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Precision Biosciences Announced dditional Preclinical Data For PBGENE-DMD, In Vivo Gene Editing Approach For Duchenne Muscular Dystrophy (DMD), Clinical Trial Application Submission Expected In 2025 With Clinical Data Expected In 2026

Author: Benzinga Newsdesk | July 16, 2025 07:05am

New preclinical data for the PBGENE-DMD final clinical candidate demonstrates an increase in dystrophin positive muscle cells across key muscle types, potentially driven by editing of muscle satellite cells.
PBGENE-DMD is a first-in-class in vivo gene editing approach for up to 60% of Duchenne Muscular Dystrophy patients, specifically those impacted by dystrophin mutations in the ‘hot spot' region between exons 45-55.
Precision targeting to submit an Investigational New Drug (IND) and/or Clinical Trial Application (CTA) for PBGENE-DMD in 2025 with clinical data expected in 2026

Posted In: DTIL