Myonexus Therapeutics has commenced a Phase I/IIa clinical trial to investigate MYO-101 for the treatment of limb-girdle muscular dystrophy type 2E (LGMD2E).

Conducted in partnership with Nationwide Children’s Hospital, the trial will include children between the age group of four and 15 with significant symptoms of LGMD2E.

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MYO-101 is a new gene therapy currently under development for beta-sarcoglycanopathy or LGMD2E.

As part of the first-in-human, placebo-controlled trial, Myonexus will analyse a single intravenous infusion of MYO-101 among children with LGMD2E.

It intends to assess up to two doses of MYO-101 in two cohorts of nine patients. The placebo arm will be eligible for a cross-over study once the biopsy results are revealed one year of treatment.

“Gene therapy has great potential to deliver functionally curative solutions for challenging rare diseases.”

The trial’s primary objective is baseline measurements, while its secondary objectives are disease progression and registry.

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Last month, Myonexus dosed the first patient in cohort 1 and is expected to complete dosing of all patients within this year. Data from the trial is scheduled to be available next year.

Myonexus Therapeutics president and CEO Michael Triplett said: “By implementing highly precise vector technology, gene therapy has great potential to deliver functionally curative solutions for challenging rare diseases.

“Based on positive pre-clinical results in both efficacy and safety, we are confident in the vector and platform selected for MYO-101 and are hopeful that MYO-101 could be the first profound therapy for patients with LGMD2E.”

LGMD2E is a rare, degenerative and fatal disease caused by a genetic mutation.