“Yes, They Can!”

…live the full, healthy lives they deserve. Together, we can make it happen!

The FOXG1 Research Foundation is blazing the trail to cure FOXG1 syndrome while radically improving the rare disease drug development landscape.

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Watch Our Inspiring Story

On Track to Treatments

Since 2017, we have built the assets and robust data necessary to de-risk our disease and develop treatments for FOXG1 syndrome. 

We have...

  • Developed the first FOXG1 genetic therapy with curative preclinical results, moving towards human clinical trials by 2026

  • Established the FOXG1 Research Center oat the University at Buffalo

  • Pioneered a novel platform using AI for clinical research, now used by >100 rare disease groups 

  • Became a Chan Zuckerberg Initiative Partner and Mentor

  • Organized and connected the community of FOXG1 families around the world 

Advocating for Radical Change

Our co-founder and rare disease thought-leader, 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."

A Dedicated FOXG1 Lab

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."

Parents are driving cures.

This recent Bloomberg article, featuring our co-founder Nicole Johnson and her daughter Josie, perfectly captures why parents and philanthropists are the ones driving promising gene therapy treatments to children.

We are disrupting the rare disease drug development model to accelerate life-saving treatments where there is a severe unmet need.

“Yes, They Can!” live healthier lives

Our preclinical gene therapy data is showing groundbreaking results, including full reversal of the structural brain abnormality and more!

See this recent publication in Molecular Therapy Methods & Clinical Development.

We’re ready to move these promising results toward treatments for children.

Our “Yes, They Can!” campaign is our rallying cry to raise the $22 million dollars necessary to bring our gene therapy through phase 1 | 2 clinical trials. 

WE HAVE THE SCIENCE. WE HAVE THE ROADMAP. WE NEED YOUR HELP.

Together,  we can change the course of this disease.
Together, we can turn “They Can’t! to “Yes, They Can!”

Gene Therapy Program Roadmap

Steps and estimated costs along the road to clinical trials.
Funding is critical to the timeline.

Total to Reach IND: $16M | Total to reach completion of Phase 1/2 Clinical Trials: $22M

The Multiplier Effect of Your Support

  • FOXG1 is an Autism-related gene; our research is supported by Simon’s Autism Research Foundation

  • FOXG1 gene is a critical transcription-factor gene linked to many major brain disorders including Alzheimer’s, schizophrenia, types of brain tumors, and more

  • We are innovators, transforming the rare disease drug development landscape

  • Our lean and efficient model for drug development is cutting the timeline and cost to trials by a third

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're disrupting the model to accelerate treatments 

Many ways to help

Thank you for your interest in joining our mission to improve children’s lives.

For our "Ways to Give" document that includes wire and stock instructions, please email gifts@foxg1research.org

Mail a check to:
FOXG1 Research Foundation
1051 Port Washington Blvd. #1496
Port Washington, New York 11050-1496

Thank you for being a FOXG1 Champion for a Cure.

Your belief in us will lead to incredible improvements for countless children with rare disorders.