Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.
Gene therapy delivered before birth may finally be inching toward reality, as UCSF researchers have submitted an FDA application to target a devastating neurodegenerative disorder that kills many children before age 3.
Also, the FDA leadership tumult is rattling investors, destabilizing rare disease drug development, and raising concern about the agency’s scientific independence.
