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/653843

00:00 - Introduction
00:34 - Causal Biology and Big Data
17:52 - FDA's Ultra-Rare Decision
27:29 - Lundbeck Acquires Longboard

To submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.

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