2025 marked a pivotal year for the FOXG1 Research Foundation.

We successfully completed IND-enabling toxicology studies and submitted an Investigational New Drug (IND) application to the FDA, advancing the first-ever FOXG1 gene therapy toward patient clinical trials in 2026.

As a global, parent-led organization, we continue to demonstrate that families can independently drive rare disease drug development—executing clinical trials with rigor, urgency, and responsibility.

Alongside this progress, we expanded patient care, research, advocacy, and global awareness, while honoring the lives lost within our community and reaffirming why this work cannot wait.

Together with our families, donors, scientific partners, and clinicians, we are building a future where treatment is possible.

2023 marked six years of the FOXG1 Research Foundation. We are a parent-driven, global foundation with the mission to help improve the lives of every person affected by FOXG1 syndrome worldwide. We are known in the industry as innovators as we  focus on three equally critical areas: FOXG1 science, FOXG1 patient data, and FOXG1 patients and community. From advancing our gene therapy program to building our own bioinformatics platform, to helping guide parents through the rare disease medical caregiver journey, this year has been a year of upward progress in all three areas. Here are the FOXG1 Research Foundation highlights from 2023 and goals for 2024.

2022 Research Highlights (see details below)

• Breakthrough gene therapy results showing rescue of FOXG1 brain structure, behavior, memory and cognition symptoms in animal models

• Positive results on initial compound drug screens to identify molecules to increase FOXG1 levels; now moving forward to larger screens

• Never-before understanding of FOXG1 biology uncovered from six FOXG1 patient human cell lines and mouse models 

• Successful testing of guide RNAs to increase FOXG1 expression with a CRISPRa Cas-9 system

• Discovered ASO sequences (antisense gene therapy) to modulate FOXG1 expression; testing initiated with Creyon Bio  

• FOXG1 data package presented to several biotech companies with high interest