Project Grant R01DK142964

The National Institute of Diabetes and Digestive and Kidney Diseases awarded The Children's Hospital of Philadelphia $695,683 on August 1, 2026, under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) to develop improved stem cell-derived beta cells for cellular therapeutics of diabetes.

The project, titled "Making a Better Beta Cell for Cellular Therapeutics of Diabetes," focuses on engineering human pluripotent stem cell-derived beta cells capable of surviving and functioning long-term in diabetic patients. The work leverages naturally occurring hyperactive mutations in the HNF1A gene that protect individuals from type 2 diabetes. HNF1A is a transcription factor critical to beta cell development and function; loss-of-function mutations in this gene are the most common cause of monogenic diabetes and have been linked to both type 1 and type 2 diabetes through genome-wide association studies. The proposed research addresses three core challenges for beta cell therapeutics: survival in the hypoxic and inflammatory environment following transplantation, long-term robustness and function under diabetic conditions, and prevention of immune attack by the host immune system.

Work is performed at The Children's Hospital of Philadelphia Research Institute in Philadelphia, Pennsylvania. The project period runs from August 1, 2026, through May 31, 2031. The award is structured as a Project Grant, the standard assistance type for investigator-initiated biomedical research under NIDDK.