Precision BioSciences Presents Preclinical PBGENE-DMD Data Highlighting Durable Dystrophin Expression and Functional Benefit at the Muscular Dystrophy Association Clinical & Scientific Conference 2026

Core Insights - Precision BioSciences presented preclinical data for PBGENE-DMD, highlighting its potential for durable dystrophin expression and functional benefits in treating Duchenne muscular dystrophy (DMD) [1] - The data indicates that PBGENE-DMD could potentially treat up to 60% of DMD patients by restoring near full-length functional dystrophin protein through permanent gene correction [1] Group 1: Preclinical Data Findings - PBGENE-DMD treatment in a humanized DMD mouse model showed improvements in muscle pathology and biomarkers of muscle damage, with a 50-65% reduction in CK levels at 90 days post-treatment [1] - Treated mice maintained approximately 81% to 84% of maximal force output and 89% to 92% of tetanic force output compared to healthy mice over a nine-month period [1] - Evidence of therapeutic levels of functional dystrophin protein was observed in both skeletal and cardiac muscle, with increasing levels over time [1] Group 2: Treatment Mechanism and Design - PBGENE-DMD utilizes a gene excision approach, differentiating it from existing treatments like microdystrophin and exon skipping [1] - The therapy targets muscle satellite cells, which are essential for muscle regeneration, potentially providing long-term benefits [1] - The treatment is designed for DMD patients with mutations in exons 45-55, representing a significant portion of the patient population [1] Group 3: Clinical Trial and Regulatory Status - The Phase 1/2 FUNCTION-DMD clinical trial is set to enroll ambulatory DMD patients aged 2-7 with specific mutations, focusing on safety, tolerability, and efficacy [1] - PBGENE-DMD has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, facilitating its development and potential market entry [1]