FDA Aligns with REGENXBIO on Accelerated Approval Pathway for NAVSUNLI Gene Therapy in Hunter Syndrome
FDA reverses course and agrees to review existing data for NAVSUNLI gene therapy in Hunter syndrome.
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REGENXBIO Q1 Earnings: Revenue Miss Overshadows Positive Duchenne Gene Therapy Data
Regenxbio’s DMD gene therapy showed strong results, boosting muscle protein and safety, moving closer to FDA approval.
uniQure Stock Tanks on FDA Comments – But Is It Really About AMT-130?
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 Rejects Regenxbio's Gene Therapy for Hunter Syndrome After Clinical Hold
FDA issues Complete Response Letter rejecting RGX-121 gene therapy for Hunter syndrome, citing trial design concerns. Regenxbio plans to resubmit with additional data.
FDA Halts REGENXBIO Gene Therapy Trials After Brain Tumor Discovery
REGENXBIO shares drop 32% as FDA places clinical hold on two rare disease gene therapies following brain tumor discovery in trial participant.
REGENXBIO Shares Jump on Promising Duchenne Gene Therapy Results
REGENXBIO stock rises 8% after positive 18-month trial data for RGX-202, its one-time gene therapy treating Duchenne muscular dystrophy in boys.