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GEN News Highlights : Jul 30, 2013

ReGenX, Audentes Team Up to Develop Rare Muscle Disease Treatments

ReGenX Biosciences is pairing up with Audentes Therapeutics to develop and commercialize products to treat X-linked myotubular myopathy (XLMTM)—a rare inherited disorder caused by mutations in the MTM1 gene—and Pompe disease using NAV vectors. Per the agreement, ReGenX granted Audentes an exclusive worldwide license to ReGenX' NAV rAAV8 and rAAV9 vectors for treatment of XLMTM and Pompe disease in humans, plus certain sublicensing rights. In return, ReGenX is receiving an undisclosed upfront payment, certain milestone fees, and royalties on net sales of products incorporating NAV rAAV8 and rAAV9.

According to ReGenX, NAV technology is based on NAV vectors, which the firm describes as the next generation of recombinant adeno-associated virus (rAAV) vectors used for in vivo therapeutic gene delivery. NAV vectors are able to enter cells and deliver their DNA contents to the nucleus, and are designed to be unable to replicate. When used for in vivo gene transfer, these NAV vectors can potentially give stable, long-term gene expression at therapeutic levels, the firm said. There are more than 100 NAV vectors, the most common of which include NAV rAAV8, NAV rAAV9, and NAV rAAVrh.10.

“We believe this exclusive license agreement is important to the successful development of NAV-based gene delivery treatments for patients with XLMTM and Pompe disease,” said Ken Mills, president and CEO of ReGenX.

“Audentes is committed to the development of new treatments for patients with XLMTM and Pompe disease using AAV gene therapy technology and we feel rAAV8 and rAAV9 are the most promising vectors to achieve this goal,” said Matthew R. Patterson, president and CEO of Audentes.