Orphan Disease Center and Loulou Foundation - 2022 CDKL5 Pilot Grants

Applications are invited for one-year grants in support of research related to CDKL5 Deficiency Disorder (CDD). Priority will be given to grants that cover the following areas:

• Novel therapeutic approaches for CDD, including, but not limited to, techniques in genome editing, RNA-based mechanisms, biologics, novel cell-based therapeutics, network modulation, and development of novel therapeutic compounds, including through small molecule repurposing or screening against validated phenotypes in human cellular systems.

• Establishment of the link between molecular function of CDKL5 and disease pathophysiology in cellular or animal disease models through rescue or modulation of molecular, cellular, or behavioral deficits via pharmacological or genetic / gene therapy interventions.

• Systems biology and computational modeling approaches to provide a deeper understanding of CDKL5 function, downstream effectors, intracellular signaling, protein interactor networks, or genetic modifiers from model organisms and human cellular models, including regulators of CDKL5 gene expression.

• Novel application of imaging and functional techniques to characterize the disease state of CDD pre-clinical models or in the clinical setting.

• Discovery and validation of CDKL5 biomarkers (molecular and functional) with the goal of their translation to the clinical setting. Of particular interest are approaches to biomarker discovery using minimally invasive testing (e.g., peripheral fluid analysis by metabolomics or other ‘omics approaches).

Selected applicants will be invited to submit a full application by March 25, 2022.