Precision BioSciences has received a Study May Proceed notification from the US Food and Drug Administration, greenlighting clinical trial site activation for its first-in-class gene editing therapy, PBGENE-DMD, in ambulatory Duchenne muscular dystrophy (DMD) patients. 

 

The Phase 1/2 FUNCTION-DMD study will evaluate safety, tolerability, and efficacy—including dystrophin protein restoration and functional outcomes—at leading specialized trial sites.

 

PBGENE-DMD is designed to permanently correct mutations in the dystrophin gene between exons 45 and 55, the “hot-spot” region affecting roughly 60% of DMD patients. Unlike current micro-dystrophin therapies, PBGENE-DMD aims to restore near full-length functional dystrophin, retaining 80% of the full-length protein, potentially offering more durable functional benefits.

 

“Duchenne muscular dystrophy is a progressive disorder caused by mutations in the dystrophin gene that disrupt the production of the functional protein, resulting in continuous muscle degeneration,” said Aravindhan Veerapandiyan, Director, Comprehensive Neuromuscular Program, Arkansas Children’s Hospital. 

 

“PBGENE-DMD is designed to restore near full-length dystrophin, with the potential to provide significant functional benefits. This is an important milestone, and I look forward to participating in the Phase 1/2 FUNCTION-DMD clinical study to evaluate a gene excision approach in DMD patients with mutations involving exons 45-55.”

 

Families and advocacy groups see PBGENE-DMD as a potential game-changer. 

 

“PBGENE-DMD is an important step forward for the Duchenne community as it’s the first ever gene editing approach designed to treat the majority of DMD patients. Families are urgently waiting for newer therapies that can meaningfully and durably change the course of this disease,” said Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy. 

 

“We look forward to learning more as the FUNCTION-DMD study begins and to continued collaboration between patient advocates, physicians and developers of new treatments to accelerate progress for every person living with Duchenne.”

 

Precision BioSciences CEO Michael Amoroso highlighted the regulatory milestone: “The Study May Proceed notification for PBGENE-DMD by the FDA represents yet another regulatory achievement for Precision BioSciences as we advance our second wholly owned program toward the clinic. 

 

"Despite approved therapies today, boys with DMD are lacking treatments that lead to functional improvements over time. We’re excited to bring this novel gene excision approach for DMD to the clinic with the goal of activating the first clinical site in the U.S. in the first half of 2026."