Precision BioSciences starts DMD gene trial
- Precision BioSciences dosed the first patient in the Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD for Duchenne muscular dystrophy.
- The trial is enrolling ambulatory DMD patients aged 2 to 7 with mutations between exons 45 and 55.
- Precision BioSciences expects to report initial safety data from the study by year-end 2026.
Precision BioSciences (NASDAQ:DTIL) announced that the first patient has been dosed in the Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD for Duchenne muscular dystrophy.
The clinical trial follows the company’s development of PBGENE-DMD, an in vivo gene editing program designed to edit the dystrophin gene and target mutations affecting a key hotspot region.
PBGENE-DMD uses two ARCUS nucleases delivered through a single AAV vector to remove exons 45-55 of the dystrophin gene, with the company stating that the approach is designed to restore a near full-length functional dystrophin protein.
Precision BioSciences stated that initial safety data from FUNCTION-DMD is expected by year-end 2026.
The Phase 1/2 FUNCTION-DMD trial is enrolling ambulatory DMD patients between ages 2 and 7 across multiple U.S. clinical sites, with eligibility focused on mutations between exons 45 and 55.
Duchenne muscular dystrophy is a genetic disorder caused by mutations affecting dystrophin production, leading to progressive muscle weakness and functional decline, with current research focused on developing therapies targeting the underlying genetic cause.
