I-Corps: Translation Potential of Rapid In-situ Forming Gel for Local Gene Delivery

I-Corps：快速原位形成凝胶用于局部基因传递的转化潜力

• 批准号: 2410778
• 负责人: Gregg Duncan
• 金额: $ 5万
• 依托单位: University of Maryland, College Park
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2024
• 资助国家: 美国
• 起止时间: 2024-03-01 至 2025-02-28
• 项目状态: 未结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=2410778&HistoricalAwards=false
• 关键词: Corps; Translation; Potential; Rapid; situ

The broader impact of this I-Corps project is based on the development of a therapeutic delivery platform that is capable of releasing gene therapies locally to diseased tissues. This technology is well-suited for diseases that affect mucosal tissues such as the eyes, nose, and gastrointestinal and female reproductive tracts. Gene therapies hold promise in slowing the progression and potentially curing a wide range of diseases affecting these tissues. By minimizing exposure to healthy tissues, this technology can improve patient safety and reduce the strain on healthcare resources that are often required to manage treatment-related complications. This I-Corps project utilizes experiential learning coupled with a first-hand investigation of the industry ecosystem to assess the translation potential of the technology. The solution is based on the development of a biomaterial system which can be administered in a liquid form and, upon contact on the target tissue, rapidly transforms into a hydrogel capable of adhering to wet mucosal tissues. Owing to the potency of gene therapies and their utility in the treatment of a wide range of diseases, this hydrogel delivery system will be used as a therapeutic depot to deliver gene therapies to the diseased site. This innovation offers several advantages over conventional drug and gene therapies: local delivery avoids interactions with proteins and enzymes present in the blood stream that can render gene therapies ineffective when delivered intravenously and delivering the therapeutic payload directly to the affected tissue minimizes systemic exposure and associated side effects, reducing the risk of complications. Gene therapy itself is an attractive therapeutic modality as multiple disease-modifying targets can be addressed simultaneously for more effective disease management. Gene therapy can also significantly lower the frequency of drug administrations over a patient’s lifetime compared to conventional small molecule (e.g. steroids and antibiotics) and biologic (e.g. monoclonal antibodies) drugs.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I-Corps项目的更广泛影响是基于开发一种能够将基因疗法局部释放到患病组织的治疗递送平台。这项技术非常适合于影响粘膜组织的疾病，如眼睛，鼻子，胃肠道和女性生殖道。基因疗法有望减缓进展，并有可能治愈影响这些组织的各种疾病。通过最大限度地减少对健康组织的暴露，这项技术可以提高患者的安全性，并减少管理治疗相关并发症所需的医疗资源的压力。 这个I-Corps项目利用体验式学习加上对行业生态系统的第一手调查来评估该技术的翻译潜力。该解决方案基于生物材料系统的开发，该生物材料系统可以以液体形式施用，并且在接触靶组织时，迅速转化为能够粘附到湿粘膜组织的水凝胶。由于基因疗法的效力和它们在治疗广泛疾病中的效用，该水凝胶递送系统将用作将基因疗法递送至患病部位的治疗性储库。与传统药物和基因疗法相比，这种创新提供了几个优势：局部递送避免了与血流中存在的蛋白质和酶的相互作用，这些蛋白质和酶在静脉内递送时可能使基因疗法无效，并且将治疗有效载荷直接递送到受影响的组织，最大限度地减少了全身暴露和相关的副作用，降低了并发症的风险。基因治疗本身是一种有吸引力的治疗方式，因为可以同时解决多个疾病修饰靶点，以实现更有效的疾病管理。与传统的小分子药物（如类固醇和抗生素）和生物药物（如单克隆抗体）相比，基因治疗还可以显著降低患者一生中的用药频率。该奖项反映了NSF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。