CDKL5 Deficiency Disorder (CDD) is a rare, X-linked neurodevelopmental condition caused by mutations in the CDKL5 gene, leading to early-onset seizures and severe cognitive impairments due to loss of enzymatic function and resulting neuronal hyperexcitability. Although CDKL5-knockout animal models reproduce some disease features, they frequently fail to model core aspects such as epilepsy, limiting their value for preclinical drug development.
CNS-3D technology provides a robust and scalable means for modeling CDKL5 Deficiency Disorder, capturing disease-related phenotypes and enabling high-throughput drug screening. This approach led to the identification of 22 promising therapeutic candidates, consisting of 15 unique biological targets in less than one year. Built on this strong foundation, CNS-3D technology is poised to make a transformative impact on therapeutic development for CDKL5 Deficiency Disorder and related CNS disorders.