Agreement Myonexus - Sarepta

GFB Onlus Announces

that Myonexus, a Biotechnology Company in which it has Invested, Signed an Exclusive Partnership with Sarepta Therapeutics for the Advancement of Multiple Gene Therapy Programs Aimed at Treating Distinct Forms of Limb-Girdle Muscular Dystrophies

On May 3rd, 2018 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, and Myonexus Therapeutics, Inc., a clinical-stage biotechnology company developing transformative gene therapies for various forms of Limb-girdle muscular dystrophies (LGMDs), entered into an exclusive partnership to advance multiple gene therapy programs aimed at treating distinct forms of LGMDs.


GFB Onlus took part at the signing of this agreement between Sarepta and Myonexus, two biotechnology companies headquartered in the United States. GFB Onlus financed most of the pre-clinical work on MYO-101.

Last June, GFB Onlus of Talamona, province of Sondrio, along with four other American organizations, joined Myonexus Therapeutics to support developing gene therapies for LGMD. For years, GFB Onlus has been working to expeditiously bring gene therapy to Europe, and in particular to Milan. Currently, it’s the only European authority participating in the company.


Under the terms of the agreement with Myonexus, Sarepta will make an upfront payment of $60 million and additional development-related milestone payments to purchase an exclusive option to acquire Myonexus at a pre-negotiated, fixed price with sales-related contingent payments. If all development-related milestone payments are met, Sarepta will make payments of up to $45 million over an approximately two-year evaluation period. Sarepta has the option to purchase Myonexus at any time, including upon review of proof-of-concept data.

"We are excited about this agreement, which we did not expect. The involvement of the American biopharmaceutical company - underlines Beatrice Vola, president of GFB Onlus - certainly represents another step forward for the gene therapy project on LGMD2E. We are hopeful that the collaboration between Myonexus and Sarepta will lead to even faster results. We have been striving for years to give our associates concrete feedback and finally we think we are on the right track.”


The program is designed, if successful, to offer first-ever corrective treatments for five distinct forms of LGMD. The most advanced of these programs is MYO-101, which is being developed to treat LGMD2E. MYO-101 has generated strong pre-clinical safety and efficacy data utilizing the AAVrh.74 vector system, the same vector used in the micro-dystrophin gene therapy program Sarepta is developing with Nationwide Children’s Hospital. A Phase 1/2a study of MYO-101 is scheduled to begin in mid-2018. The companies plan to report on 60-day biopsy data in late-2018 or early 2019. Additionally, Myonexus is advancing MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L.  Like MYO-101, all programs rely upon transfecting a restorative gene utilizing the AAVrh.74 vector.


Quoting from the May 3rd press release, Mr. Doug Ingram, president and chief executive officer of Sarepta, stated:

"We are very excited to enter a partnership with an option to acquire Myonexus. Myonexus and its focus on gene therapy using the AAVrh.74 vector to treat forms of LGMD aligns brilliantly with our vision to emerge as one of the most meaningful global precision genetic medicine companies by focusing on the use of genetic medicine to improve the lives of those with rare fatal diseases. We are also delighted to extend our collaboration with Dr. Rodino-Klapac, a renowned thought leader in the field of gene therapy. We share her dedication to rapidly bring life-changing medicines to children suffering and dying from genetic disease.”


Dr. Rodino-Klapac is a principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital and an associate professor at the Ohio State University.  She has dedicated her research program to developing gene therapies treatment for muscular dystrophies.  She is also the Chief Scientific Officer for Myonexus and has worked in close collaboration with Dr. Jerry Mendell for well over a decade to bring therapies to the clinic.

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