PBGENE-DMD is Precision's wholly owned in vivo gene editing program designed to durably improve function. This novel approach, aiming to restore near full-length dystrophin, is applicable for up to 60% of DMD patients with mutations in a key hot spot region. By employing two complementary ARCUS nucleases in a single AAV, PBGENE-DMD excises exons 45-55 of the dystrophin gene with the aim of restoring a near full-length functional dystrophin protein that more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches.

"Dosing the first patient in the FUNCTION-DMD study earlier this month was a significant milestone for Precision BioSciences and for the Duchenne community," said Sam Collins, M.D., Senior Vice President, DMD Clinical Development of Precision BioSciences. "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."

"A therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy represents a meaningful step forward for individuals and families living with this condition," said Aravindhan Veerapandiyan, M.D., Director of the Comprehensive Neuromuscular Program at Arkansas Children’s Hospital. "We’re proud that our center was the first to dose a patient in the FUNCTION-DMD study. PBGENE-DMD is designed to restore near full-length, functional dystrophin, and we look forward to evaluating its safety and potential to provide durable functional benefits. This innovation could open the door to an entirely new approach for treating Duchenne."

"Seeing PBGENE-DMD move from research into the clinic turns the possibility of gene editing for Duchenne into reality — a novel approach that could address some of the limitations of currently available therapies," said Pat Furlong, President, Parent Project Muscular Dystrophy. "Families have been waiting for options like this, and PPMD is encouraged to see this program advance. We look forward to learning more as the study progresses and continuing collaboration on behalf of all our Duchenne families."

The study is currently enrolling ambulatory DMD patients between the ages of 2 and 7 with mutations between exons 45 and 55, representing up to 60% of boys living with DMD, across multiple U.S. clinical trial sites. The study is actively recruiting patients at specialized Duchenne care centers with initial safety data expected by year-end 2026.