uniQure shares fell about 33% after FDA Commissioner Marty Makary's comments on rare disease drugs. But analysts say his remarks likely targeted another therapy, not uniQure's AMT-130 for Huntington's disease.
FDA issues Complete Response Letter rejecting RGX-121 gene therapy for Hunter syndrome, citing trial design concerns. Regenxbio plans to resubmit with additional data.
REGENXBIO shares drop 32% as FDA places clinical hold on two rare disease gene therapies following brain tumor discovery in trial participant.
REGENXBIO stock rises 8% after positive 18-month trial data for RGX-202, its one-time gene therapy treating Duchenne muscular dystrophy in boys.
