Precision BioSciences has announced the activation of the first clinical trial site and the opening of patient screening and enrollment for PBGENE-DMD. PBGENE-DMD, the first-in-class in-vivo gene editing treatment for Duchenne Muscular Dystrophy (DMD), is being evaluated in the phase 1/2 FUNCTION-DMD study.

PBGENE-DMD is Precision’s wholly owned in-vivo gene editing program designed to potentially provide durable functional muscle improvement for patients with DMD who have mutations between exons 45 and 55, the largest molecular subset of boys living with the disease. The phase 1/2 FUNCTION-DMD study will enroll ambulatory patients and is designed to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes.

Michael Amoroso, Chief Executive Officer, Precision BioSciences, said, “The activation of Arkansas Children’s Hospital marks a critical step in advancing PBGENE-DMD into the clinic and expanding access for patients and families who are looking for novel options to address this devastating genetic disease. Dr. Aravindhan Veerapandiyan and the team at Arkansas Children’s Hospital bring deep experience in running clinical trials and research in Duchenne. As the inaugural site for the FUNCTION-DMD study, we are pleased to open screening and enrollment at this center while we continue to work to activate other trial sites for inclusion in the FUNCTION-DMD study.”

Arkansas Children’s is a Parent Project Muscular Dystrophy (PPMD)-certified Duchenne Care Center, recognised for delivering specialised, multidisciplinary care for patients with DMD. PPMD’s Certified Duchenne Care Center Program is intended to help ensure that participating centers maintain high standards in clinical and sub-specialty services, rapidly incorporate evidence-based knowledge, and provide standardised multidisciplinary Duchenne care. Additionally, Arkansas Children’s is a designated Muscular Dystrophy Association (MDA) Care Center, providing specialised, multidisciplinary care for neuromuscular diseases including diagnosis, personalised treatment plans, and comprehensive support for patients.