Clinical Stage LGMD Gene Therapy
Therapies in Development

Understanding Gene Therapy

The first-ever gene therapy candidates to treat five sub-types of Limb Girdle Muscular Dystrophy.

Myonexus aims to deliver the first-ever corrective treatments for LGMD through the promise of gene therapy technology. Gene therapy is ideally suited to address rare genetic diseases based on its precision delivery of genetic material.

Gene therapy delivers a healthy copy of the compromised gene directly into the affected muscle cells – enabling the body to permanently produce healthy cells.

Our gene therapy technology uses a viral vector – called the adeno-associated virus, or AAV – to deliver these genes into the target cells. This is a virus that doesn’t cause any human disease – it’s a delivery vehicle for functional genes to override the faulty genetic material.

Through an intravenous injection, the AAV—armed with updated genetic information—travels through the bloodstream to the affected muscle cells, where the AAV is uncoated to reveal the corrected DNA.

The gene therapy constructs vary according to the disease sub-types. In the case of LGMD2E, the muscle cells subsequently read the new DNA and begin producing the missing muscle protein, which works with cellular material to support a strong link between the cell and surrounding muscle matrix. This stabilizes the cellular membrane to protect it from ordinary muscular wear and tear, and restores the muscle tissue’s ability to repair and grow.

Using this advanced, precision gene therapy approach, the Myonexus candidates have the potential to correct the underlying genetic defect that causes these debilitating diseases. In fact, if diagnosed and treated early enough in the patient’s course of disease, gene therapy may have the potential to preserve and promote functional ability and, if diagnosed at birth, may even help prevent the onset of debilitating symptoms.

The Myonexus Gene Therapy Vector

The adeno-associated viral vector used in the Myonexus pipeline of candidates, AAAVrh.74, was selected based on more than a decade of research in the lab of L. Rodino-Klapac at Nationwide Children’s Hospital. It is designed to optimize gene expression in all relevant muscles in order to address the underlying mechanisms of specific sub-types of LGMD. Research to date indicates the vector offers effective and safe penetration of target muscle tissue important in LGMD, with ideal systemic biodistribution and gene expression approaching 100% in preclinical testing.

Our Pipeline

The first-ever gene therapy candidates to treat five sub-types of Limb Girdle Muscular Dystrophy.

Program Indication Gene Construct Preclinical Phase 1 IM Phase 1/2a ILP Phase 1/2a ILP
Preclinical Phase 1 IM Phase 1/2a ILP Phase 1/2a ILP
MYO-101¹ for LGMD2E
MYO-102² for LGMD2D
MYO-103 for LGMD2C
MYO-201³ for LGMD2B
MYO-301 for LGMD2L
  1. For MYO-101, FDA granted permission to move directly to a systemic, intravenous (IV) delivery Phase 1/2a study
  2. For MYO-102, FDA required an initial intramuscular (IM) Phase 1 study and an Isolated Limb Perfusion (ILP) Phase 1/2a study to assess safety
  3. For MYO-201, FDA required an initial IM Phase 1 study to assess safety; dual vector design