UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More

BioSpace · Mar 4, 2026 · 23m

The FDA is dominating the headlines once again thisweek.

Days after FDA Commissioner Marty Makary appeared to question uniQure’s gene therapy candidate for Huntington’s disease, the company revealed that the agency will require it to conduct a randomized, double-blind, sham surgery–controlled Phase 3 study. The FDA also published anothercomplete response letter (CRL), this one for REGENXBIO’s gene therapy for Hunter syndrome. The rejection, sustained by the biotech early last month, was driven by issues with the study’s population, controls and use of surrogate markers to measure efficacy…