Sarepta Therapeutics exercised its option to acquire Nationwide Children’s gene therapy spinout Myonexus Therapeutics after paying at least $60m for the right in May 2018.

Myonexus Therapeutics, a US-based gene therapy developer spun out of Nationwide Children’s Hospital, an affiliate of Ohio State University, is to be acquired by rare disease medicine producer Sarepta Therapeutics for $165m. The deal is subject to the fulfilment of certain closing conditions. Founded in 2017, Myonexus Therapeutics is working on five gene therapies for different variants of limb-girdle muscular dystrophy (LGMD), a group of conditions characterised by muscle deterioration in the hips and shoulders that spreads into the arms and shoulders. Three of the gene therapies are in clinical development, and Sarepta was expected to present initial clinical results from the furthest advanced of these, MY-101 for the 2E subtype of LGMD on February 27. All of Myonexus’s candidates exploit a viral delivery vector called AAVrh74 that is thought to robustly target cardiac and skeletal muscle without inadvertently penetrating the blood-brain barrier. AAVrh74 is also utilised in a micro-dystrophin gene therapy collaboration between Sarepta and Nationwide Children’s Hospital which shares inventors with Myonexus, and both programs have achieved similar safety outcomes during pre-clinical studies. Sarepta took an option to acquire Myonexus in May 2018 in exchange for a $60m upfront payment, plus development milestones potentially worth $45m over two years. The spinout previously closed a $2.5m seed round in December 2017 backed by public-private seed fund CincyTech, Rev1 Ventures and Jain Foundation. Doug Ingram, president and CEO of Sarepta, said: “The five LGMD gene therapies being developed fit brilliantly with Sarepta’s mission to develop therapies with the potential to rescue the lives of patients with serious life-limiting rare genetic diseases.”

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