspective Therapeutics presents new data on neuroendocrine tumor treatment at ASCO25. Concerns about RFK Jr.'s vaccine campaign and its potential to increase distrust in vaccines are raised in the editorial. Cancer news, cell and gene therapy updates, upcoming events, job listings, and a call for reader suggestions on coverage topics are also covered.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##News 1: FDA approves new drug for treatment of rare genetic diseaseIn a significant development for the rare disease community, the FDA has approved a new drug for the treatment of a rare genetic disease. The drug, which is the first of its kind, has shown promising results in clinical trials and is expected to provide much-needed relief for patients suffering from this condition.##News 2: Biotech company announces breakthrough in cancer researchA leading biotech company has announced a major breakthrough in cancer research. The company's new treatment has shown significant efficacy in treating a variety of cancers, offering hope to patients who previously had limited treatment options. This development marks a significant step forward in the fight against cancer.##News 3: Pharma company reports positive results in COVID-19 vaccine trialsA pharmaceutical company has reported positive results in clinical trials for its COVID-19 vaccine candidate. The vaccine has shown high efficacy rates in preventing infection and reducing the severity of symptoms in vaccinated individuals. This news comes as a welcome development in the ongoing battle against the global pandemic.##News 4: Biotech startup secures funding for innovative gene therapyA promising biotech startup has secured funding for its innovative gene therapy approach. The therapy, which targets a range of genetic disorders, has shown promising results in preclinical studies and is now moving towards clinical trials. This funding will help accelerate the development of the therapy and bring it one step closer to market availability.##News 5: Pharma giant announces collaboration with leading research institutionA major pharmaceutical company has announced a collaboration with a leading research institution to advance drug discovery efforts. The partnership aims to leverage the expertise of both organizations to accelerate the development of new treatments for a variety of diseases. This collaboration represents a significant step forward in the quest for innovative therapies.##News 6: Biotech conference showcases latest advancements in gene editing technologyA recent biotech conference showcased the latest advancements in gene editing technology. Researchers presented cutting-edge techniques that have the potential to revolutionize the field of genetic engineering. These advancements hold promise for developing new treatments for a wide range of diseases and disorders.##That's all for today's episode of Pharma and Biotech Daily. Stay tuned for more updates on the latest developments in the world of pharmaceuticals and biotechnology. Thank you for listening!

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Fired employees from the Department of Health and Human Services are suing Robert F. Kennedy Jr. and Elon Musk, claiming their terminations were based on inaccurate and error-ridden information. The lawsuit alleges that HHS leadership knew the records used to guide layoff decisions were flawed. UniQure's regulatory progress has sparked optimism for Huntington's disease patients, with analysts reacting positively to the company's accelerated approval pathway for a gene therapy submission in 2026. Cullinan has made a back-heavy deal in China worth over $700 million for autoimmune T cell engager technology, while Sarepta has won FDA's platform technology designation for gene therapy development. Rapt is shrinking after discontinuing a drug, agileAD is gearing up to challenge J&J in the multiple myeloma Car-T market, and Lilly has signed a deal for long-acting obesity drugs.

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.

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.

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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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.

We talk about Jennifer Anniston home invasion, Gta 6 trailer, Gears of war reloaded, Destiny 2 new content, jaws anniversary popcorn bucket, Hawkeye 2, Alcatraz, Deas biggest bust potentially, 5 to 7 minute standing ovations on movies, nier creator says ai will replace video game developers and more. If you like the podcast please comment and follow.

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.

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.

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.

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