PBGENE-DMD - (FUNCTION-DMD)

November 5, 2026: Precision BioSciences (NASDAQ: DTIL) said the independent DSMB completed its review of the first patient treated with PBGENE-DMD in the Phase 1/2 FUNCTION-DMD trial in Duchenne muscular dystrophy and found no safety concerns through the initial 8-week observation period. No serious adverse events or adverse events of special interest were reported, and the DSMB endorsed dosing additional patients in Part 1 of the study.

PBGENE-DMD is an in vivo gene-editing program 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. FUNCTION-DMD is enrolling ambulatory DMD patients ages 2-7 with mutations between exons 45 and 55, a group the company says represents up to 60% of boys with DMD. The trial is evaluating safety, tolerability, dystrophin protein expression, and functional outcomes.

The company said it plans to provide safety and efficacy updates from FUNCTION-DMD throughout 2027. PBGENE-DMD previously received FDA Orphan Drug Designation in July 2025, Rare Pediatric Disease Designation, and Fast Track designation in February 2026.