Biologics manufacturer Lonza and ocular gene therapy specialist Avalanche Biotechnologies established a global manufacturing collaboration to aid in the latter's gene therapy programs. Avalanche’s lead wet age-related macular degeneration (wet-AMD) gene therapy candidate AVA-101 started in early clinical trials at the end of 2011.
The partnership will focus on the process development and scale-up of a baculovirus-based technology for manufacturing adeno-associated viral (AAV) vectors. As part of their collaboration Avalanche and Lonza will in addition make the resulting AAV production technology available to third parties and share relevant revenues. The process is based on a stable baculovirus technology licensed to Avalanche by Virovek, which will also collaborate on the manufacturing project.
Avalanche is focused on the development of technologies and products for the sustained delivery of therapeutic proteins to the eye for the treatment of disorders including wet AMD. Existing anti-VEGF approaches to treating wet AMD involve frequent injections of recombinant proteins into the eye. In contrast, Avalanche’s Ocular BioFactory gene therapy approach delivers the therapeutic gene directly into cells of the eye using a single injection of an AAV vector. The firm says that data from preclinical studies suggest that a therapeutic effect would be maintained for at least 18 months and potentially for several years after a single injection.
AVA-101 is being developed through an ocular therapeutics collaboration with the University of Western Australia’s Lions Eye Institute, signed in November 2012. Researchers at the Lions Eye Institute pioneered the Ocular Biofactory technology. AVA-101 is based on delivery of the gene for a therapeutic anti-VEGF protein licensed exclusively to Avalanche by Merck Sharp & Dohme.