BioCentury This Week

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

Chinese biotech is the lone bright spot for the biopharma financial markets in 1H25, as macro concerns about the most favored nation (MFN) pricing policy and FDA weigh on the prospects for biotech elsewhere. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the positives — and negatives — impacting investor sentiment headed into the second half of the year. The team also discuss where the quantum computing revolution could have its biggest impact on drug development, and review the proposals and accomplishments of FDA Commissioner Marty Makary over his first 100 days in office. This episode of BioCentury This Week was sponsored by IQVIA Biotech.View full story: https://www.biocentury.com/article/656482#biotech #biopharma #pharma #lifescience #financialmarkets #FDA #quantumcomputing00:01 - Sponsor Message: IQVIA Biotech04:43 - Q25 Financial Markets Preview22:20 - Quantum Computing Impact on Biopharma30:07 - Makary's First 100 DaysTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

There are many reasons why it may be the European biomedical ecosystem’s moment to shine, and advances at the academia-industry interface are a core part of that story, particularly in Cambridge, U.K. — the site of the inaugural BioCentury Grand Rounds – Europe conference.On a special edition of the BioCentury This Week podcast, BioCentury’s analysts are joined by four leaders in the Cambridge, U.K., life sciences community to preview the Sept. 17-19 meeting: Kathryn Chapman, executive director of Innovate Cambridge; Anne Horgan, partner, Cambridge Innovation Capital; Sally Mardikian, business development director, Astrazeneca; and Kristin-Anne Rutter, executive director of Cambridge University Health Partners.The BioCentury team and its Cambridge counterparts discuss the conference’s themes and how they relate to the region’s strengths, and offer both some tough love and a warm invitation to early-stage innovators looking for science-forward investors and partners.View full story: https://www.biocentury.com/article/656373#biotech #biopharma #pharma #lifescience #Discovery #Translation #BioCenturyGrandRoundsEurope00:00 - Introduction01:53 - Grand Rounds Cambridge Themes10:14 - Why Cambridge?23:43 - Tough Love28:02 - Join us at GlastonGeekTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Oncology regulation has been less affected by recent changes in FDA leadership than other parts of the agency. On a special sponsored edition of the BioCentury This Week podcast, BioCentury’s analysts assess what has and hasn’t changed at FDA when it come to regulating cancer therapeutics. Special Guest Liz O’Brien joins BioCentury’s analysts to discuss how biotechs can navigate FDA’s Project Optimus and EMA’s Joint Clinical Assessment as well as the differences between the two agencies. O’Brien is a former EMA regulator who is now therapeutic expert, oncology and women’s health, drug development solutions at BioCentury This Week sponsor ICON Biotech. View full story: https://www.biocentury.com/article/656371#biotech #biopharma #pharma #lifescience #FDA #cancer #CAR_T00:01 - Sponsor Message: ICON Biotech02:30 - FDA Cancer Regulations07:48 - Project Optimus25:12 - CAR T REMS RemovalTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Treg-based cell therapies are nearing a clinical proof-of-concept inflection point, but in an environment that has dramatically shifted with the advent of CAR T therapies for autoimmune diseases. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss what these cell therapies need to show to be relevant in the fast-changing I&I world, and which types of indications may best suit the cells’ unique profile.The team also explores the biotech sector’s ongoing struggle to capture the interest of generalist investors, and what the next major theme might be to draw them back.The analysts then break down the key provisions in the recently passed U.S. reconciliation tax bill, highlighting the positive and negative developments, as well as the missed opportunities for the industry. This episode of BioCentury This Week was sponsored by IQVIA Biotech. View full story: https://www.biocentury.com/article/656376#biotech #biopharma #pharma #lifescience #CellTherapy #TregTherapy #CAR_T00:01 - Sponsor Message: IQVIA Biotech01:19 - Treg Inflection Point10:57 - Biotech's Next Big Story22:36 - Tax Bill ConsequencesTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

The Trump administration’s overhaul of FDA is still underway, but the agency has already veered away from its decades-long trajectory in ways that will profoundly reshape medical product development and the lives of U.S. patients. On the latest BioCentury This Week podcast, BioCentury’s analysts assess who the winners and losers are likely to be as FDA changes shape.The analysts discuss a pair of in vivo CAR T companies: Esobiotec, whose story offers a case study in product-focused strategy, and Capstan Therapeutics, whose in vivo CAR T platform for immunological disorders attracted a buyer in Abbvie. The team also analyzes why data from Compass Pathways for psilocybin therapy COMP360 disappointed investors despite hitting the endpoint, and how molecular glues could be the future of selective CDK2 targeting. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656341#biotech #biopharma #pharma #lifescience #RandD #drugapproval00:01 - Sponsor Message: ICON Biotech02:26 - BioCentury Grand Rounds Europe05:02 - FDA's Future15:03 - Psychedelics22:53 - Esobiotech's Fast Exit31:37 - Molecular Glues and CDK234:43 - Abbvie's Capstan BuyTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Publicly traded U.K. investment firm and company builder Syncona is restructuring its fund amid ongoing market challenges in the biopharma industry. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss how the firm will steer its portfolio toward returns for shareholders, while aiming to build a new fund away from public markets. The analysts then assess Eli Lilly's takeout of cardiovascular base editing company Verve, and what Washington Editor Steve Usdin calls FDA’s new “two-track” future for evaluating new therapies for approval — those with clear-cut benefits and those with ambiguous efficacy safety and efficacy profiles — in light of the many departures of senior FDA staff. They also discuss NASDAQ’s largest biopharma IPO — by Caris —  in two years, the latest obesity readouts, FDA Commissioner Marty Makary’s priority pathway and Usdin’s Q&A with new BIO Chair Fritz Bittenbender. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656266#biotech #biopharma #pharma #lifescience #finance #CV #FDA00:01 - Sponsor Message: ICON Biotech02:14 - Syncona Restructures Fund10:08 - Lilly's $1B Verve Takeout21:45 - FDA's 2-Track FutureTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

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

In this engaging discussion, Ed Hodgkin, managing partner at Syncona, sheds light on the urgent challenges academia faces amid shifting VC priorities and federal funding risks. He emphasizes the need for innovative collaboration between academia and pharma to unlock first-in-class innovations. The exploration of drug development complexities and advancements in delivery systems reveals a bright future for biopharmaceuticals, while also addressing endometriosis challenges and the exciting potential of Europe’s biotech landscape.

Translational trends at this year’s ASCO meeting featured new and selective ways to target cell surface receptors on solid tumors. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the findings from Executive Director of Biopharma Intelligence Lauren Martz’s deep dive into first-in-human studies at the American Society of Clinical Oncology meeting, including how immunocytokines, solid tumor CAR Ts and Chinese innovation are thriving in early trials.The analysts also examine the signs of strain and resilience in biotech’s crossover investors, as well as FDA’s plans for revamping rare disease regulation. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656139#biotech #biopharma #pharma #lifescience #RandD #DrugDevelopment00:01 - Sponsor Message: ICON Biotech02:11 - ASCO’s First-in-Human Trials12:46 - Crossover Investor Health Check22:54 - FDA's Rare Disease PlansTo submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text