Axovant and Benitec estbalish gene therapy partnership

Pharmaceutical company Axovant Sciences has signed a gene therapy licensing and development deal with Australia-based Benitec Biopharma to expand its pipeline of products.

Under the terms of the collaboration, Axovant will have exclusive global licensing rights to Benitec’s Silence-and-Replace gene therapy programme being developed to treat oculopharyngeal muscular dystrophy (OPMD).

The investigational gene therapy technology could deliver DNA-directed RNA interference, which represents silence, and a functional copy of the gene, which represents replace, within a single vector construct.

The Silence-and-Replace technique can also be applied for other genetic diseases such as the autosomal dominant disorders caused due to nucleotide repeat expansion.

The lead programme using this approach is AXO-AAV-OPMD, which is currently in pre-clinical development, but Axovant intends to launch its placebo-controlled clinical study next year.

The partnership also involves the research and development of five gene therapy products across the neurological disorders area.

Axovant Sciences CEO Pavan Cheruvu said: "This expansion of Axovant’s pipeline further demonstrates our commitment to advancing innovative gene therapies for serious neurological diseases."

"This expansion of Axovant’s pipeline demonstrates our commitment to advancing innovative gene therapies for neurological diseases."

As part of the deal, Axovant will make an upfront payment of $10m to Benitec, which will also be eligible for additional payments based on development, regulatory and commercial sales milestones.

Benitec will also receive 30% of net profits on worldwide sales of AXO-AAV-OPMD and tiered royalties on the other gene therapy products resulting from the collaboration.

Benitec Biopharma executive chairman Jerel Banks said: "This agreement with Axovant further demonstrates the importance of Benitec’s core technology and our strategic focus on rapidly progressing these programmes into the clinic.

"We believe Axovant is the ideal partner to take these programs forward, and look forward to working closely with them to develop AXO-AAV-OPMD and other neurological gene therapies."

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