NEW ORLEANS — Last year, hundreds of people poured in to hear Peter Marks, the Food and Drug Administration’s head of biologics, speak at the American Society of Gene and Cell 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 forced out by the Trump administration. And among the biggest questions facing the gene therapy field is what stance Vinay Prasad, the fiery hematologist and frequent 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 the Powell Gene Therapy Center at the University of Florida. “He’s very outspoken.”
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