April 29, 2026 —
Precision BioSciences has activated the first clinical trial site and begun patient screening for its PBGENE-DMD program, marking the clinical advancement of a first-in-class in vivo gene editing therapy for Duchenne Muscular Dystrophy.
The initial site, Arkansas Children’s Hospital, is now enrolling patients in the Phase 1/2 FUNCTION-DMD study, which will evaluate safety, tolerability, and early efficacy signals in ambulatory pediatric patients.
PBGENE-DMD represents a differentiated approach within the DMD treatment landscape. The therapy leverages Precision’s proprietary ARCUS® gene editing platform and is delivered via Adeno-associated virus (AAV) to enable targeted excision of exons 45–55 in the dystrophin gene. This strategy aims to restore expression of a near full-length functional dystrophin protein, potentially improving muscle function in a more physiologically relevant way compared to traditional microdystrophin approaches.
The targeted mutation range represents the largest molecular subset of DMD patients—accounting for up to 60% of affected boys—making the program particularly significant from both clinical and population perspectives.
The FUNCTION-DMD trial is designed to enroll patients aged 2 to 7 years and will assess key endpoints including dystrophin protein expression and functional outcomes, alongside safety monitoring. The study also incorporates immune modulation strategies to support AAV delivery and gene editing activity.
The selection of Arkansas Children’s Hospital as the inaugural site reflects its specialized capabilities. The institution is both a Parent Project Muscular Dystrophy (PPMD)-certified Duchenne Care Center and a Muscular Dystrophy Association (MDA) Care Center, underscoring its experience in managing complex neuromuscular diseases and conducting clinical research in DMD.
PBGENE-DMD has already received Orphan Drug and Fast Track designations from the FDA, and the program is eligible for a rare pediatric disease priority review voucher (PRV)—highlighting both the urgency of the indication and the regulatory support for innovative approaches.
The program’s advancement also reflects a broader shift in the field toward in vivo gene editing delivered via AAV, where the goal is not just gene replacement but precise genomic correction within the body. For DMD, where durability and functional restoration remain key challenges, this approach could represent a meaningful evolution in therapeutic strategy.
