CDKL5 Drug Repurposing

CDKL5 Drug Repurposing is a therapeutic program developed by Child's Cure Genetic Research (CCGR) in partnership with Rarebase, PBC. The program centers on an undisclosed FDA-approved small molecule, originally used as an adjunct therapy for epilepsy, which has been repurposed to treat CDKL5 deficiency disorder (CDD). The drug's mechanism of action involves targeting CDKL2, a genetic paralog of CDKL5 that shares approximately 33% functional overlap. By modulating CDKL2, the treatment aims to provide functional compensation for the deficient CDKL5 protein. Preclinical validation was conducted at the Royal College of Surgeons in Ireland using CDKL5 mutant cell lines and mouse models, showing promising results. The program is currently in the preclinical stage, with clinical trials planned at Johns Hopkins University, and some patients have already accessed the medication via off-label compassionate use under medical supervision.

Other names

Child's Cure Genetic Research-CDKL5-small moleculeCDKL2 modulatorCDKL-2 modulatorCDKL 2 modulator