Myonexus Therapeutics (Myonexus) received orphan drug designation for MYO-102, an investigational gene therapy for Alpha-sarcoglycanopathy (LGMD2D) also known as Limb Girdle Muscular Dystrophy Type 2D (LGMD2D). Myonexus is a clinical-stage gene therapy venture development, and is the first-ever correct gene therapies for limb girdle muscular dystrophies (LGMD). It also has licensed the technology from Nationwide Children’s. Nationwide holds the IND for MYO-102.
LGMD2D is a severe, debilitating condition caused by a defect in the gene that produces the alpha-sarcoglycan protein. Lack of function of this protein causes inflammation and progressive loss of muscle fiber, which is replaced with fat and fibrotic scars. LGMD2D is believed to be the most common sarcoglycanopathy, with conservative estimates indicating it affects approximately 3 million people. Presently there is no treatment or cure. Myonexus is developing treatments for LGMDs based on research from Nationwide Children’s Hospital, a leader in neuromuscular gene therapy discovery and translational research. Based in New Albany, OH, Myonexus was founded in 2017 and is developing five gene therapy programs.Source: APNews