Genetics

Axovant Sciences (NASDAQ:AXON) today announced that it has licensed exclusive global rights to an investigational Silence-and-Replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy (OPMD), and has also entered into a research collaboration for the development of five additional gene therapy products in neurological disorders. As quoted in the press …

Axovant Sciences (NASDAQ:AXON) today announced that it has licensed exclusive global rights to an investigational Silence-and-Replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy (OPMD), and has also entered into a research collaboration for the development of five additional gene therapy products in neurological disorders.

As quoted in the press release:

The Silence-and-Replace gene therapy technology is designed to deliver a combination of DNA-directed RNA interference (silence) along with a functional copy of the gene (replace) in a single vector construct. This approach is applicable to various genetic diseases, including autosomal dominant disorders caused by nucleotide repeat expansion.

The lead program, AXO-AAV-OPMD, is in preclinical development, and Axovant plans to initiate a placebo-controlled clinical study in 2019. OPMD is a neuromuscular disease that is caused by mutations in the gene coding for polyA-binding protein nuclear 1 (PABPN1), which can lead to formation of intranuclear inclusion bodies causing muscle cell pathology. Patients with OPMD may have swallowing difficulties with potentially life-threating consequences, including malnutrition and aspiration pneumonia.  OPMD is estimated to affect at least 15,000 patients in North America and Europe, and there are no products approved for treatment of the disease. AXO-AAV-OPMD is an adeno-associated viral (AAV) vector gene therapy delivered via a one-time intramuscular administration, which both silences the mutant PABPN1 gene and replaces it with a functional copy. The U.S. Food & Drug Administration and European Commission have granted Orphan Drug Designation to AXO-AAV-OPMD for the treatment of OPMD.

Click here to read the full press release.

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