Myonexus Therapeutics

Myonexus Therapeutics, Inc. operated as a clinical-stage biotechnology firm specializing in the development of gene therapies for various forms of Limb-Girdle Muscular Dystrophies (LGMDs). The company was established in 2017 as a spinout from the Research Institute at Nationwide Children's Hospital, a center with deep expertise in neuromuscular gene therapy. Its founding team included Michael Triplett, Ph.D., who served as President and CEO, Bruce Halpryn, Ph.D., as COO, and Louise Rodino-Klapac, Ph.D., as Chief Scientific Officer. Dr. Rodino-Klapac was the primary inventor of the company's portfolio of LGMD candidates, developed in collaboration with Dr. Jerry Mendell. Their extensive research, spanning over a decade at Nationwide Children's Hospital, formed the scientific bedrock of Myonexus. Dr. Triplett brought experience in specialty pharmaceuticals and new ventures, having previously been the CEO of N8 Medical, Inc.

The company's core business focused on advancing a pipeline of five gene therapy candidates designed to address different genetic subtypes of LGMD, a group of rare, degenerative neuromuscular diseases. These conditions cause progressive muscle wasting, often leading to severe disability and early mortality. Myonexus’s therapeutic strategy involved using an adeno-associated virus vector, specifically AAVrh.74, to deliver a correct copy of the faulty gene to a patient's cells. This vector was selected for its ability to target both skeletal and cardiac muscle, addressing the systemic nature of the disease. The pipeline included programs for LGMD types 2E, 2D, 2B, 2C, and 2L, with three having reached the clinical trial stage at the time of its acquisition.

Myonexus Therapeutics' business model was centered on the research and development of these high-value gene therapies, with a clear path toward commercialization. In December 2017, the company secured $2.5 million in seed funding from investors including CincyTech and Rev1 Ventures to advance its lead candidate, MYO-101 for LGMD2E, into clinical trials. A pivotal milestone occurred in May 2018 when Myonexus entered into a strategic partnership with Sarepta Therapeutics. Sarepta made an upfront payment of $60 million for an exclusive option to acquire Myonexus. This collaboration provided the necessary capital and strategic alignment to accelerate the development of all five LGMD programs. Following positive interim clinical trial results for MYO-101 in February 2019, Sarepta exercised its option and acquired Myonexus outright for $165 million, integrating the promising LGMD pipeline into its broader genetic medicine portfolio.

Keywords: Myonexus Therapeutics, gene therapy, Limb-Girdle Muscular Dystrophy, LGMD, Sarepta Therapeutics, Michael Triplett, Louise Rodino-Klapac, Nationwide Children's Hospital, AAVrh.74 vector, rare disease, neuromuscular disorders, clinical-stage biotechnology, genetic medicine, MYO-101, LGMD2E, LGMD2D, LGMD2B, beta-sarcoglycanopathy, muscular dystrophy research, CincyTech, Rev1 Ventures, gene therapy development, orphan drugs