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First Project CASK Request for Applications! 


Project CASK has launched its first Request for Applications, building on the discussion at the CASK Think Tank on 4 December 2023. Managed in collaboration with the Orphan Disease Center, University of Pennsylvania, we are seeking proposals that will make a significant impact on the CASK community and accelerate our path to effective therapeutics for CASK gene disorders in one of two categories:


Seed grants of up to $50,000 for 12 months:

These grants will support studies to better understand CASK gene disorders and/or support clinical studies. Proposals should respond to key gaps in the knowledge of or research assets for CASK disorders that are critical to the pursuit of therapeutic development, including future clinical trials.


Translational grants of up to $250,000 for 24 months:

These grants will drive forward translational research from drug repurposing and small molecules to gene therapies including forward-looking approaches to address current limitations specific to the CASK gene, RNA editing or other RNA based approaches, and novel techniques for x-linked neurological disorders.


Deadline for the Letter of Intent: 16 February 2024


Please download the application and instructions on the ODC website here

For questions, please contact



Our children's bright future is in your hands. Thank you for your passion for science, urgency and transparency. We look forward to seeing your fruitful hard work and collaboration!




Project CASK is dedicated to finding a cure for CASK gene disorders. To this end, we aim to support translational collaborative research that moves laboratory findings to clinical trials as urgently as possible, with safety and efficacy as guiding principles.


We will also support basic research studies that respond to the most critical unanswered questions about CASK gene disorders that if answered will help discern the best therapeutic strategies to take forward. 


Based on the roadmap, the small grants program will be the engine to expand basic science research, drug repurposing and the development of novel therapeutics, and the exploration of curative therapies across avenues of gene therapy, ASOs and RNA strategies, among others. Sparking new lines of inquiry will be essential to accelerating the path to a cure, reducing risk across therapeutic options, and enabling treatment options for all people affected by CASK gene disorders regardless of the type of mutation. The program will be useful for junior faculty in the early stages of their careers, established investigators in other areas of research who wish to pursue CASK research, or investigators working on CASK who are seeking funding to support pilot studies in new areas of research. All projects supported through the small grants program must focus on translational research – taking research from the lab and applying it to methods that will directly benefit people affected by CASK - or basic research for critical unanswered questions as defined by Project CASK.


Project CASK will fund avenues to identify possible biologically active compounds to be developed into clinical candidates. This will include validation of a drug target to optimizing drug safety and effectiveness for human clinical trials.


As promising treatment and curative avenues emerge from the small grants program or through other avenues, Project CASK will raise funding to ensure that necessary resources are available to realize these options.

Per a Board-directed policy, Project CASK does not support indirect costs for grants.

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