Myonexus Launches Development of Gene Therapies Targeting Limb-Girdle MD

A new clinical-stage biotech, Myonexus Therapeutics, is developing gene therapies for limb-girdle muscular dystrophies (LGMDs).

Launch plans include the accelerated development of five investigational gene therapies that have the potential to become the first standard-of-care therapies for LGMDs.

Myonexus’ pipeline includes three clinical-stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B), and two preclinical gene therapy programs (LGMD2C and LGMD2L).

LGMD2D (alpha sarcoglycanopathy) and LGMD2B (dysferlinopathy) are in Phase 1 development, while LGMD2E (beta-sarcoglycanopathy) is expected to advance into the clinic later this year.

The two preclinical programs targeting LGMD2C (gamma-sarcoglycanopathy) and LGMD2L (anoctamin-5) also are to be advanced simultaneously.

The first-in-human Phase 1 trials will be held at the Research Institute at Nationwide Children’s Hospital Center for Gene Therapy in Columbus, Ohio.

The limb-girdle muscular dystrophy community is supporting Myonexus with initial financing, support in scientific developments, and patient engagement through the nonprofit LGMD2D Foundation.The foundation is working to expedite the development of a cure or therapy for LGMD2D.

Myonexus also announced the addition of Bruce Halpryn, PhD, as CEO, and Louise Rodino-Klapac, PhD, as chief scientific officer.

The gene therapies were developed by the research team of Rodino-Klapac and Jerry Mendell, MD, principal investigators at the Center for Gene Therapy.

“Myonexus Therapeutics’ highly innovative experimental gene therapies offer potentially transformative quality of life improvements for limb-girdle muscular dystrophy patients,” Michael Triplett, PhD, president and CEO of Myonexus, said in a press release. “We are committed to advancing the trailblazing work of Dr. Rodino-Klapac, Dr. Mendell, and the entire Research Institute at Nationwide Children’s Hospital Center for Gene Therapy team.”

Mendell said the researchers in the Center for Gene Therapy are excited to help Myonexus launch products that show promise in the laboratory.

“Our past performance of clinical success in neuromuscular gene therapy clinical trials, combined with our world-class research gene therapy capabilities, is consistent with and validates our long-term vision and strategy to position Nationwide Children’s Hospital and Columbus, Ohio, as a global leader in addressing clinical unmet needs associated with neuromuscular diseases,” Mendell said.

Bryan Barber, co-founder of Myonexus Therapeutics and president of the LGMD2D Foundation, said the global LGMD community is excited to support the efforts of Myonexus. “We look forward to transforming the lives of LGMD patients throughout the world,” he said.

“As we have seen in recent cases, strong patient advocacy support is critical to the success of rare disease companies. I am thrilled to support the company and to join its board of directors,” he added.