Gene therapy offering hope to kids with genetic disorders

Gene therapy offering hope to kids with genetic disorders

In many situations, it isn’t a cure, but it may slow the damage.

FDA to review Regenxbio's gene therapy BLA for Hunter syndrome

The agency has granted the application priority review, setting a Prescription Drug User Fee Act (PDUFA) target action date of 9 November 2025. The therapy has secured several The post FDA to review Regenxbio’s gene therapy BLA for Hunter syndrome appeared first on Pharmaceutical Business review.