The Readout Loud

Kaylin West, mother of 7-year-old Cooper with Duchenne muscular dystrophy, shares her powerful insights on Sarepta's decision to halt its gene therapy, Elevidys. She reflects on her family's journey through the hopeful yet tumultuous world of gene treatments. The podcast also delves into the emotional impact of such medical developments, highlighting the importance of community support for families navigating similar challenges. Alongside biotech innovations and new drug developments, Kaylin's personal story adds a poignant depth to the discussion.

Just how many employees is Sarepta Therapeutics laying off? And why did the Food and Drug Administration reject Ultragenyx’s rare disease drug over manufacturing qualms? Ultragenyx CEO Emil Kakkis joins us to discuss what was in the rejection letter his company received late last week, and whether he still has confidence in FDA Commissioner Marty Makary. We also discuss Capricor’s drug rejection, and Sarepta’s decision to lay off hundreds of staffers, but give raises to some executives.

On this week’s episode of the Readout LOUD: A closer look at the NIH’s grant-cutting legal playbook, a not-so-transparent transparency push by the FDA commissioner, and another big biotech acquisition. Our colleague Anil Oza joins us to unravel a previously unseen memo he obtained from HHS that lays out the legal framework being used to justify the termination of NIH grants. The disclosure comes as legal fights over the legitimacy of these funding cuts are being challenged in multiple courts.

Helen Branswell, a senior writer for infectious diseases at STAT, and Chelsea Cirruzzo, a Washington correspondent for STAT, dive into the latest CDC happenings, including a heated confirmation hearing for its new nominee. They discuss significant findings from the American Diabetes Association meeting, highlighting challenges with obesity drug side effects. The duo also laments the summer movie season's disappointments while reminiscing about classic blockbusters like 'Jaws.' It’s a mix of health insights and cinematic nostalgia worth a listen!

A tragic turn of events unfolds as two teenage boys die after gene therapy, stirring controversy over the FDA's approval standards. Leadership turmoil at the FDA raises questions about safety and political influence in drug approvals. Meanwhile, a private equity firm steps in to fund Harvard research, highlighting concerns over corporate impact on academia. In brighter news, a groundbreaking HIV prevention drug gains approval, and insights from a massive biotech convention provide a glimpse into the future of healthcare.

Kathryn Edwards, a vaccine expert and former member of the CDC advisory panel, joins to discuss the upheaval within the CDC's vaccine advisory group and its impact on public trust. She sheds light on the controversial new appointments and their implications for vaccine safety assessments. The conversation also addresses complexities in current drug pricing policies, including the challenges pharmaceutical companies face amid government guidelines. Additionally, they explore the struggles in manufacturing mRNA-based vaccines and the shift in public health dynamics.

Brittany Trang, a STAT colleague and AI expert in the healthcare sector, joins to discuss the FDA's new AI tool, ELSA, which struggles with rectifying past mistakes. She sheds light on Sanofi's $9 billion acquisition of Blueprint Medicines and how it's shaking up the biotech landscape. The conversation also delves into Novo Nordisk's challenges in the obesity drug market, focusing on their strategic errors and the implications of leadership changes, while raising ethical questions about aggressive marketing.

Helen Branswell, an infectious disease reporter at STAT, shares her insights on some pressing public health issues. She delves into RFK Jr.'s controversial move to alter COVID vaccine recommendations and the cancellation of a significant Moderna contract. The discussion highlights the implications of these decisions on pandemic preparedness. Additionally, the conversation touches on the Make America Healthy Again Commission's report, emphasizing the need for improved vaccine transparency and monitoring in light of recent safety concerns.

Jason Mast, a STAT reporter specializing in gene therapy, brings a wealth of knowledge to the discussion. He shares insights from the recent American Society of Cell and Gene Therapy meeting, highlighting both excitement and uncertainty in the gene editing field. The conversation touches on recent mergers, including Sanofi's acquisition of Vigil Neurosciences, and the changing landscape fueled by leadership shifts at Novo Nordisk. They also delve into the FDA's review of cancer drugs, navigating both data challenges and clinical implications.

Daniel Payne, STAT's D.C. correspondent specializing in health policy and pharma, dives into the complexities of President Trump's aggressive drug pricing policy aimed at making U.S. prices competitive with other nations. He also discusses the mixed outlook for biotech markets and the potential launch of a new blood-testing company founded by Billy Evans, which draws parallels to the notorious Theranos. The episode paints a vivid picture of skepticism in the industry as market fluctuations and past scandals loom large.