精密生物科学公司宣布公布PBGENE-MED的临床前数据、Duchenne肌营养不良症（DID）的体内基因编辑方法、预计2025年提交临床试验申请，预计2026年临床数据

2025-07-16 19:05

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