PBGENE-DMD

PBGENE-DMD is an investigational gene editing therapy developed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a pair of ARCUS nucleases delivered by a single adeno-associated virus (AAV) vector to excise a large mutation "hot spot" region (exons 45-55) in the dystrophin gene, enabling the body to produce a near full-length, functional dystrophin protein. This approach aims to provide more durable and broadly applicable outcomes than current microdystrophin or exon-skipping therapies, potentially addressing up to 60% of DMD patients. Preclinical studies have demonstrated that PBGENE-DMD restores functional dystrophin expression across multiple muscle types—including skeletal muscle, heart, and diaphragm—resulting in significant improvements in muscle resilience and force output. Editing of muscle satellite stem cells suggests potential for permanent correction and long-term therapeutic benefit[1][3][5].

Other names

Keytruda-Precision BioSciences, Inc.-Duchenne muscular dystrophy