Help Us Say….
“YES, They Can!”
…live healthier, easier lives”
Let’s Bring the FOXG1 Gene Therapy to Children Worldwide, Together!
Watch The Parent-Driven Drug Development Story
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Watch The Parent-Driven Drug Development Story *
Make history with us.
Be part of the first parent-led rare disease gene therapy program to advance through full-scale, multi-site International clinical trials independently.
“Gene therapy is the most direct and promising approach to treat the root cause of FOXG1 syndrome.”
-Gai Ayalon, Ph.D.
FOXG1 Research Foundation
Chief Drug Development Officer
A New Path for Rare Disease Drug Development
This Bloomberg article perfectly captures why parents and philanthropists are driving promising gene therapy treatments to children independently.
When pharma says “no,” but science says "yes,” the FOXG1 Research Foundation is showing how.
While rare diseases represent the greatest unmet need in medicine, parents are proving a new way forward.
The FOXG1 Research Foundation is pioneering a nonprofit-led, highly efficient gene therapy model. Driving treatments independently— this is only possible with donor support.
“The FOXG1 Research Foundation is radically transforming the rare disease drug development landscape”
-Tania Simoncelli
The FOXG1 Gene Therapy is showing groundbreaking pre-clinical results!
We’re ready to begin patient clinical trials in 2026!
WE HAVE THE SCIENCE.
WE HAVE THE TEAM.
WE NEED YOUR HELP.
Together, we can change the course of this disease!
Many ways to help
Thank you for joining our mission to improve children’s lives.
There are many ways to support the FOXG1 Research Foundation.
Mail a check to:
FOXG1 Research Foundation
1051 Port Washington Blvd. #1496
Port Washington, New York 11050-1496
To request our "Ways to Give" document that includes instructions for: wire, stock, crypto, and DAF donations, please email gifts@foxg1research.org
The Impact of Your Support is Immediate and Far-Reaching
Our lean and efficient model for drug development is cutting the timeline and cost through clinical trials by a third
Children will begin receiving this treatment in clinical trial starting in 2026
We are paving the way to accelerate rare disease treatments at large
Bet on us to succeed
Parent-led rare disease organizations have an unparalleled track record and competitive advantage in drug development
We are not motivated by money or publications
Our ROI is our children's lives
We have the deepest understanding of the patient experience
We’re efficient, innovative, passionate, and relentless
A Treatment is Within Reach
With your help, we can bring it to children all over the world who urgently need it.
Help spread the mission.
Watch Leadership in Action
Our co-founder Nasha Fitter, spoke at The White House on how parent-led organizations, like ours, are driving drug development for our children's diseases. Watch this powerful speech and see how leaders from the private and public sector cited the determination and efficiency of the FOXG1 Research Foundation to "transform the rare disease research landscape."
Tom Horton, former CEO of American Airlines and grandfather to Gianna, who has FOXG1 syndrome, visits the FOXG1 Research Center at the University at Buffalo with his wife Janet Horton. Tom says, "Looking at the lab and the path to gene therapies, gives me a lot of hope."
He Did It!
Tom Horton Walked 500 Miles for the FOXG1 Syndrome Gene Therapy!
A pilgrimage of prayer and action to bring hope to families worldwide
Thank you to our wonderful sponsors and every donor
for being a FOXG1 Champion for a Cure.
Your belief in us will lead to incredible improvements for countless children with rare disorders.
