Precision BioSciences doses first patient in DMD gene-editing trial

Precision BioSciences, Inc. (Nasdaq: DTIL) said on August 24, 2026 that the first patient has been dosed in the Phase 1/2 FUNCTION-DMD clinical trial evaluating PBGENE-DMD for the treatment of Duchenne muscular dystrophy, marking the first clinical gene-editing program for DMD.¹ The dosing took place at Arkansas Children's Hospital, a PPMD Certified Duchenne Care Center and designated MDA Care Center.¹

PBGENE-DMD is described as Precision's wholly owned in vivo gene editing program designed to durably improve function.¹ The company said the approach aims to restore near full-length dystrophin and is designed to be applicable for up to 60% of DMD patients whose mutations fall in a specific hot spot region of the gene.¹ Mechanistically, the program uses two complementary ARCUS nucleases packaged in a single AAV to excise exons 45-55 of the dystrophin gene, with the goal of producing a near full-length dystrophin protein rather than the truncated microdystrophin used in other approaches in development.¹

The trial is enrolling ambulatory DMD patients ages 2 to 7 with mutations between exons 45 and 55, a population representing up to 60% of boys with DMD, at multiple U.S. sites.¹ The company said initial safety data are expected by year-end 2026.¹ The trial is registered as NCT07429240, according to the release.

Regulatory history noted in the filing includes Orphan Drug Designation from the FDA in July 2025¹ and Fast Track designation from the FDA in February 2026.¹ The program is also eligible for a Priority Review Voucher through the Rare Pediatric Disease program, enacted February 3, 2026 as part of the Consolidated Appropriations Act of 2026.¹