“Truly evil” FDA rejection of gene therapy overturned after Trump official ousted
Summary
A gene therapy for Huntington’s disease is back on the FDA radar after the ouster of several Trump-era regulators, with UniQure reporting a pathway to accelerated approval using a standard-care control group. The article recounts the debate over trial design (sham surgeries vs. external controls), the departure of Vinay Prasad and other officials, and the FDA’s renewed engagement with the company. It highlights the potential implications for patients and for regulatory processes in high-stakes biotech therapies.