Funded Research
Where Your Gift Goes
Community-funded. Peer-reviewed. Field-advancing.
The Foundation awards research grants through an open, competitive process. Every proposal is reviewed by our Scientific Advisory Board against clear scientific criteria and community priorities.
The projects listed here are the direct result of donations from families, friends, and partners around the world.
Funded Projects to Date
Investigating FOXP1 dosage restoration in a new mouse model
Testing whether restoring normal FOXP1 protein levels can correct key symptoms of FOXP1 syndrome. By selectively reactivating FOXP1 at different developmental stages, the team will assess impact on activity levels, vocalisation and lean mass — and establish a valuable new mouse model for the wider scientific community.
Modeling FOXP1 syndrome using human brain organoids
Using patient-derived stem cells to generate three-dimensional brain organoids and assembloids that replicate the neural network disruptions caused by FOXP1 variants — a human-based platform with the potential to accelerate preclinical therapeutic testing.
Advancing clinical trial readiness through objective neurodevelopmental phenotyping
Scalable, objective assessments of motor function, language, cognition and autism symptoms in children aged 1–20. In-person and remote testing allows broad participation, refining the assessment tools future clinical trials will depend on.
Three-dimensional organoid model of FOXP1 syndrome
Developing a medium-to-high throughput assay to screen compound libraries for candidates that normalise the FOXP1 organoid phenotype — the first step in a drug discovery pipeline built on patient-derived cell lines.
Functional restoration of FOXP1 haploinsufficiency using AAV-mediated gene rescue
Delivering a working copy of FOXP1 to brain cells via AAV viral vector in a mouse model that mirrors the genetics of FOXP1 syndrome, then testing whether gene replacement corrects behavioural deficits — and how late in development it still works.
Testing a potential treatment in striatal organoids from individuals with FOXP1 variants
Investigating whether the mitochondrial dysfunction and PDE10A dysregulation found in mouse models also appear in human cells, and whether the inhibitor MP-10 improves mitochondrial function in patient-derived striatal organoids.
FOXP1 qualitative assessment and Clinical Global Impression Scale development
No tool currently exists to measure treatment response in FOXP1 syndrome. This study quantifies key parental concerns across age groups to build a FOXP1-specific CGI scale for use as an endpoint in future clinical trials.
Where we focus community-
funded science
Basic Science
Natural History
Clinical Care
Trial Readiness
Fund the next FOXP1 breakthrough
Every gift directly supports projects like these.