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

这个小企业创新研究（SBIR）二期项目的更广泛的影响/商业潜力是进一步开发和商业化一种眼部基因疗法，以逆转干性年龄相关性黄斑变性（AMD）的影响，这种疾病影响着超过1100万美国人，是美国老年人失明的主要原因。拟议的治疗方法有可能保持AMD患者的独立性、经济稳定性和生活质量，并通过一次治疗降低美国的医疗保健成本。这项小企业创新研究（SBIR）二期项目技术计划将在大型动物模型中优化一种研究性疗法的配方、剂量和给药途径。该项目将首先开发各种载体的基因治疗配方。通过初步的药代动力学分析，将评估该制剂注射入眼后在视网膜组织中的效率。将进行毒理学研究，以评估新配方和注射途径在3个月内不同剂量的安全性和有效性。随后，该项目将评估制造基因运载工具的风险，以确保能够制造出高浓度的基因运载工具，供未来临床使用。该奖项反映了美国国家科学基金会的法定使命，并通过使用基金会的知识价值和更广泛的影响审查标准进行评估，被认为值得支持。

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.