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

SBIR II 期：眼部抗氧化增强基因递送载体治疗干性黄斑变性的临床前验证

• 批准号: 2208096
• 负责人: Ramaswamy Ramkumar
• 金额: $ 100万
• 依托单位: OCULOGENEX
• 依托单位国家: 美国
• 项目类别: Cooperative Agreement
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-05-01 至 2024-10-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=2208096&HistoricalAwards=false
• 关键词: SBIR; Phase; II; Preclinical; Validation

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.

这个小型企业创新研究（SBIR）第二阶段项目的更广泛的影响/商业潜力是进一步开发和商业化眼部基因疗法，以逆转干性年龄相关性黄斑变性（AMD）的影响，这种疾病目前影响着1100多万美国人，是美国老年人失明的主要原因。这项小型企业创新研究（SBIR）二期项目技术计划将在大型动物模型中优化研究性治疗的配方、剂量和给药途径。该项目将首先开发具有各种载体的基因治疗制剂。将通过初步药代动力学分析评估注射至眼内的制剂在视网膜组织中的有效性。将进行毒理学研究，以评估3个月内不同剂量的新型制剂和注射途径的安全性和有效性。随后，该项目将评估生产基因传递载体的风险，以确保高浓度可以生产用于未来的临床使用。该奖项反映了NSF的法定使命，并已被认为是值得通过使用基金会的智力价值和更广泛的影响审查标准进行评估的支持。