Retinal Vasculopathy with Cerebral Leukoencephalopathy (RVCL)
Innovative Research Award: This award supports investigators, whether based in academia or industry, with funds to conduct novel studies that generate insights into and treatment of RVCL.
Purpose: Supporting innovative research ideas is essential to develop treatments for RVCL, an ultra-rare vasculopathy caused by mutations in the TREX1 gene. RVCL is a disease that begins in middle-age and is associated with the destruction of small blood vessels, leading to brain lesions (strokes, visual impairment, dementia) and multiorgan vascular damage. The autosomal dominant mutations are within the C-terminal ~120 amino acids of TREX1. The disease process is 100% penetrant and 100% lethal. RVCL is thought to be noninflammatory and ischemic in nature, characterized by endothelial swelling and obliteration of the lumen of small blood vessels. Underlying pathology has been speculated to be a consequence of alterations in DNA damage repair, dysregulation of innate immune pathways, acceleration of cellular senescence, or dysregulated handling of cellular debris.
Research topics related to RVCL and TREX1: Gene therapy, DNA damage repair, TREX1, endothelial cells, macrophages, stroke, ischemia, chronic kidney disease, ischemic bowel, liver failure, vascular disease, vascular dementia, glycosylation, vaso-occlusion, rare diseases, cell senescence.
Award Amount: Up to six awards per year will be awarded, $100,000 – $300,000 (maximum $300,000 per year) for 1-2 years, with an option to renew yearly based on progress. The Clayco Foundation indirect rate is zero.
We require a non-binding Letter of Intent to be submitted by March 16, 2022, with funding to begin August 1, 2022.
Application requirements:
A two-page research proposal in NIH format that includes the following sections: (A) Specific Aims, Background,(B) Significance, (C) Innovation,(D) Research Approach, and(E) References. (References are not included in page limit.)
NIH Biosketches for all key personnel
A one-page outline to include: (A) Budget justification, (B)Facilities and research environment, (C) Animal or human research subjects (if applicable), (D) Proposed milestones at end year one, for continued funding of a two-year research proposal (if applicable)
Preliminary data encouraged, but not required.
Mouse models and cell lines may be provided with support from the Foundation.
If you have questions about your eligibility or submitting your application, please contact Matthew Halpert, RVCL Project Manager, at halpertm@claycorp.com.
Letters of intent and applications can be submitted via email to halpertm@claycorp.com.
