CDKL5 Pilot Grant Program

CDKL5 Pilot Grant Program

Status: ACTIVE
Funder: Trustees Of The University Of Pennsylvania
Amount: US $150,000
Last Updated: March 31, 2026

Summary

The CDKL5 Pilot Grant Program offers a one-year grant of $150,000 to support research into CDKL5 Deficiency Disorder (CDD), a serious neurodevelopmental condition. This program invites innovative proposals aimed at discovering new treatments or understanding the disease mechanisms. Priority will be given to projects exploring novel therapeutic approaches, linking molecular functions to disease, and developing biomarkers. Institutions are encouraged to waive indirect costs to maximize funding for research efforts.

Overview

All applicants must first submit a Letter of Interest (LOI) to be reviewed for consideration of a full application submission. Applicants will be notified via email with a decision regarding their LOI, which, if positive, will invite the applicant to submit a full application ( full proposal above). CDKL5 Pilot Grant Program The ODC and Loulou Foundation CDKL5 Pilot Grant Program provides a one‐year grant for $150,000.00 (total cost) to support research related to CDKL5 Deficiency Disorder (CDD). The number of awards is not fixed, and may vary. Background CDKL5 Deficiency Disorder (CDD) is a monogenic, neurodevelopmental disorder characterized by treatment-resistant epilepsy and severe neurodevelopmental delay. The disease is caused by loss-of-function mutations in the CDKL5 gene, which encodes a protein kinase whose expression is required for proper neuronal function. The mechanisms by which loss of CDKL5 expression leads to this neurodevelopmental disorder remain unclear. The gene encoding this protein is located on the X chromosome, with heterozygous females primarily affected. The disease does not exhibit neurodegeneration, and animal models strongly suggest the potential for reversibility of phenotypes associated with loss of CDKL5. While clinical development of novel therapeutics is underway, the current standard of care is not fully effective at managing seizures in all patients, or in the treatment of non-seizure symptoms such as neurodevelopmental or motor deficits. We are seeking grant applications that progress the discovery or development of treatments and/or cures for CDKL5 Deficiency Disorder. Because many gaps still remain in our understanding of the biology of CDKL5 and its role in neurological development and function, applications that address such gaps in basic science are welcome, provided that they are tethered to the development of a potential therapy. While the RFA is broad in scope, priority will be given to grants that cover the following areas: Novel therapeutic approaches for CDDEstablishment of a link between molecular function of CDKL5 and disease pathophysiologySystems biology and computational modeling approachesDiscovery and validation of CDKL5 biomarkers:Molecular biomarker discovery and validation of candidate biomarkersNovel application of imaging and functional techniques Funding The total award amount is $150,000 (including direct and indirect costs). It is strongly preferred that applicant institutions waive indirect costs, thereby allowing the total amount to go towards research, but indirect costs up to a maximum of 10% could be accepted in exceptional cases.

Eligibility

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