Sarepta partners with Myonexus to treat severe forms of muscular dystrophies

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Sarepta Therapeutics has entered into an exclusive license agreement with Myonexus Therapeutics, a spinout of Nationwide Children’s Hospital, Center for Gene Therapy to develop gene therapies for various forms of Limb-girdle muscular dystrophies.

Sarepta Therapeutics, a biopharmaceutical company focused on developing precision genetic medicine to treat rare neuromuscular diseases, has announced that it has entered into an exclusive partnership with Myonexus Therapeutics.

Founded in 2017 and headquartered in Ohio, Myonexus Therapeutics is focused on developing gene therapies for rare diseases and is the first company to develop a treatment for Limb-girdle muscular dystrophies (LGMDs). Its development pipeline is based on research conducted by Dr Louise Rodino-Klapac at the Center for Gene Therapy at Nationwide Children’s Hospital. She is the chief scientific officer and co-founder of Myonexus.

Under the terms of the licensing agreement, Myonexus will receive an upfront payment of $60 million from Sarepta. Sarepta will also make additional development-related milestone payments and has the option to purchase to acquire Myonexus. If all development-related milestone payments are met, Sarepta will make payments of up to $45 million over an approximately two-year evaluation period.

Myonexus Therapeutics’ pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L). The lead program LGMD2E, is intended to treat beta-sarcoglycanopathy, a severe and debilitating form of LGMD characterized by progressive muscle fiber loss. The therapy uses an AAVrh.74 vector system to deliver the gene coding for beta-sarcoglycan protein. Based on the promising pre-clinical safety and efficacy data obtained for LGMD2E, Myonexus is planning to begin the Phase 1/2a study in mid-2018.

AAVrh.74 vector system is also being used in the micro-dystrophin gene therapy program Sarepta is developing in partnership with Nationwide Children’s Hospital for treating Duchenne muscular dystrophy. Dr Rodino-Klapac is one of the principal investigators of the micro-dystrophin program.

Dr. Rodino-Klapac stated: “The culmination of this partnership with Sarepta is important to Myonexus’ mission of rapidly advancing our LGMD pipeline. As the inventor of Myonexus’ LGMD approach and co-inventor of the DMD gene therapy approach, both of which utilize the AAVrh.74 vector system, we are excited to leverage this knowledge from our work in DMD and now apply it to our LGMD portfolio.”

Source: Sarepta Therapeutics Announces Partnership with Myonexus Therapeutics for the Advancement of Multiple Gene Therapy Programs Aimed at Treating Distinct Forms of Limb-Girdle Muscular Dystrophies; Press Release