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.

I-Corps 项目的更广泛影响是基于治疗传递平台的开发，该平台能够向患病组织局部释放基因疗法，该技术非常适合影响眼、鼻、鼻等粘膜组织的疾病。基因疗法有望减缓疾病的进展，并有可能治愈影响这些组织的多种疾病。通过最大限度地减少与健康组织的接触，该技术可以提高患者的安全性并减轻对医疗资源的压力。需要管理该 I-Corps 项目利用体验式学习以及对行业生态系统的第一手调查来评估该技术的转化潜力，该解决方案基于可在环境中管理的生物材料系统的开发。由于基因疗法的效力及其在治疗多种疾病中的效用，这种水凝胶递送系统将呈液体形式，接触目标组织后迅速转化为能够粘附在湿粘膜组织上的水凝胶。用作与传统药物和基因疗法相比，这项创新具有多种优势：局部递送避免了与血流中存在的蛋白质和酶的相互作用，这些相互作用可能导致基因疗法在均匀递送和递送治疗剂时无效。有效载荷直接作用于受影响的组织，最大限度地减少了全身暴露和相关副作用，降低了并发症的风险。基因治疗本身就是一种有吸引力的治疗方式，因为可以同时解决多个疾病缓解目标，从而实现更有效的疾病管理。超过一段时间的用药频率与传统小分子（例如类固醇和抗生素）和生物（例如单克隆抗体）药物相比，该奖项反映了 NSF 的法定使命，并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，被认为值得支持。