Research Overview
Over $650,000 committed. 11 active studies. One goal: treatments and a cure for CASK gene disorders.
Project CASK was built on a simple conviction: that parents who refused to wait could change the course of science. Since our founding, we have committed over $650,000 across 11 active studies. Funding gene therapy, biorepository development, iPSC cell line research, small molecule treatments, natural history studies, and more. Every dollar is guided by our global Scientific Advisory Board and coordinated through the Global CASK Therapeutic Roadmap.
$650,000+
committed to CASK research
11
active studies
54
countries in our community
How We Fund Science
Project CASK funds research through a rigorous, SAB-reviewed grants process. Every application is evaluated by independent scientific experts before any dollar is committed. Our two-pronged grant strategy — supporting both foundational science and translational research — ensures we are building knowledge and moving toward clinical application at the same time.
Our Scientific Advisory Board includes leading researchers in neuroscience, gene therapy, rare disease drug development, and clinical medicine. They guide every investment decision, ensuring that Project CASK funds the science most likely to reach our children.
Funded Research
Our full grant portfolio — every study we fund, every researcher we partner with, and the science they are building toward treatments and a cure.
Research Infrastructure
The biological foundation behind the science — our biorepository and iPSC cell line collection through COMBINEDBrain at Van Andel Institute, open-access to researchers worldwide.
→ Explore research infrastructure
Roadmap to a Cure
The Global CASK Therapeutic Roadmap coordinates every research investment across the PC network toward a single integrated strategy — guided by our SAB, updated annually.
Community Research Assets
The ways our CASK families power the science — the patient registry, natural history study, biorepository participation, and more. Every family can contribute.
What We Are Funding
CASK gene disorders are complex. No single therapeutic approach will reach every child. That is why Project CASK funds across multiple therapeutic avenues simultaneously — building a diverse research portfolio that maximizes the chances of a breakthrough reaching patients.
A Global Commitment to the Same Science
Project CASK's research mission extends around the world. Through the PC Global Network, affiliated CASK organizations share our Scientific Advisory Board and our Global CASK Therapeutic Roadmap. Every dollar raised across the network supports the same scientific priorities — moving us all closer to treatments and a cure for CASK gene disorders.
Every Dollar Has a Direction.
Project CASK does not fund science in isolation. Every grant, every study, every research partnership is coordinated through the Global CASK Therapeutic Roadmap — a living strategy guided by our Scientific Advisory Board and updated annually as the science evolves.
When you give to Project CASK, you are not donating into a general fund. You are investing in a specific, accountable, expert-guided research program — one built by parents who refused to wait, and sustained by a community that refuses to stop.
Support the research → projectcask.org/give