San Diego, CA (PressExposure) November 27, 2013 -- Abraham Scaria, Senior Scientific Director at Sanofi-Genzyme will present on "Gene Therapy for the Treatment of Neovascular AMD" at GTC's 6th Annual Ocular Diseases & Drug Discovery Conference held on March 20-21, 2014 in San Diego, CA.
VEGF plays a critical role in neovascular age-related macular degeneration and proliferative diabetic retinopathy. VEGF antagonists are useful for treating such disorders; however current treatments require monthly intravitreal injections. We have designed a soluble anti-VEGF molecule (sFLT01) and delivered it by intravitreal injection of an adeno-associated viral (AAV) vector since AAV vectors are capable of long-term gene expression. AAV2-sFLT01 inhibited retinal neovascularization in the murine oxygen-induced retinopathy model and in the laser-induced choroidal neovascularization (laser-CNV) model in mice. In the eyes of rodents and cynomolgus monkeys, AAV2-sFLT01 gives expression levels persistent for at least one year. We also performed laser-CNV experiments 5 months after vector administration in non-human primates and showed that sFLT01 is effective at inhibiting neovascularization in this model. Results of our 12-month safety study of AAV2-sFLT01 administered intravitreally in cynomolgus monkeys will be discussed. In summary, we have demonstrated long-term efficacy with minimal side effects following intravitreal delivery of AAV-sFLT01 in rodents and non-human primate models. These results suggest an alternate method for the long-term treatment for diseases of ocular neovascularization, without the need for repeated intraocular injections. A Phase I clinical trial is almost completed at four clinical sites in the USA.
Dr. Scaria obtained his Ph.D. in Molecular Biology from Indiana University School of Medicine in Indianapolis. He did postdoctoral training at St. Louis University School of Medicine working on RNA splicing mechanisms in adenoviruses and virus-host interactions. Dr. Scaria then worked as a Senior Fellow at University of Washington School of Medicine in Seattle on gene delivery to the liver using viral vectors before joining Genzyme Corporation in 1994 to work on gene delivery to the lungs for Cystic Fibrosis. Dr. Scaria has 20 years of experience in gene therapy using adenoviral and AAV vectors from discovery research to preclinical development, RAC application and IND filing. Currently Dr. Scaria is a Senior Scientific Director at Genzyme where he heads up the "Gene Therapy - Ophthalmology" group. His other research interests include host immune responses to viral vectors and immunomodulation for the treatment of autoimmune diseases.
The Ocular Diseases & Drug Discovery Conference continues to promote the discovery of ocular disease by offering presentations on novel and innovative therapeutics in ocular drug development and discovery. The conference brings together leading scientists, researchers and experts to discuss and collaborate on the latest research and development, safety assessment, regulatory issues and drugs in development for combating and curing age-related macular degeneration (AMD), diabetic retinopathy, glaucoma, DME, uveitis and other ocular diseases.
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