Priscilla Chan gets emotional talking about the FOXG1 gene therapy journey
On April 13, 2026, Stanford Medicine's "The Future of Medicine" podcast featured Dr. Priscilla Chan — pediatrician, co-founder of the Chan Zuckerberg Initiative, and co-founder of Biohub — in a wide-ranging conversation about AI, rare disease, and the future of medicine.
The interview covers extraordinary ground: the work CZI is doing to build a "virtual cell" — a computational model of human biology — and how patient-led research communities are driving breakthroughs that traditional science could not.
And then she talks about us, the FOXG1 Research Foudnation and Nasha’s promise to Amara, saying, "I Can't Even Say This Without Getting a Little Teary."
Speaking about the impact of CZI's Rare As One grants to patient-led research communities, Dr. Chan singled out the FOXG1 Research Foundation as the example that moved her most. In her own words:
"One that I'm particularly excited about this week is around the FOXG1 mutation, which causes a rare neurodevelopmental disorder in children. The group just got FDA approval for their clinical study for gene therapy. And I can't even say this without getting a little teary. The mom who started the group promised her daughter they'd have a clinical study by the time the child was 10 — and they got it approved on Amara's 10th birthday. That is incredible. It's also incredibly fast. That work has been incredible and inspiring — to see how you can do science differently."
— Priscilla Chan
The Impact of CZI’s FOXG1 Research Foundation Rare As One Grant
In 2021, the Chan Zuckerberg Initiative awarded the FOXG1 Research Foundation a $500,000 Rare As One grant — specifically to build a new kind of patient data platform designed to accelerate the path to real treatments for rare diseases. That is exactly what the program set out to accomplish: fund patient-led communities with the tools and infrastructure to move science faster than traditional models allow.
The grant helped FRF build one of the most innovative natural history study designs in rare disease — a real-world, longitudinal data platform now used for more than 350 rare diseases. It became the foundation for FRF's path to clinical trials for FRF-001, our FOXG1 gene therapy — contributing to an FDA alignment that could allow our Phase I/II trial to serve as a registrational study.
That is the work Priscilla Chan is describing. That is what she gets emotional about.
Nasha Fitter promised Amara — her daughter, our co-founder's daughter — that they would have a clinical study by the time she turned 10. The FDA cleared FRF-001 for first-in-human clinical trials in January 2026. Amara turned 10 that same month.
When the co-founder of one of the world's largest science philanthropies — on Stanford Medicine's platform — gets emotional talking about your work, it means something. It means the model is working. It means the science is real. And it means the children who need this therapy are one step closer to getting it.
Watch the Full Interview
The FOXG1 Research Foundation is grateful to Dr. Priscilla Chan and the Chan Zuckerberg Initiative for their belief in our work — and for helping make this moment possible.
Learn more about the FOXG1 gene therapy “Yes, They Can!” campaign here.