Myogene Bio LLC

Myogene Bio LLC is a woman-owned small business biotechnology company located in San Diego, California, specializing in advanced gene editing therapies with a primary focus on rare genetic disorders, particularly Duchenne muscular dystrophy (DMD). Registered in SAM.gov since August 2018, the company operates as a limited liability partnership and has positioned itself as an innovative research entity developing cutting-edge gene editing technologies using CRISPR/Cas9 approaches. The company leverages technical expertise in adeno-associated virus (AAV) vector technologies and molecular biology to advance therapeutic interventions for genetic muscle disorders.

Myogene Bio LLC has secured multiple federal grant awards from prestigious research agencies, including the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Center for Advancing Translational Sciences, and the Defense Health Agency. These grants, totaling approximately $1.2 million, support research focused on developing gene editing therapies for Duchenne muscular dystrophy. Specifically, the company's research involves optimizing AAV vector manufacturing processes, conducting preclinical studies in humanized mouse models to assess gene editing efficacy, and exploring innovative approaches to restore dystrophin protein function. The research aims to develop a dual AAV vector gene editing therapy that can delete specific DMD gene exons, potentially offering treatment for approximately 50% of DMD patients by restoring the gene's reading frame and generating a functional dystrophin protein. Each grant supports different aspects of research, including manufacturing process optimization, biomarker assessment, and long-term durability studies of the gene editing therapy.