In the past two decades, Cure SMA has invested $15 million in funding for 128 basic research grants that have furthered our understanding of SMA. Our Scientific Advisory Board identifies priorities for funding, which are released in RFPs. Once submitted, all proposals are evaluated using a scoring system based on both scientific quality and relevance to the Cure SMA research mission.
Click here to access the full RFP.
Click here to access the grant application form.
Cure SMA wants to encourage applications on novel research that will enhance our understanding of SMA disease pathology at the molecular, cellular, and biochemical level; generate key reagents and tools to facilitate drug development and clinical trials; and identify new therapeutic strategies for SMA, in particular combination approaches. Cure SMA welcomes high risk, high reward projects and values the ability to provide seed funding for these types of exploratory projects. Please note that high risk projects are generally not awarded maximum funding without compelling preliminary data.
We envision that studies funded by this Research RFP will fall into one the following key areas:
- Studies focused on the molecular and biochemical mechanisms regulating SMN expression or mediating SMN function. Results should lead to a better understanding of the requirements for SMN protein biologically. There is a particular interest in identifying genetic modifiers, upstream regulators of SMN expression / splicing / function, and downstream effectors of SMN functional activity, resulting in new drug targets for SMA.
- Studies resulting in greater understanding of the pathophysiology of SMA, using well-validated animal or cellular models of the SMA. This includes focus on the tissue or timing requirements for SMN protein, the cellular autonomy of the disease in motor neurons and other cells, understanding the basis of motor neuron selectively, peripheral versus central manifestations of SMA, and others.
- Studies focused on early proof-of concept assessment of novel therapeutic approaches for SMA in well-validated animal or cellular models of the disease or on progressing aspects of ongoing preclinical drug programs for SMA towards IND. Proposed SMN enhancing approaches should have advantage over current candidates or have the ability to be used in combination. Particular interest exists in non-SMN based approaches with the potential for combination use with SMN up-regulation strategies. This includes approaches to be used in combination or alone to address the unmet needs of patients in symptomatic phases of disease.
- Work focused on generating research or clinical trial tools for SMA, such as new animal models, phenotypic cellular assays, activity assays for SMN function, biomarkers or outcome measures for clinical trials.
- RFP Release Date: April 18, 2022
- Proposal Due Date: July 15, 2022 (midnight ET)
- Award Notification: By December 1, 2022