Precision BioSciences announced the dosing of the first patient in its Phase 1/2 FUNCTION-DMD clinical trial evaluating PBGENE-DMD for Duchenne muscular dystrophy. The treatment was administered at Arkansas Children’s Hospital, a designated care center. PBGENE-DMD utilizes the company's ARCUS platform to excise exons 45-55 of the dystrophin gene in patients with specific mutations, aiming to restore near full-length functional dystrophin protein. Initial safety data is expected by year-end 2026. Precision BioSciences operates as a clinical-stage biotechnology company developing in vivo gene editing therapies using its proprietary ARCUS platform.
