Also known as: Precision Biosciences, Precision BioSciences
Precision BioSciences is a clinical-stage gene-editing company focused on in vivo therapies for serious diseases. It uses its ARCUS platform to develop programs including candidates for hepatitis B, Duchenne muscular dystrophy, and mitochondrial disease.
Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
A Phase 1 trial of azer-cel, a CAR T-cell therapy using donor T-cells to target B-cells, is recruiting patients with progressive multiple sclerosis at eight U.S. sites. Separately, multiple randomized trials are comparing hematopoietic stem cell transplantation against high-efficacy disease-modifying therapies.
