Precision BioSciences Inc. (NASDAQ: DTIL) presented preclinical data for its PBGENE-DMD gene therapy at the Muscular Dystrophy Association Clinical & Scientific Conference 2026 in Orlando, Florida.

The study used a humanized DMD mouse model that replicates muscle degeneration seen in patients with Duchenne muscular dystrophy. PBGENE-DMD treatment showed improvements in muscle damage markers including ALT, AST, and CK. At 90 days after treatment, CK levels decreased by approximately 50-65%, indicating muscle integrity improvements.

The therapy also demonstrated improvements in muscle pathology assessments with lower composite injury scores across multiple muscle tissues compared to control mice. Previously reported data showed treated mice maintained approximately 81% to 84% of maximal force output and 89% to 92% of tetanic force output observed in healthy mice through nine months following treatment.

PBGENE-DMD produced dystrophin protein levels in skeletal and cardiac muscle, with levels increasing over time through nine months. The therapy showed dystrophin-positive fibers across key muscle groups including quadriceps, gastrocnemius, heart, and diaphragm.

The company designed PBGENE-DMD to treat patients with mutations in exons 45-55, representing up to 60% of boys with DMD. The therapy uses two ARCUS proteins to permanently edit DNA within the dystrophin gene, intended to produce a near full-length, functional dystrophin protein.

PBGENE-DMD has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Following IND clearance in early 2026, Precision BioSciences is working to initiate clinical site activations in the U.S. in the first half of 2026. The Phase 1/2 FUNCTION-DMD study will enroll ambulatory DMD patients between ages 2-7 with mutations between exons 45 and 55.