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

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.