Gene editing company Precision BioSciences Inc (Nasdaq: DTIL) announced on Wednesday that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD, its investigational in vivo gene editing therapy for Duchenne muscular dystrophy (DMD).

The designation provides benefits including development incentives and up to seven years of market exclusivity upon potential approval.

PBGENE-DMD employs dual ARCUS nucleases delivered via a single AAV vector to excise exons 45–55 of the dystrophin gene, aiming to restore production of a near full-length functional dystrophin protein. The approach could benefit up to 60% of the DMD patient population.

In preclinical studies, PBGENE-DMD demonstrated durable dystrophin restoration across key skeletal and cardiac muscle groups and showed the ability to edit satellite muscle stem cells, supporting long-term therapeutic durability. The program was recently granted FDA Rare Pediatric Disease designation, and IND-enabling studies are progressing. A first-in-human trial is being planned with clinical material in preparation and initial data anticipated in 2026.