– New preclinical data show PBGENE-DMD drove up to a 3x higher dystrophin protein restoration in skeletal muscle and up to 12x higher in respiratory muscle in early-juvenile mice compared to late-juvenile mice –

– Findings further support evaluation of PBGENE-DMD in DMD patient populations as young as 2 years of age –

– Greater efficacy with earlier intervention further differentiates PBGENE-DMD from microdystrophin approaches –

Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced new preclinical data from its PBGENE-DMD program in an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting in Boston, Massachusetts. The new data show that treatment with PBGENE-DMD in early-juvenile mice resulted in significantly higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. This new data further supports evaluating PBGENE-DMD in younger DMD patient populations, including the 2- to 3-year-old patients, who are a key demographic of the ongoing Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD in boys ages 2 to 7.

The greater efficacy observed in early-juvenile mice is also expected to be an important point of differentiation for PBGENE-DMD versus microdystrophin approaches. With microdystrophin approaches the AAV dilution effect driven by muscle growth and turnover would be expected to be even more pronounced in younger DMD patients.