Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is the leading cause of blindness in the US, with more than 1.6 million people affected. Wet AMD is a more severe progression of AMD, affecting 10 percent of patients with AMD. One of the treatments for wet AMD is ranibizumab (trade name Lucentis©); however, these treatments require frequent injections into the affected eye.
We believe that gene therapy offers a potential long-term solution to treat wet AMD with one injection, and there is a strong rationale for combination therapy to become the standard of care in wet AMD. We have defined our preferred target profile and are proceeding with a comprehensive review of possible targets.