Precision BioSciences receives Rare Pediatric Disease Designation for Duchenne Muscular Dystrophy therapy

From Nasdaq: 2025-06-25 07:40:00

Precision BioSciences receives Rare Pediatric Disease Designation from the FDA for gene therapy candidate PBGENE-DMD to treat Duchenne muscular dystrophy. The designation may make Precision eligible for a Priority Review Voucher if PBGENE-DMD is approved, potentially shortening the FDA review time for another product. IND-enabling toxicology studies are ongoing, with initial data expected in 2026. The program aims to excise exons 45-55 of the dystrophin gene, offering potential improvement for up to 60% of DMD patients.



Read more at Nasdaq: Precision BioSciences Gets Rare Pediatric Disease Status For Duchenne Muscular Dystrophy Therapy