Gene editing company Precision BioSciences Inc (Nasdaq:DTIL) reported on Wednesday that it has activated the first clinical trial site and commenced patient enrollment in its Phase 1/2 FUNCTION-DMD study evaluating PBGENE-DMD, an in vivo gene editing therapy for Duchenne muscular dystrophy.

The initial site, Arkansas Children's Hospital, is now screening and enrolling ambulatory patients aged 2 to 7 with mutations between exons 45 and 55, representing up to 60% of boys affected by the disease. The study will assess safety, tolerability and efficacy, including dystrophin protein expression and functional outcomes.

PBGENE-DMD is designed to restore near full-length functional dystrophin by excising targeted gene segments using Precision's ARCUS gene editing platform. The therapy aims to deliver durable functional improvement in a condition where no approved treatments currently provide sustained clinical benefit.

The programme has received Orphan Drug Designation and Fast Track status from the U.S. Food and Drug Administration, and is eligible for a Priority Review Voucher under the Rare Pediatric Disease programme.