The Readout Loud

Marissa Russo, a STAT fellow and PhD candidate at the Mayo Clinic focusing on glioblastoma, joins to delve into the hotly debated topic of alternatives to animal testing. She explores the innovative methodologies like organ-on-chip models and the push from the FDA and NIH towards more humane research practices. The conversation also touches on the complexities of these new approaches and the challenges in ensuring their reliability. Additionally, insights on the shifting landscape of mRNA technology add to the discussion's depth.

In this episode, Natalie Holles, former CEO of Third Harmonic Bio, shares her insights on the tough decisions behind dissolving the company and returning cash to shareholders. She discusses the challenges of biotech leadership amid failures, including Eli Lilly's disappointing obesity drug results and Replimmune's FDA rejection. Holles emphasizes the importance of transparency and data-driven approaches in the industry. The conversation wraps up with a humorous take on the challenges of balancing personal life with professional aspirations in biotech.

In this discussion, guests Brian Skorney, a senior research analyst at Baird, Ned Sharpless, former acting FDA commissioner, and Robert Califf, former FDA commissioner, delve into the impact of Vinay Prasad's departure from the FDA. They examine the political turbulence influencing FDA operations and the biotech sector, especially regarding drug approvals and leadership changes. The conversation highlights the challenges of regulatory integrity, the evolving landscape of gene therapies, and the urgent need for improved data collection in post-marketing drug safety.

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.