Diabetic Retinopathy (DR) is a progressive disease that affects blood vessels in the retina. Approximately 30 million individuals in the U.S. are affected by diabetes1. Approximately eight million individuals in the U.S. have DR, which is the leading cause of vision impairment and blindness among working-age adults2. DR is a result of microvascular damage to the blood vessels in the retina caused by blood sugar. In early stages, patients may develop non-proliferative diabetic retinopathy (NPDR), which often has no symptoms and can progress to proliferative diabetic retinopathy (PDR). In advanced stages, PDR can result in new blood vessels growing on the surface of the retina, potentially causing vision loss.
Our gene therapy candidate for diabetic retinopathy (DR) is ADVM-022, administered as a single intravitreal (IVT) administration. We plan to file an IND for ADVM-022 for DR in the first half of 2020. This therapy utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is designed to provide sustained therapeutic levels of aflibercept, minimize the burden of frequent anti-VEGF injections, and improve real-world vision outcomes for patients.