FDA Places Clinical Hold on Regenxbio's Gene Therapy for Hunter Syndrome

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Regenxbio's experimental gene therapy, RGX-121, for Hunter Syndrome. The decision follows the discovery of spinal scan abnormalities in five study participants. RGX-121 was being developed as a one-time treatment for Hunter Syndrome, a rare inherited disorder causing progressive damage to the brain and other organs.