STAT+: Day 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data
A hot (if cautious) topic at the big gene therapy meeting: what Vinay Prasad's appointment might mean for approvals in rare disease treatments.
NEW ORLEANS — Greetings from the home stretch of American Society of Gene and Cell Therapy conference, where everyone is still talking about Baby KJ and no one wants to talk about Vinay Prasad.
The Vinay Prasad in the room
Last year, hundreds of people poured in to see Peter Marks, the FDA’s head of biologics, speak at at ASGCT. The regulator was seen by many advocates, researchers and executives as an ally in their efforts to get rare disease treatments approved, sometimes despite limited or contradictory evidence.
Now, of course, Marks has been fired by the Trump administration. And among the biggest questions facing the gene therapy field is what stance Vinay Prasad, the fiery hematologist and oft-pharma critic who replaced him, will take toward such rare disease treatments.
