Precision BioSciences, Inc. announced on May 14, 2025, the strategic prioritization and acceleration of PBGENE-DMD, its first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD). This decision is based on compelling preclinical evidence presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
Preclinical data demonstrated significant and durable functional improvement in a humanized DMD mouse model. PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein broadly across multiple muscles, including cardiac and skeletal muscles, and edited dystrophin mRNA transcript in muscle satellite stem cells, supporting long-term durability.
The company is targeting an Investigational New Drug (IND) and/or Clinical Trial Application (CTA) filing for PBGENE-DMD in 2025, with clinical data anticipated in 2026. This program addresses up to 60% of DMD patients, representing a significant unmet medical need.
To accelerate PBGENE-DMD and maintain operational capability for its lead PBGENE-HBV program, Precision plans to pause development of PBGENE-3243 for m.3243-associated mitochondrial disease. This strategic allocation of resources aims to progress the two lead wholly-owned programs through Phase 1 clinical readouts.
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