CASK GENE RESEARCH STRATEGY
Project CASK believes a “cure” for CASK-related disorders is possible. This could be in the form of a single gene therapy or multiple therapeutics, depending on a person’s variant. Getting there requires close, transparent and iterative collaboration among basic and translational researchers, clinicians, biotechs, pharmaceutical companies and the CASK community.
It also requires an engaged community working with urgency to contribute to the toolkit of research assets necessary for testing therapeutic options and to make strategic investments to move the science as quickly and safely as possible toward improving the lives of people with CASK gene disorders and their families.
CASK SCIENTIFIC COLLABORATION
One of the first initiatives Project CASK organized was a CASK Think Tank in December 2023. The meeting was the first-ever multidisciplinary scientific meeting on CASK gene disorders and brought together 30 scientists to discuss the most critical unanswered questions and identify the most promising strategies to pursue. The meeting informed Project CASK’s research strategy, including the focus of the first Request for Applications launched in January 2024. It also sparked new research partnerships, creating opportunities to fast-track the exploration of potential and novel treatment avenues.
Collaboration is at the heart of Project CASK’s approach. Building on the experience of the Think Tank, Project CASK will continue to convene scientists from different disciplines to exchange perspectives and incremental discoveries of relevance to accelerate the path to treatments. This will enable Project CASK to harness the speed of discoveries and new technologies for treating rare diseases and ensure an efficient and transparent research strategy. This collective effort will limit duplication of research, end laboratory isolation, and promote continuous learning and knowledge-sharing.
Thematic meetings and ad hoc research updates with Project CASK grantees will ensure new findings can systematically inform ongoing research, prospective research and Project CASK’s overall research strategy.