Jason Mast , 2025-05-16 21:35:00
NEW ORLEANS — Last year, hundreds of people poured in to see Peter Marks, the FDA’s head of biologics, speak at the American Society for Cell and Gene Therapy conference. 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.
It’s not a question, though, that everyone wants to address. “Gosh, I don’t know,” said Barry Byrne, head of Powell Gene Therapy Center at the University of Florida. “He’s very outspoken.”
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