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SBIR Phase I: Preclinical Validation of an Ocular Antioxidant Enhancing Gene Delivery Vehicle to Target Dry Macular Degeneration

SBIR Phase I: Preclinical Validation of an Ocular Antioxidant Enhancing Gene Delivery Vehicle to Target Dry Macular Degeneration
SBIR 第一阶段:针对干性黄斑变性的眼部抗氧化剂增强基因递送载体的临床前验证
批准号:
2111704
负责人:
Ramaswamy Ramkumar
金额:
$25.6万
依托单位:
依托单位国家:
美国
项目类别:
Standard Grant
财政年份:
2021
资助国家:
美国
项目状态:
已结题
起止时间:
2021-05-01 至 2021-10-31

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中文摘要
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英文摘要
The broader impact /commercial potential of this Small Business Innovation Research (SBIR) Phase I project is to develop and commercialize a gene therapy to reverse the effects of dry age-related macular degeneration in over 200 million people worldwide over the age of 50. This therapy may prevent loss of vision and independence by helping people retain their ability to drive, handle finances, see faces of loved ones, and live independently. Not only would this improve the quality of life in patients with macular degeneration, but it would also decrease the economic burden of visual disability on the US healthcare system. The proposed therapy is a durable treatment that can potentially last a lifetime and be delivered into the eye with an in-office procedure. No treatments currently exist for this disease. This technology will meet an unmet need for hundreds of millions of patients with no other treatment options. This Small Business Innovation Research (SBIR) Phase I project seeks to optimize the design and dose required to safely reach the retina with desired expression levels and elicit pan-retinal protection from light-induced retinal degeneration. The proposed AAV-mediated modifier therapy is an epigenetic regulator that modulates gene expression involved in neural stem cell renewal rather than replacing various genes associated with macular degeneration. The proposed SBIR Phase 1 technical plan addresses the various tests for optimization of the delivery capsid, which will deliver an episomal gene that acts as an epigenetic regulator and boosts innate antioxidant genes, reduces cellular senescence, repairs DNA damage, and increases the expression of anti-apoptotic genes in retinal cells. In this SBIR Phase 1 study, we will conduct in vitro (retinal pigment epithelium cells) and in vivo experiments to determine the dosage, safety and efficacy of the treatment for subsequent studies.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.
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SBIR Phase II: Preclinical Validation of an Ocular Antioxidant Enhancing Gene Delivery Vehicle for Dry Macular Degeneration
  • 批准号:
    2208096
  • 项目类别:
    Cooperative Agreement
  • 资助金额:
    $100.0万
  • 财政年份:
    2022
  • 负责人:
    Ramaswamy Ramkumar
  • 依托单位:
国内基金
海外基金
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  • 批准号:
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  • 项目类别:
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  • 资助金额:
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  • 批准年份:
    2024
  • 负责人:
    YUICHIRO NAKAI
  • 依托单位:
ATLAS实验探测器Phase 2升级
  • 批准号:
    11961141014
  • 项目类别:
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  • 资助金额:
    3350万元
  • 批准年份:
    2019
  • 负责人:
    刘衍文
  • 依托单位:
地幔含水相Phase E的温度压力稳定区域与晶体结构研究
  • 批准号:
    41802035
  • 项目类别:
    青年科学基金项目
  • 资助金额:
    12.0万元
  • 批准年份:
    2018
  • 负责人:
    张里
  • 依托单位:
基于数字增强干涉的Phase-OTDR高灵敏度定量测量技术研究