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.

这项小型企业创新研究（SBIR）II期项目的更广泛的影响/商业潜力是进一步开发和商业化眼基因疗法，以扭转与当今有1100万美国人的疾病，并扭转了与干燥年龄相关的黄斑变性（AMD）的影响，该疾病如今影响了超过1100万美国人，并代表了美国最大的盲人症状。拟议的治疗方法有可能维护受AMD影响的人的独立性，经济稳定和生活质量，并仅通过一种治疗来降低美国的医疗保健成本。这项小型企业创新研究（SBIR）II期项目技术计划将优化大型动物模型中投资治疗的公式，剂量和管理途径。该项目将首先使用各种车辆开发基因治疗配方。将通过初步的药代动力学分析来评估对眼睛注射的残留组织中配方的效率。将进行毒理学研究，以评估新型配方的安全性和效率，并在3个月内以各种剂量进行注射途径。随后，该项目将评估制造基因输送工具的风险，以确保可以为将来的临床使用制造高浓度。该奖项反映了NSF的法定任务，并通过使用基金会的知识分子和更广泛的影响审查标准来评估，被认为是珍贵的支持。