Myonexus Therapeutics announces initiation of a gene therapy clinical trial for LGMD2E

On November 8, 2018, Myonexus Therapeutics announced the initiation of a clinical trial evaluating MYO-101, a novel gene therapy candidate for beta-sarcoglycanopathy, also known as limb girdle muscular dystrophy type 2E (LGMD2E). The trial is underway at Nationwide Children’s Hospital’s Center for Gene Therapy in Columbus, Ohio, and Dr. Jerry Mendell is the principal investigator of the study.

“This whole body trial, a first for LGMD, will evaluate the safety and efficacy of this treatment. This trial will generate valuable insights that may help advance other candidates in our pipeline oriented toward other subtypes of LGMD, which we expect to be whole body studies as well,” says Michael Triplette, Myonexus’ chief executive officer. “The first patient has received a single dose of MYO-101, and they are being monitored. Three patients in the first cohort will be dosed this year and preliminary results will be evaluated in early 2019.” Myonexus’ full announcement can be found here.

This is exciting news for the entire LGMD community, and similar gene therapy strategies may have the potential to treat or cure LGMD2A. The Coalition to Cure Calpain 3 Gene Therapy Initiative seeks to accelerate the understanding of gene therapy as a treatment for LGMD2A by funding the research of multiple gene therapy approaches.

Click here to support gene therapy research for LGMD2A!

In addition to gene therapy, C3 will continue to explore and fund other approaches that could lead to therapies that can slow the progression of the disease or cure LGMD2A.

Myonexus Therapeutics announces initiation of a gene therapy clinical trial for LGMD2E