SBIR Phase II: Preclinical Validation of an Ocular Antioxidant Enhancing Gene Delivery Vehicle for Dry Macular Degeneration

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The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase II project is to further develop and commercialize an ocular gene therapy to reverse the effects of dry age-related macular degeneration (AMD), a disease affecting over 11 million Americans today and representing the leading cause of blindness in the elderly in the United States. The proposed treatment has the potential to preserve independence, economic stability, and quality of life of those affected by AMD and reduce US healthcare costs with just a single treatment.This Small Business Innovation Research (SBIR) Phase II project technical plan will optimize the formulation, dose, and route of administration of an investigational therapy in a large animal model. The project will first develop gene therapy formulations with various vehicles. The formulations’ efficiency in retinal tissues with an injection into the eye will be assessed with preliminary pharmacokinetic analyses. Toxicology studies will be performed to assess safety and efficacy of the novel formulation and the injection route at various doses over 3 months. Subsequently, the project will assess risk in manufacturing the gene delivery vehicles to ensure that high concentrations can be manufactured for future clinical use.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.