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Agreements

Date: 2017-06-05

Type of information: Establishment of a new subsidiary in the US

Compound:

Company: Myonexus Therapeutics (USA - OH)

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Type agreement: establishment of a new company in the US

Action mechanism:

Disease:

Details:

  • • On June 5, 2017, Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies for limb-girdle muscular dystrophies (LGMDs), announced its launch plans. The company is committed to accelerating development of five experimental gene therapies pioneered within the laboratories of Louise Rodino-Klapac, Ph.D. and Jerry Mendell, M.D., Principal Investigators at the Research Institute at Nationwide Children’s Hospital Center for Gene Therapy in Columbus, Ohio, USA.
  • Myonexus Therapeutics’ pipeline includes Phase I programs for LGMD2D (-sarcoglycanopathy) and LGMD2B (dysferlinopathy). Additionally, the company will advance its LGMD2E (-sarcoglycanopathy) program into the clinic by late 2017 and simultaneously will advance two preclinical programs targeting LGMD2C (-sarcoglycanopathy) and LGMD2L (anoctamin 5). The Research Institute at Nationwide Children’s will manufacture Phase I clinical supplies and will be the site of first in-human Phase I clinical trials.
  • Joining Dr. Triplett on the leadership team are Bruce Halpryn, Ph.D., Chief Operating Officer, and Dr. Rodino-Klapac, Chief Scientific Officer. Providing expertise in specialty pharmaceuticals, drug delivery innovation and new ventures, Dr. Triplett previously served as Chief Executive Officer of N8 Medical, Inc. and as an executive at Battelle. Dr. Halpryn previously led early phase R&D, EU regulatory affairs, and technology scouting for Procter & Gamble Pharmaceuticals, overseeing development of numerous successful drug development programs. Dr. Rodino-Klapac is a recognized leader in LGMD and Duchenne muscular dystrophy gene therapy discovery and development. Dr. Mendell, a recognized global leader in clinical gene therapy and muscular dystrophy therapeutic development, will serve as a clinical development consultant to Myonexus.
  • The LGMD community is providing Myonexus with initial financing, scientific contributions, and patient engagement assistance.

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