Ep. 259 - Causal Biology and Big Data, Ultra-Rare Drugs at FDA

There is a growing mandate among researchers and VCs to provide proof of causal human biology for new targets. On the latest BioCentury This Week podcast, BioCentury’s editors discuss the different strategies being deployed to identify causal links to disease using observational patient data or human cell models, including the challenges that come with each approach and the various computational methodologies companies are using.They also discuss the outcome of FDA’s advisory committee meeting on Barth syndrome candidate elamipretide from Stealth Biotherapeutics, and the implications of the discussion for review of ultrarare disease therapies more broadly.Diving into the deal of the day, the editors review the proposal by H. Lundbeck to acquire Longboard Pharmaceuticals for $2.6 billion, and discuss how the biotech’s therapy for developmental epilepsies may stack up against competitors.View full story: https://www.biocentury.com/article/65384300:00 - Introduction00:34 - Causal Biology and Big Data17:52 - FDA's Ultra-Rare Decision27:29 - Lundbeck Acquires LongboardTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text