SYNOPSIS

Coalition to Cure Calpain 3 (C3) provides funding for research and translational projects to expand the understanding of calpain 3 and Calpainopathy (including LGMD2A/R1 and LGMD1I/D4) to enable development of therapeutic approaches for this disease. 

This award is intended for both US and non-US based academic and biopharmaceutical organizations. The requested budget should not exceed $250,000 in direct costs. This award cannot be used for indirect costs and overhead charges.

GRANT FOCUS AREAS

C3 is actively soliciting grant proposals for research related to understanding Calpainopathy in the context of developing a therapeutic including, but not limited to: 

• Identification and testing of small molecules that protect or restore degenerating and/or dysfunctional skeletal muscle, or non-genetic agents that increase calpain 3 levels/stability
• Development of calpain 3 activity assay that works in human muscle biopsies
• Identification of serum biomarkers
• Creation of an assay to assess pathogenicity of variants of unknown significance in the CAPN3 gene
• Development of reagents that are accessible to the research community, such as an antibody that provides accurate immunostaining of calpain 3 in human muscle biopsies
• Studies to explore the consequences of dominant and recessive CAPN3 variants on calpain 3 biochemistry
• Innovation in Calpainopathy clinical trial readiness including the development of outcome assessments

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