Precision BioSciences, Inc. announced on July 16, 2025, updated additional preclinical data for PBGENE-DMD, further validating its scientific rationale for Duchenne muscular dystrophy (DMD). The new data supports the rapid development of this first-in-class in vivo gene editing approach towards first-in-human clinical investigation.
In a long-term durability study in a DMD diseased mouse model, PBGENE-DMD demonstrated up to a three-fold increase in dystrophin-positive muscle cells between three and nine months across key muscle types, including quadricep, gastrocnemius, heart, and diaphragm. Specifically, up to 85% of cells in the gastrocnemius were dystrophin-positive.
These findings build upon previous preclinical data showing significant and sustained improvement of maximum force output. The broad increase in dystrophin-positive cells, along with increased dystrophin protein, further validates improved muscle function and may be attributable to edited satellite cells, which are crucial for long-term durability.
PBGENE-DMD is designed to permanently edit a patient’s own DNA sequence, resulting in naturally produced, near full-length dystrophin protein. The company is currently conducting final IND-enabling toxicology studies, targeting an IND/CTA filing in 2025, with initial clinical data expected in 2026.
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