I-Corps: Translation Potential of a Gene Therapy to Treat Animal Diabetes

This I-Corps project focuses on the development of a new gene therapy designed to treat diabetes in pets through a single, long-lasting injection. Diabetes is a growing health issue among companion animals, with hundreds of thousands of dogs and cats affected in the United States alone. Current treatments often require daily insulin injections, which can be stressful, expensive, and difficult for pet owners to manage. The technology addresses that challenge by creating a simpler, more effective solution that helps regulate blood sugar levels with just one treatment. The therapy works by helping the animal’s body produce a natural protein that improves how it uses insulin and protects against damage caused by diabetes. The scale of the problem is significant, with the number of diabetic pets increasing and limited treatment options available. This therapy has the potential to reduce long-term veterinary costs and make diabetes care more accessible. Early results also show improvements in both body and brain health, supporting the potential for wider therapeutic benefits. The project also looks at how this treatment can be scaled up for use in veterinary clinics, filling a major gap in long-term care for pets with chronic diseases. 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. This solution is based on the development of a gene therapy platform designed to express a molecule with antioxidant and tissue-repair properties. The therapy targets metabolic dysfunction in diabetic animals by enhancing insulin sensitivity, reducing weight gain, and protecting against diabetic neuropathy. The approach involves a single intramuscular injection of a viral vector engineered to deliver the gene encoding this protein, resulting in sustained systemic expression. Preclinical studies in diet-induced diabetic mice demonstrate significant improvements in glucose regulation and cognitive function, with plasma protein levels maintained for several months post-injection. This method contrasts with existing treatments that rely on frequent insulin administration, offering a long-acting, low-maintenance alternative. The therapy’s design allows for scalability to larger animals, such as cats and dogs, using standard veterinary injection techniques. The scientific innovation lies in the use of a gene-based delivery system to achieve durable therapeutic effects from a single administration, reducing the need for ongoing intervention. Users benefit from improved disease control, reduced treatment burden, and lower risk of complications such as hypoglycemia. 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.