The Orphan Disease Center (ODC) – Loulou Foundation: CDKL5 Program of Excellence – Pilot Grant Program

The ODC and Loulou Foundation are seeking grant applications that progress the discovery or development of treatments and/or a cure for CDKL5 Deficiency Disorder (CDD). Basic science projects that address these gaps are welcome as long as 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:

  1. Novel therapeutic approaches for CDD, including but not limited to techniques in genome editing, RNA-based mechanisms, biologics, and small molecule repurposing.
  2. Approaches to validate phenotypes in CDKL5 function or disease pathophysiology through rescue of phenotypic deficits with pharmacological or genetic / gene therapy techniques. Phenotypic reversal in rodent models will focus on the use of adult (2 months of age or older) animals. In particular, approaches are encouraged which allow the identification of individual CDKL5 protein isoforms (arising from alternative splicing, alternative promoter usage, or post-translational modifications) which can rescue these phenotypes.
  3. Systems biology and computational modeling approaches to provide a deeper understanding of CDKL5 function, downstream effectors, signaling, protein:protein interactors, or modifiers, including regulators of CDKL5 gene expression (transcriptional, post-transcriptional/RNA processing, translational, post-translational).
  4. Novel imaging and functional approaches to phenotyping CDD in pre-clinical models or the clinical setting. A non-exclusive list of topics that would be responsive to this RFA is listed: Functional/structural MRI; diffusion tensor imaging (DTI); Magnetic resonance spectroscopy (MRS); Stimulus-induced event-related potentials: impact of CDKL5 genetic / gene therapy or pharmacological interventions on deficits in stimulus-induced event potentials (visual, auditory, or other) in CDD disease models
  5. Discovery and validation of CDKL5 biomarkers and their translation to the clinical setting.

Funding & Duration

One-year grants of US $150,000 (approx. AUD $192,000) are available. The number of awards may vary.


All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible.

Submission Requirements & Due Dates

Further Information

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