Research & Development
FOXG1 Gene Therapy Patient Trials on Track for 2026!
Our current lead therapeutic program is the FOXG1 AAV9 gene replacement therapy and we are on track to begin patient clinical trials in 2026.
Advancing Multiple Paths to Treatment
A cure for FOXG1 syndrome could involve a single genetic therapy or multiple therapeutics, depending on each child’s specific mutation. Our mission is to continuously work towards the most successful curative therapeutics for every child in the world with FOXG1 syndrome.
Scientific and R&D Advisory Board
Meet the experts guiding our path to treatment. This multidisciplinary group includes leading biotech executives, top researchers, and key opinion leader clinicians in FOXG1 syndrome. Together, they help shape strategy, advise on critical programs, and ensure we advance the most promising science with world-class partners and institutions.
Key FOXG1 Publications
Explore pivotal peer-reviewed publications that have shaped our understanding of FOXG1 syndrome. This extensive collection spans early discoveries of FOXG1 mutations, clinical insights into patient characteristics, and groundbreaking advancements in gene therapy research.
For Researchers: Assets & Grants
We have developed and continue to develop a suite of assets such as mouse models and patient-derived iPSC lines that we make openly available for researchers worldwide to access.
We have partnered with Autism BrainNet, a Simons Foundation initiative that promotes research on donated brain tissue to improve the understanding of autism-related neurodevelopmental conditions like FOXG1 syndrome.
FOXG1 Brain Donation
FOXG1 Stem Cell Biobank
We partnered with the Coriell Institute to create a comprehensive FOXG1 Biobank, housing patient-derived iPSCs and blood samples from individuals with FOXG1 syndrome and their families. These resources are available to scientists worldwide to support research and accelerate therapeutic development.
