Avalanche Biotechnologies and the University of Washington Enter Into Exclusive License Agreement to Develop Gene Therapy Medicines to Treat Color Blindness

Source: Avalanche Biotechnologies

Avalanche Biotechnologies announced that it has entered into an exclusive license agreement with the University of Washington in Seattle to develop products based on Avalanche’s proprietary Ocular BioFactory Platform for the treatment of color vision deficiency (CVD), commonly known as red-green color blindness, according to a company news release.

Avalanche also announced, outside of the scope of the license agreement, that Drs. Jay and Maureen Neitz, faculty in the University of Washington’s Department of Ophthalmology and CVD experts, have joined its Scientific Advisory Board. They will be technical advisors to the company on the science of vision.

“This agreement with the University of Washington and world-renowned vision scientists Drs. Jay and Maureen Neitz will help us further advance our goal of developing therapeutic products for the millions of people who suffer from CVD,” Thomas W. Chalberg, Jr., PhD, founder and chief executive officer of Avalanche Biotechnologies, said in the news release. “Our proprietary technology enables us to target the retina through intravitreal adeno-associated virus delivery, presenting, for the first time, the opportunity to pursue previously untreatable ophthalmic conditions such as CVD.”

Avalanche will build on gene therapy research conducted by the Neitz research team at the University of Washington. They used gene therapy to confer color vision in two adult male squirrel monkeys that had been colorblind since birth. This groundbreaking work demonstrating proof-of-concept for treating CVD was published in the journal Nature.

“Avalanche is performing pioneering research that has the potential to cure CVD,” Jay Neitz, PhD, professor of ophthalmology at the University of Washington. “CVD is just one of the many cone diseases that could be treated with Avalanche’s technology.”

In addition, Avalanche launched www.colorvisionawareness.com, a disease awareness website designed to help people suffering from color blindness better understand their condition. The website provides the opportunity for people to sign up to be among the first to receive information about potential research study opportunities or therapies for color blindness that become available.

About CVD and Avalanche’s Targeted Development Program

Color vision deficiency (CVD), also known as red-green color blindness, is among the most common genetic diseases. CVD affects approximately 8% of males and 0.5% of females, or more than 10 million people in the U.S alone. CVD is a visual impairment that impacts many aspects of everyday life, resulting in limitations in professional choices, compromised health and safety, and the inability to perform many activities of daily living.

Photopigments in the retina are crucial for perceiving color. People with normal color perception have three different types of photopigments. These photopigments are tuned to perceive either long wavelengths (red), middle wavelengths (green) or short wavelengths (blue), referred to as L-, M- and S-opsin. The most common forms of CVD are due to genetic defects that lead to missing either the L-opsin (protan defects) or the M-opsin (deutan defects).  Affected individuals have trouble distinguishing between red and green and between colors that contain red or green hues.

Avalanche has two drug candidates targeting these areas. AVA-322 carries the gene for L-opsin and is being developed for the treatment of protan defects. AVA-323 carries the gene for M-opsin and is being developed for the treatment of deutan defects.


Related Content