Pitt Hopkins Research Foundation

About Pitt Hopkins Research Foundation

The Pitt Hopkins Research Foundation concentrates its giving on research for Pitt Hopkins syndrome and related disorders, with awards directed to work that can move toward treatment and an eventual cure. In 2024, its largest recent grant went to Nationwide Children's Hospital in Columbus for Pitt Hopkins syndrome research projects, signaling a strong emphasis on academic medical research. The foundation also funded the University of California - San Diego for the same purpose, and its current program design centers on therapeutic development, clinical research, and translational science. That pattern fits the foundation’s stated mission: support research dedicated to finding a treatment and eventual cure for Pitt Hopkins Syndrome while also supporting the Pitt Hopkins community with resource recommendations, parental support, and medical information. Its active grant programs include research grants, young investigator awards, and named awards for early-career researchers. The funding model is project-based rather than individual-focused, and the organization uses targeted grants to support academic labs and research organizations working on PTHS, gene therapy, preclinical research, and clinical studies. Leadership is listed under Diane Sellew.

What Pitt Hopkins Research Foundation Funds

A major theme in the foundation’s giving is direct support for Pitt Hopkins syndrome research projects. It awarded $62,183 to the University of California - Davis in 2024 and $16,606 to the University of Colorado Denver, both described as support for research projects on Pitt Hopkins syndrome. Gene therapy is another clear priority. The Ann D. Bornstein Gene Therapy Grant supports pilot gene therapy research for Pitt Hopkins syndrome, including collaborative lab work on AAV-based TCF4 gene transfer. The foundation’s 2025 research funding priorities also point to bringing gene therapy programs to clinical trial and supporting small molecule discovery targeting the TCF4 pathway. Early-career researchers are part of the portfolio as well. The Kindal Kivisto Award for Promising Young Researchers supports promising early-career researchers working on Pitt Hopkins-related science, and the Tilly Hadlow Young Investigator Award serves a similar function for young investigators pursuing PTHS research.

How Pitt Hopkins Research Foundation Gives