Precision BioSciences accelerates PBGENE-DMD, plans IND/CTA in 2025; pauses PBGENE-3243

PRECISION BIOSCIENCES INC

PBGENE-DMD is a first-in-class in vivo gene editing approach targeting exons 45-55; up to 60% of DMD patients carry mutations in this hotspot region.
Preclinical data shows durable functional improvement in a humanized DMD mouse model over 9 months with restored dystrophin expression.
Precision targets to file IND/CTA for PBGENE-DMD in 2025, with clinical data expected in 2026.
Company pauses development of PBGENE-3243 (m.3243 mitochondrial disease) to prioritize PBGENE-DMD and HBV; cash runway sufficient for both through Phase 1.
PBGENE-DMD preclinical data presented at ASGCT Annual Meeting on May 14, 2025; webcast held May 15.

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