For the DMD program PBGENE-DMD, Precision BioSciences anticipates starting patient dosing in late first or early second quarter of 2026, following expected IND approval. The Phase 1/2 FUNCTION-DMD study will treat ambulatory patients with mutations in exons 45-55. Initial data from multiple patients are expected by the end of 2026.
PBGENE-DMD – Phase 1/2 FUNCTION-DMD Trial in Duchenne Muscular Dystrophy (DMD) Patients
PBGENE-DMD, a novel first-in-class gene editing therapy, utilizes a gene excision approach, which is clearly differentiated from existing microdystrophin and exon skipping treatments. PBGENE-DMD is designed to potentially provide durable functional muscle improvement for DMD patients with mutations in exons 45-55 impacting up to 60% of boys with DMD. A single AAV encodes two ARCUS proteins designed to permanently edit a patient’s DNA within the dystrophin gene, resulting in a naturally-expressed, near full-length, functional dystrophin protein. Supported by robust preclinical evidence, PBGENE-DMD is designed to drive functional improvement over time by targeting muscle satellite cells. Learn More: Precision BioSciences PBGENE-DMD Restores Production of Nearly Full-Length Dystrophin Protein
Following clearance of the investigational new drug (IND) application, the FUNCTION-DMD Phase 1/2 clinical trial is expected to dose the first patient in late-Q1 or early-Q2 2026. The study will employ an appropriate immune modulation regimen and safety monitoring program to treat ambulatory patients with mutations in exons 45-55 at world class specialized DMD clinical sites. Initial data from multiple patients is expected by year end 2026, with early efficacy assessed by the percentage of near full-length dystrophin protein expression from muscle biopsies. Following supportive data from at least 10 DMD patients, the company would meet with the FDA to align on a regulatory path forward.
Read More: Clinical Trials for Duchenne (List of All Researches)
