Accelerating FOXP1 research
Driving the science forward
All FOXP1 families want the best for their FOXP1 children. And while there is a growing body of knowledge about FOXP1 syndrome, much remains to be learned. At the IFF, we’re funding research grants, supporting data collection programs like RARE-X and Citizen, and connecting families with active studies to accelerate FOXP1 research. We also host scientists at family conferences, maintain relationships with key researchers around the globe, and support the next generation of young scientists to continue research into FOXP1 syndrome.
Laying the Groundwork for Future Treatments
01. Brain development and organoids
Two organoid projects to better understand the role of FOXP1 in brain development and to develop a potential screening tool for candidate therapeutics
02. Restoring FOXP1 at different stages
A project to develop a genetically engineered FOXP1 knockout mouse that allows restoration of FOXP1 at different stages of development
03. Gene therapy delivery
A genetic therapy study in FOXP1 “knockout” mice evaluating delivery of FOXP1 to brain cells using a viral vector
04. Evaluating a drug candidate
A drug candidate evaluation using genetically engineered cells and mice to evaluate a potential therapeutic for symptoms of FOXP1 syndrome
05. Measuring clinical change
A project involving parental interviews and qualitative assessments to develop a Clinical Global Impression Scale measurement tool that could be used as an endpoint in future clinical research
06. The FOUND natural history study
The FOUND study, a project involving detailed quantitative assessments and other measures to characterize the natural history (development over time) of FOXP1 syndrome
Some of these have now completed and the results are guiding next steps in FOXP1 research. One of the ongoing studies, the FOUND project, is a relatively long-term effort that will provide critical insights into the developmental trajectory for FOXP1 syndrome while also supporting future clinical trials with quality reference data when evaluating candidate therapeutics.
Led by Dr. Rujuta Wilson and her team at UCLA, the study exemplifies how some projects can simultaneously help meet our larger research objective of helping individuals with FOXP1 syndrome and their families with knowledge and insights now, while also supporting the development of novel candidate treatments later.
The future candidate treatment angle is also of interest to the FOXP1 Medical Research foundation, with which we are jointly funding the study. The FMRF’s focus is on “advancing rigorous, innovative medical research to discover treatments—and ultimately a cure—for FOXP1 syndrome” (see FMRF’s website), hence their interest in the project. Over time, other projects will certainly arise that may interest both organizations, and this is why we communicate frequently – to seize joint funding opportunities and to avoid duplicating efforts.
Research Centered on Individuals and Families
As our funded research projects have progressed, we’ve also sought input from the community on your research priorities. Your input and other considerations are helping shape how we think about future work. One area of interest includes projects that have a high probability of success in delivering insights for families over the short or medium terms while the longer game of product development plays out. I like to think of this as person-and-family-centered research.
Examples can include projects exploring the impacts of FOXP1 syndrome on the family, strategies to manage caregiver stress, best speech therapy and communication approaches, and other ways to help our children with approved medications or established behavioral therapies. Some of this will come from clinic-based projects, some from surveys, some may even be done in collaboration with other rare-disease communities or be based on work with those communities.
Beyond research grants
How We Support FOXP1 Research
Data collection
Facilitate access to FOXP1 data collection platforms (RARE-X, Citizen)
Study participation
Connect families to researchers looking for participants
Family and therapy research
Coordinate and/or fund family- and therapy-focused research (e.g., caregiver burden, best speech therapy approaches for FOXP1)
Bringing families and researchers together
Fund and host family-focused events where researchers often share research and speak to family concerns, such as the biennial International Family Conference and other family gathering
Working together and keeping you informed
Like the jointly-sponsored FOUND study, the IFF and FMRF both actively engage in the first two efforts. These two projects and the FOUND study exemplify how the two foundations seek to share knowledge, information, and priorities to make FOXP1 research as a whole as efficient and impactful as possible.
The above lists of priority research areas and related activities reflect our priorities as of 2026, and these may shift over time. We'll communicate clearly about any major changes so that you, the FOXP1 community, understand how we are focusing our time and your generous contributions.