OUR STRATEGY TO FIND TREATMENTS AND A CURE FOR CASK

Our strategy - characterized by continuous, multidisciplinary collaboration and a two-prong grant strategy - will help overcome challenges related to fragmented funding and research efforts while also sparking innovation to explore emerging avenues to harness the incredible scientific and technological revolution underway.

We will explore multiple therapeutic avenues, including drug repurposing, gene therapy, antisense oligonucleotides (ASO) strategies, RNA strategies, as well as studies that will help address symptoms such as epilepsy and low mitochondrial function. While there are promising avenues opening all the time, we recognize that our role is also to de-risk the science by engaging the community in creating much-needed research assets and having as many options

underway as possible to increase the likelihood of positive outcomes. We will determine the best strategies to start with through two initiatives: First, in November, we will organize a CASK Think Tank in partnership with the Orphan Disease Center at UPenn, bringing together 25-35 researchers and clinicians to discuss what we do and don't know about the CASK gene. We will fund at least two small grants from proposals on areas of needed research, submitted after the Think Tank. 

Second, we are exploring direct grant opportunities through outreach to scientists proposing an approach that seems promising for CASK gene disorders. We will fund proof of concept studies for avenues that could lead to CASK treatments and a cure. The final decision on which avenues to pursue will be taken by the Board based on the recommendations and guidance of the Scientific Advisory Board and our Chief Scientific Officer. We will also start funding the development of Induced Pluripotent Stem Cells (iPSCs) and mouse models that most researchers need to test therapeutic options for the CASK gene.