Understanding Gene Therapy
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.