Precision BioSciences doses the first patient in its Duchenne trial

Precision BioSciences dosed the first patient in FUNCTION-DMD on 24 August 2026, a Phase 1/2 study of PBGENE-DMD run at Arkansas Children’s Hospital, a certified Duchenne care centre. The trial enrols ambulatory boys aged two to seven whose mutations fall between exons 45 and 55, a window the company puts at up to 60 percent of boys with Duchenne muscular dystrophy.

What separates this from the approved exon-skipping drugs is the size of the protein it targets. Exon skipping instructs the cell to omit the damaged section, producing a heavily truncated dystrophin that holds the muscle membrane together imperfectly. PBGENE-DMD edits the gene itself, and the stated goal is a near full-length, functional dystrophin rather than a shortened stand-in.

The programme carries Orphan Drug designation from July 2025 and Fast Track from February 2026. Initial safety data are expected by the end of 2026.

One dosed patient establishes that the therapy has entered humans, and nothing more. In vivo gene editing delivered systemically is the same class of intervention that has produced this year’s most serious trial deaths, and the first readout from this study will be a safety readout, not an efficacy one.