RegenxBio Inc. (NASDAQ:RGNX), Washington, D.C. and Biogen Inc. (NASDAQ:BIIB), Cambridge, Massachusetts, have announced the completion of an exclusive worldwide research license agreement allowing Biogen to access RegenxBio’s NAV adeno-associated virus (AAV) AAV8 and AAV9 vectors for achromotopsia and choroideremia. The parties declined to release financial terms but confirmed that upfront payments, fees, milestones and royalties were part of the transaction for the development of gene therapy treatments for the two rare vision disorders. RegenxBio originally obtained rights to the natural AAV isolates from the University of Pennsylvania (UPenn). Last month, UPenn and Biogen Inc. announced a separate collaboration on the development of gene therapies for eye disease, skeletal muscle disorders and CNS conditions. Under the terms of the transaction the University may receive as much as $2 billion in research funding, options and milestone payments, in addition to an initial upfront payment of $20 million with a further $62.5 million in R&D funding over a 3 to 5 year period. The deals reflect an increasing interest in and appetite for the commercialisation of gene therapy products over the past 24 months.
According to information released by RegenxBio, the company has provided Biogen with exclusive research rights, including sub-licensing rights, to the NAV AAV8 and AAV9 vectors to be used for the further development of gene therapy products to treat the rare vision disorders, achromotopsia and choroideremia. Biogen will be entitled to select a single vector for each indication following which the research license may convert to a commercial license. In return, RegenxBio will receive a series of staged payments and will be eligible for royalties on net sales of products that incorporate the licensed RegenxBio intellectual property. Earlier this year RegenxBio had announced positive results for its investigational gene therapies RGX-314, for the treatment of wet age-related macular degeneration (wet AMD), and RGX-121, for the treatment of mucopolysaccharidosis Type II (MPS II), in preclinical animal models. As of December 31, 2015, RegenxBio state that their NAV Technology Platform is being applied in the development of 28 product candidates for a variety of diseases, including five internally developed candidates and 23 partnered candidates developed by RegenxBio’s licensees.
Commenting on the milestone Kenneth T. Mills, President and CEO of RegenxBio stated, “This license agreement provides new validation of the potential of our NAV Technology Platform in ocular indications and is an important step in advancing NAV-based gene therapies to people suffering from rare genetic vision disorders. We are pleased that Biogen, a respected biotechnology leader, has selected our NAV Technology Platform for the development of innovative gene therapies to improve treatment options in areas of significant unmet need.”