Precision BioSciences announces additional PBGENE-DMD preclinical data

Precision BioSciences (DTIL) announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the company’s in vivo gene editing approach for Duchenne muscular dystrophy, towards first-in-human clinical investigation. The final IND-enabling toxicology studies are currently underway with IND and/or CTA filing targeted in 2025 and initial clinical data expected in 2026. The Company believes that its current cash runway will be sufficient to progress both PBGENE-HBV, its current Phase 1 asset, and PBGENE-DMD through Phase 1 clinical readouts. “In a long-term durability study in a DMD diseased mouse model, we have observed up to a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius, heart, and diaphragm. In the gastrocnemius, up to 85% of cells were dystrophin-positive,” said Cassie Gorsuch, chief scientific officer at Precision BioSciences. “These new data build upon the preclinical data shared at ASGCT in May 2025, demonstrating that PBGENE-DMD treatment resulted in significant and sustained improvement of maximum force output at the same three- and nine-month timepoints. This broad increase in dystrophin-positive cells, along with the increased dystrophin protein detected in tissues, further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells, which were also observed in this study. We believe these results demonstrate the unique potential of the PBGENE-DMD gene editing approach to produce a sustained functional benefit without the need for AAV persistence, which is required of microdystrophin approaches. We look forward to presenting the complete dataset at a future scientific conference. Given the totality of preclinical evidence, we remain excited and steadfast in advancing this program towards clinic.”