Precision BioSciences Commences Dosing in Phase 1/2 FUNCTION-DMD Study
-First patient dosed with PBGENE-DMD, the first clinical gene-editing program for DMD –
-First patient dosed at Arkansas Children’s Hospital, a PPMD Certified Duchenne Care Center and designated MDA Care Center–
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 the dosing of the first patient in August in the Phase 1/2 FUNCTION-DMD clinical trial evaluating PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
PBGENE-DMD is Precision's wholly owned in vivo gene editing program designed to durably improve function. This novel approach, aiming to restore near full-length dystrophin, is applicable for up to 60% of DMD patients with mutations in a key hot spot region. By employing two complementary ARCUS nucleases in a single AAV, PBGENE-DMD excises exons 45-55 of the dystrophin gene with the aim of restoring a near full-length functional dystrophin protein that more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches.
