New Orleans was abuzz last year as hundreds of people gathered to listen to Peter Marks, the head of biologics at the Food and Drug Administration, speak at the American Society of Gene and Cell Therapy conference. Many advocates, researchers, and executives saw Marks as their ally in the push to approve treatments for rare diseases, even with limited or conflicting evidence.
Fast forward to now, and Marks has been ousted by the Trump administration. The gene therapy community is now left wondering about the stance that Vinay Prasad, a fiery hematologist and known pharma critic who has taken over Marks’ position, will take on rare disease treatments.
Not everyone is eager to tackle this question head-on. Barry Byrne, the head of the Powell Gene Therapy Center at the University of Florida, simply said, “Gosh, I don’t know. He’s very outspoken.” Some uncertainty lingers as to what the future holds in this new era of gene therapy regulation.
As the gene therapy field navigates this transition, there are concerns about how Prasad will approach the approval process for rare disease treatments. The community is eagerly awaiting his first moves and decisions. Maybe it’s just me, but it feels like there’s a lot riding on the direction he takes in this role. Will he continue Marks’ legacy or forge a new path? Only time will tell.
