Cure SMA is committed to supporting the most promising SMA research, with more than $82 million in funding to date in support of basic research grants and drug discovery programs.
Cure SMA issues requests for proposals in three major research areas:
Basic Research: investigate the causes and biology of SMA, revealing new, more effective ways of treating.
Drug Discovery: Converts basic research ideas into new drug candidates.
Our basic research program is directed by our Scientific Advisory Board, and our drug discovery program by our Translational Advisory Council—both groups of independent experts who carefully evaluate each proposal for scientific excellence and relevance to the Cure SMA mission.
Cure SMA’s research model is intended to attract industry and government funding for clinical trials. Thus, we do not typically issue RFPs in this area. Instead, we focus on clinical trial readiness, infrastructure, and on connecting researchers with potential study participants.
We fund clinical care research through our Cure SMA Care Center Network, and do not typically issue separate RFPs in this area.
For general questions on our research funding model and RFP process for basic research, drug discovery, and newborn screening, please email [email protected]. For questions on the Cure SMA Care Center Network, please contact [email protected].
Basic Research Funding Opportunities
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
- Timra Gilson, Ph.D.
- Emily Welby, Ph.D.
- Dmytro Morderer, Ph.D.
- Rashmi Kothary, Ph.D.
- Stephen Meriney, Ph.D.
- Umrao Monani, Ph.D.
- Lyndsay Murray, Ph.D.
- Charlotte Sumner, M.D.
- Laxman Gangwani, Ph.D.
- Umrao Monani, Ph.D.
- Alberto Kornblihtt, Ph.D.
- Krysta Engel, Ph.D.
- Jocelyn Cote, Ph.D.
- Chad Heatwole, M.D.
- Charlotte Sumner, M.D.
- Bakri Elsheikh, M.D.
- Allison Ebert, Ph.D.
- Brunhilde Wirth, Ph.D.
- Robert Kalb, M.D.
- Robin Parks, Ph.D.
- Kathryn Swoboda, M.D.
Drug Discovery Funding Opportunities
In the past two decades, Cure SMA has invested $20 million in funding for 12 drug development projects that have led to effective treatments for SMA. Our continued pre-clinical drug discovery program invests funding in innovative and promising projects that will lead directly to creating new viable drugs for treating SMA, both in combination and new muscle-targeting therapies.
We do not currently have an RFP open for drug discovery. Please check back often for an announcement on the expected timing of our next drug discovery RFP. Email Cure SMA’s Research Team at [email protected].
See more information about the drug discovery programs that we have funded: