Core Insights - Precision BioSciences announced that new preclinical study data for PBGENE-DMD will be presented at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference, highlighting its potential safety and long-term efficacy [1] Company Overview - Precision BioSciences, Inc. is a clinical-stage gene editing company utilizing its proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet needs [1] - The company has received Investigational New Drug clearance from the U.S. FDA for PBGENE-DMD, allowing the initiation of clinical trial site activation [1] Product Details - PBGENE-DMD is a first-in-class gene editing therapy designed to provide durable functional muscle improvement for Duchenne Muscular Dystrophy (DMD) patients with mutations between exons 45 and 55, affecting up to 60% of boys with DMD [1] - The therapy employs a gene excision approach, differentiating it from existing treatments like microdystrophin and exon skipping [1] - Preclinical studies demonstrated significant and durable functional improvements in a humanized DMD mouse model, restoring production of a near full-length functional dystrophin protein across multiple muscle types [1] Clinical Development - The upcoming presentation at the MDA conference will focus on the abstract titled "PBGENE-DMD gene editing treatment leads to safe and long-term functional improvement in humanized DMD-disease mouse model" [1] - The presentation is scheduled for March 9, 2026, and will be a poster presentation [1]
Precision BioSciences Announces Presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference