FOXG1 Research Foundation: Reinventing Rare Disease Drug Development

In 2026, the FOXG1 Research Foundation stands at a historic moment — and this is how they got here.

Co-founders Nicole Johnson and Nasha Fitter started the FOXG1 Research Foundation when they were told there was no path forward for their daughters. The rare disease drug development landscape was not in their favor. The market had no interest. The odds were against them. They forged ahead anyway.

What followed was nine years of building — a global organization, a world-class scientific program, a new model for patient data, and a community of families across 34 countries united by one mission.

Today, FRF has FDA clearance to begin first-in-human clinical trials for FRF-001, a FOXG1 gene replacement therapy.

They are also doing something historic: independently sponsoring their own gene therapy program through multi-site clinical trials. No pharma backing. FRF-001 represents a new model for rare disease drug development — parent-led, nonprofit, and independent — made possible by innovation, efficiency, determination, love, and philanthropy.

Watch Nicole and Nasha, alongside Tom Horton, former CEO of American Airlines and a FOXG1 grandfather, share the FOXG1 Research Foundation's journey.