Precision BioSciences Inc. (NASDAQ:DTIL) presented new preclinical findings for its PBGENE-DMD gene therapy at the Muscular Dystrophy Association Clinical & Scientific Conference 2026 held in Orlando, Florida.
The research utilized a humanized Duchenne muscular dystrophy (DMD) mouse model designed to mirror the muscle degeneration seen in patients with the disease. Treatment with PBGENE-DMD resulted in improvements in several biomarkers linked to muscle damage, including ALT, AST and CK. Ninety days after treatment, CK levels declined by roughly 50–65%, suggesting better muscle integrity.
The therapy also produced improvements in muscle pathology, with treated animals showing lower composite injury scores across multiple muscle tissues compared with control mice. Earlier data had indicated that treated mice maintained about 81% to 84% of maximal force output and 89% to 92% of tetanic force output measured in healthy mice for up to nine months after treatment.
PBGENE-DMD also generated dystrophin protein in both skeletal and cardiac muscle, with levels continuing to rise through the nine-month observation period. Dystrophin-positive muscle fibers were observed in several key muscle groups, including the quadriceps, gastrocnemius, heart and diaphragm.
The therapy is designed to target patients with mutations in exons 45–55 of the dystrophin gene, which account for as many as 60% of boys diagnosed with DMD. PBGENE-DMD uses two ARCUS proteins to permanently edit DNA within the dystrophin gene, with the aim of restoring production of a near full-length, functional dystrophin protein.
PBGENE-DMD has received multiple regulatory designations from the U.S. Food and Drug Administration, including Fast Track, Rare Pediatric Disease and Orphan Drug status. After receiving investigational new drug (IND) clearance in early 2026, Precision BioSciences is preparing to activate clinical trial sites in the United States during the first half of the year.
The upcoming Phase 1/2 FUNCTION-DMD study will enroll ambulatory patients aged 2 to 7 with DMD who carry mutations between exons 45 and 55.