Avalanche Biotechnologies and the University of Washington Enter Into Exclusive License Agreement to Develop Gene Therapy Medicines to Treat Color Blindness
Avalanche also announced today, outside of the scope of the license agreement, that Drs. Jay and Maureen Neitz, faculty in the
"This agreement with the
Avalanche will build on gene therapy research conducted by the Neitz research team at the UW. They used gene therapy to confer color vision in two adult male squirrel monkeys that had been colorblind since birth.1 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," said
In addition, today 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 percent of males and 0.5 percent of females, or more than 10 million people in the U.S alone.2 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.3
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.
Avalanche is a biopharmaceutical company committed to improving or preserving the sight of people suffering from blinding eye diseases with a clear unmet medical need. Avalanche's proprietary Ocular BioFactory™ is a platform for discovering and developing novel medicines with the potential to offer life-changing therapeutic benefit. Avalanche's lead product candidate, AVA-101, is in mid-stage clinical development for patients with wet age-related macular degeneration. For more information, please visit www.avalanchebiotech.com.
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding Avalanche's plans, potential opportunities, expectations, projections, goals, objectives, milestones, strategies, product pipeline, clinical studies, product development and the potential benefits of its products under development, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including Avalanche's expectations regarding its ability to advance its AVA-101, AVA-322 and AVA-323 product candidates, initiate its Phase 2b and Phase 3 clinical trials with respect to AVA-101, and improve outcomes for patients suffering from blinding eye diseases. Statements regarding the projected size of the CVD patient population are also forward-looking statements. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process, including the regulatory approval process, the timing of our regulatory filings and other matters that could affect the availability or commercial potential of our product candidates. Avalanche undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties relating to the business of Avalanche, see our Annual Report on Form 10-K for the year ended
1 Mancuso, K. et al. Gene therapy for red–green colour blindness in adult primates. Nature. 2009; 461:784-787.
2 Sharpe LT, et al. Opsin genes, photopigments, color vision and color blindness. Gegenfurtner KR, Sharpe LT (eds.) Color Vision. Cambridge UP: Cambridge, 1999.
3 Cole, B. The handicap of abnormal colour vision. Clin Exp Optom. 2004; 87: 4-5:258–275.
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