Myonexus Therapeutics Secures $2.5 Million Seed Financing to Clinically Advance Limb-Girdle Muscular Dystrophy (LGMD) Gene Therapies

MYO-101 Phase 1/2a systemic gene therapy trial for LGMD2E to begin in early 2018

New Albany, OH, 13 December 2017Myonexus Therapeutics, Inc. (Myonexus), a clinical-stage biotechnology company developing transformative gene therapies for limb-girdle muscular dystrophies (LGMDs), today announced the completion of a $2.5 million seed financing. The company is committed to accelerating development of five gene therapies pioneered within the lab of Louise Rodino-Klapac, Ph.D. and under clinical guidance by Jerry Mendell, M.D., each a Principal Investigator at Nationwide Children’s Hospital Center for Gene Therapy in Columbus, Ohio, USA.  CincyTech, LLC, Rev1 Ventures, The Jain Foundation, and GFB ONLUS joined initial investors from the LGMD community.

The seed financing will enable Myonexus to initiate a systemic Phase 1/2a clinical trial of MYO-101, the company’s gene therapy candidate for treating LGMD2E (beta-sarcoglycanopathy), in early 2018.  Myonexus’ pipeline also includes MYO-102, a gene therapy candidate for LGMD2D (alpha-sarcoglycanopathy) currently completing a Phase 1/2a clinical trial, MYO-201, a gene therapy candidate for LGMD2B (dysferlinopathy) currently in Phase 1, MYO-103, a preclinical gene therapy candidate for LGMD2C (gamma-sarcoglycanopathy), and MYO-301, a preclinical gene therapy candidate for LGMD2L (anoctamin 5).

“MYO-101’s compelling preclinical data strongly supported the case for clinical translation, validated by our own subsequent clinical trial results as well as general advances in the neuromuscular disease gene therapy field,” said Michael Triplett, PhD, Myonexus’ President and Chief Executive Officer. “We are committed to rapidly advancing our pioneering pipeline of gene therapy candidates, with the ultimate goal of providing first-ever corrective LGMD treatments, and we plan to initiate the Phase 1/2a study of MYO-101 next year.”

“We have a history of investing in world class pediatric innovations in partnership with Cincinnati Children’s Hospital,” said John Rice, PhD, Director of Life Sciences at CincyTech. “We are pleased to find another investment with an immediate and lasting impact on children living with these challenging and rare diseases. Nationwide Children’s deserves great credit for developing this technology in house and proving it out clinically.”

“There are currently no approved treatments for the limb-girdle muscular dystrophies.  Initial LGMD human clinical studies demonstrated expression in muscles exposed to MYO-102 and MYO-201. MYO-101’s Phase 1/2a clinical trial represents the first intravenous systemic exposure, potentially providing the first evidence of functional improvements in LGMD patients following treatment with these gene therapies,” said Bruce Halpryn, PhD, Myonexus’ Chief Operating Officer.

“With a ground-breaking approach Myonexus is poised to transform the way limb-girdle muscular dystrophy is treated, and we are thrilled to help accelerate their growth,” said Tom Walker, CEO of Rev1 Ventures. “This news underscores the critical role that the Research Institute at Nationwide Children’s Hospital is playing in advancing drug development research and identifying partners with the determination and ability to commercialize these discoveries for the ultimate benefit of patients. We believe the partnership with Myonexus is setting the stage for big things in gene therapy.”

“The Jain Foundation is excited to continue its support of the dysferlin gene therapy technologies emerging from Nationwide Children’s and be part of the clinical development efforts happening at Myonexus.  We believe gene therapy has great potential to be the first effective therapy for LGMD2B patients and to dramatically improve their quality of life,” said Doug Albrecht, PhD, Co-President of the Jain Foundation Inc.

About Myonexus Therapeutics

Myonexus Therapeutics is a clinical stage, rare disease gene therapy company developing first ever treatments for Limb-girdle muscular dystrophies (LGMDs) based on research at Nationwide Children’s Hospital, a leader in neuromuscular gene therapy discovery and translational research.  Myonexus Therapeutics’ pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2LO).  Founded in 2017, Myonexus is headquartered in New Albany, Ohio. More information is available at

About CincyTech LLC

CincyTech, one of the Midwest’s most active seed investors, helps to transform innovation into high performing life science and digital companies in Southwest Ohio. Our team provides advice and seed capital to entrepreneurs, helps research institutions commercialize technology through startups, and catalyzes investment from individuals and institutions into regional companies. Learn more at

About Rev1 Ventures

Rev1 is a venture fund that helps entrepreneurs build great companies. Combining investment capital with a unique blend of services through our startup studio, we propel innovation for startups and corporate innovation teams. Our seasoned, data-driven team helps lay the foundation for scalable growth with the skills to evolve a product, sell to customers, and build the right team. Named a top VC investor in the Great Lakes Region, Rev1 manages a continuum of financial support from corporate and community partners, as well as the Ohio Third Frontier. Rev1 was named the Most Active VC in Ohio in 2017 by CB Insights. For more information, visit

About the Jain Foundation

The Jain Foundation is a privately funded nonprofit foundation focused on finding a cure for muscular dystrophies caused by dysferlin deficiency (LGMD2B/Miyoshi Myopathy).  The Jain Foundation supports academic research, conducts independent studies with contract research organizations, and supports clinical studies in pursuit of this mission.  The foundation also assists patients in receiving a confirmed genetic diagnosis of dysferlinopathy, and maintains a patient registry for the disease.

Media Inquiries:

Michael Triplett, Ph.D.
President and CEO, Myonexus Therapeutics, Inc.
+1 614.477.5445

Peg Rusconi

Aimee Eichelberger
Rev1 Ventures

Douglas E. Albrecht, PhD.
Jain Foundation Inc.