Precision BioSciences (NASDAQ: DTIL) has announced that the FDA has granted a Study May Proceed notification, allowing initiation of clinical trial site activation for the Phase 1/2 FUNCTION-DMD trial of PBGENE-DMD in ambulatory Duchenne muscular dystrophy (DMD) patients at specialized clinical sites.
The objective of the study is to evaluate safety, tolerability and efficacy, including dystrophin protein expression and functional outcomes in patients afflicted with DMD.
In a statement, Aravindhan Veerapandiyan, MD, Director, Comprehensive Neuromuscular Program, Arkansas Children’s Hospital, University of Arkansas for Medical Sciences, commented, “DMD is a progressive disorder caused by mutations in the dystrophin gene that disrupt the production of the functional protein, resulting in continuous muscle degeneration. PBGENE-DMD is designed to restore near full-length dystrophin, with the potential to provide significant functional benefits. This is an important milestone, and I look forward to participating in the Phase 1/2 FUNCTION-DMD clinical study to evaluate a gene excision approach in DMD patients with mutations involving exons 45-55.”
