Precision BioSciences Inc. (NASDAQ: DTIL) has dosed the first patient in its Phase 1/2 FUNCTION-DMD clinical trial, marking a milestone in gene editing for Duchenne muscular dystrophy (DMD).
The trial, conducted at Arkansas Children’s Hospital, evaluates PBGENE-DMD, a gene therapy designed to excise exons 45-55 of the dystrophin gene using two ARCUS nucleases delivered via a single AAV vector. The approach aims to restore near full-length functional dystrophin protein, a critical protein missing in DMD patients. The therapy could benefit up to 60% of U.S. DMD patients with mutations in exons 45-55.
The study is enrolling ambulatory DMD patients aged 2 to 7 with confirmed mutations between exons 45 and 55 across multiple U.S. clinical sites. DMD, a rare genetic disease, affects approximately 15,000 patients in the U.S., driven by mutations in the dystrophin gene that prevent protein production.
Precision BioSciences received Orphan Drug Designation from the FDA in July 2025 and Fast Track designation in February 2026. The company is also eligible for a Priority Review Voucher through the Rare Pediatric Disease program. Initial safety data from the trial is expected by the end of 2026, with efficacy, dystrophin protein expression, and functional outcomes among the evaluation criteria.
Sam Collins, Senior Vice President of DMD Clinical Development at Precision BioSciences, stated, 'Dosing the first patient in the FUNCTION-DMD study earlier this month was a significant milestone for Precision BioSciences and for the Duchenne community.'
