PBGENE-DMD - (FUNCTION-DMD)

August 24, 2026: Precision BioSciences (NASDAQ: DTIL) said the first patient was dosed earlier in August in the Phase 1/2 FUNCTION-DMD trial of PBGENE-DMD for Duchenne muscular dystrophy. The first dose was given at Arkansas Children’s Hospital.

PBGENE-DMD is described as the first clinical gene-editing program for DMD and is designed to excise dystrophin exons 45-55 using two ARCUS nucleases delivered in a single AAV, with the goal of restoring near full-length functional dystrophin. The approach is intended for ambulatory patients ages 2-7 with mutations between exons 45 and 55, a group the company says represents up to 60% of boys with DMD.

FUNCTION-DMD (NCT07429240) is evaluating safety, tolerability, dystrophin protein expression, and functional outcomes at multiple U.S. sites and is actively recruiting. Precision said initial safety data are expected by year-end 2026.

Regulatory milestones noted in the release: FDA Orphan Drug Designation for PBGENE-DMD in July 2025 and FDA Fast Track designation in February 2026. The company also said the program is eligible for a Rare Pediatric Disease Priority Review Voucher under legislation signed February 3, 2026.