Avalanche adds color to pipeline with UW deal
This article was originally published in Scrip
Several months after its successful IPO, Avalanche Biotechnologies has inked a licensing agreement with the University of Washington in Seattle to use the biotech's technology platform and gene therapy research conducted at the university to find treatments for color vision disorder, or red-green color blindness. The Avalanche Ocular BioFactory is a proprietary adeno-associated virus (AAV)-based gene therapy discovery platform that was created for the development of ophthalmic disease therapies. The company's drug delivery system is designed to use the body's own cells to produce therapeutic proteins after only one injection with a gene therapy. AVA-322 and AVA-323 target the two genes that cause the condition, L-opsin and M-opsin, respectively, and use Avalanche's non-surgical intravitreal injection method to deliver the genes to the back of the eye. The company expects to conduct IND-enabling studies this year. Drs Jay and Maureen Neitz of UW will join the scientific advisory board of Avalanche to consult on the development of the drugs. Color blindness is one of the most common inherited genetic diseases, affecting nearly 10m people – virtually all males. The work with UW expands the biotech's pipeline. Currently, Avalanche only has one drug in the clinic so far, AVA-101 for wet age-related macular degeneration. The compound is partnered with Regeneron and could bring in $640m in milestone payments plus royalties should it reach the market.