Myonexus Therapeutics

Myonexus Therapeutics was a U.S. clinical-stage rare disease gene therapy company focused on developing corrective AAV-based gene therapies for limb-girdle muscular dystrophy (LGMD). The company was formed in 2017 to commercialize technology licensed from Nationwide Children’s Hospital, centered on the AAVrh.74 vector for systemic delivery to skeletal, diaphragm, and cardiac muscle. Its pipeline comprised five LGMD programs: MYO-101 for LGMD2E, MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. In May 2018, Sarepta Therapeutics entered an exclusive partnership with Myonexus, and in February 2019 Sarepta exercised its option to acquire the company for $165 million plus potential sales-related contingent payments. Following the acquisition, Myonexus ceased operating as an independent company and its programs were integrated into Sarepta’s LGMD gene therapy pipeline.