Precision Biosciences (NASDAQ: DTIL) has announced the dosing of the first patient in August in the Phase 1/2 FUNCTION-DMD clinical trial evaluating its in vivo gene editing program, PBGENE-DMD, for the treatment of Duchenne muscular dystrophy (DMD).
According to Precision, the first patient was dosed at Arkansas Children’s Hospital. The study is currently enrolling ambulatory DMD patients between the ages of two and seven with mutations between exons 45 and 55, representing up to 60% of boys living with DMD, across multiple U.S. clinical trial sites. PBGENE-DMD was granted Orphan Drug Designation by the FDA in July 2025.
In a statement, Sam Collins, MD, senior vice president, DMD Clinical Development of Precision, commented, “Dosing the first patient in the FUNCTION-DMD study earlier this month was a significant milestone for Precision BioSciences and for the Duchenne community. PBGENE-DMD represents a paradigm shift from currently available approaches. Rather than delivering a highly truncated form of synthetic dystrophin as many therapies in development do today, PBGENE-DMD is designed to permanently edit the patient’s own dystrophin gene to endogenously produce a near full-length, functional dystrophin protein. We are grateful to the patient, their family, and the clinical team for their commitment to advancing this important work, and we look forward to reporting initial safety data by year-end 2026.”
