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项目的更广泛的影响是基于能够将基因疗法释放到解剖组织的治疗递送平台的发展。该技术非常适合影响粘膜组织，例如眼睛，鼻子和胃肠道和女性生殖道。基因疗法有望减慢进展并可能治愈影响这些组织的广泛疾病。通过最大程度地减少对健康组织的接触，该技术可以改善患者的安全性，并减少经常需要管理与治疗相关并发症的医疗资源的压力。这个I-Corps项目利用专家学习以及行业生态系统的第一手投资来评估技术的翻译潜力。该解决方案基于生物材料系统的发展，该系统可以以液态形式给药，并在目标组织接触后，迅速转化为能够粘附在湿粘膜组织上的水凝胶。由于基因疗法的效力及其在治疗多种疾病方面的效用，该水凝胶递送系统将被用作治疗沉积物，以将基因疗法递送到解剖部位。这项创新比传统的药物和基因疗法具有多个优势：局部分娩避免了与血液中存在的蛋白质和酶相互作用，这些蛋白质和酶在静脉内交付时可能使基因疗法无效，并将治疗有效负荷直接传递给影响的受影响的组织，以最大程度地减少系统性暴露和相关的副作用，从而降低综合性的风险。基因疗法本身是一种有吸引力的治疗方式，因为可以简单地解决多种疾病改良靶标的疾病管理。与传统的小分子（例如类固醇和抗生素）和生物学（例如单克隆抗体）药物相比，基因治疗还可以显着降低患者一生中药物施用的频率。这奖反映了NSF的宣传任务，并通过评估通过基金会的知识优点和广泛的actitia crietia crietia croctiria来评估，这是NSF的立法任务。