Basic Research Funding Opportunities

Basic research is one of the cornerstones of Cure SMA’s research strategy. We believe that continued investigation into the causes and biology of SMA is necessary to grow and improve our drug pipeline.

Each year, our Scientific Advisory Board identifies specific priorities for funding, which are then released in a request for proposals. Once submitted, all proposals are evaluated using an NIH-like scoring system based on both scientific quality and relevance to the Cure SMA research mission. 

Current RFP

Cure SMA is pleased to announce that we are accepting grant applications for funding of research projects, under a competitive review by our Scientific Advisory Board (SAB). View the complete SAB roster. Please note that Cure SMA will not release a separate drug discovery RFP in the next twelve months.

Program Overview

This RFP is now closed. Decisions on funding will be announced in 2017. Applications will be accepted for one or two years of funding with a maximum funding level per year of $75,000 and a total award of $150,000 (including 8% overhead) over the duration of the entire project. Applications will be accepted from either principal investigators or post-doctoral fellows.

Scientific Priorities

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.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, 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.
  • 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, and newborn screening protocols / technologies.

Application Process

DOWNLOAD A GRANT APPLICATION 

To apply for a basic research grant, please download a copy our grant application form. Please submit an electronic copy as well as 4 hard copies and the original to Cure SMA. The electronic copy should be sent to jill@curesma.org - include the PI’s surname and Basic Research in the subject line. Hard copies should be sent to:

Cure SMA
925 Busse Road
Elk Grove Village, IL 60007

Review Criteria

All applications are reviewed by our Scientific Advisory Board (SAB), on both scientific quality and relevance to the Cure SMA research mission of accelerating the discovery of safe and effective treatments for SMA.

Timeline

RFP Release Date: July 12, 2016
Proposal Due Date: September 2, 2016 (midnight ET)
Award Notification: By January 31, 2017

Questions?

Email Jill Jarecki, PhD, research director, at jill@curesma.org.

Past Funding

Since 2004, we've funded nearly $11 million in basic research grants. See more information about our past grants:

Scientific Advisory Board

Research Director

Jill Jarecki, PhD

Elliot J. Androphy, MD,  Kampen-Norins Professor and Chair of the Department of Dermatology of Indiana University School of Medicine; SMA Biology. 

Arthur H. Burghes, PhD, Professor of Molecular and Cellular Biochemistry at the Ohio State University; SMA Biology and Animal Models. 

Tom Crawford, MD, Associate Professor of Neurology and Pediatrics at the Johns Hopkins School of Medicine; Child Neuromuscular Disorders.  

Stephen J. Kolb, MD, PhD, Associate Professor of Neurology and Biological Chemistry & Pharmacology, The Ohio State Wexner Medical Center; Molecular Pathology of SMA, Biomarkers, and Clinical Trials.

Rashmi Kothary, PhD, Associate Director and Senior Scientist at the Ottawa Hospital Research Institute and a Professor at the University of Ottawa; SMA Biology and Animal Models. 

Adrian Krainer, PhD, Professor at the Cold Spring Harbor Laboratory; SMN splicing. 

Umrao Monani, PhD, Associate Professor of Pathology and Cell Biology, Columbia University; SMA Biology and Animal Models.

Samuel Pfaff, PhD, Investigator of the Howard Hughes Medical Institute and Professor at the Gene Expression Laboratory at the Salk Institute for Biological Studies in La Jolla, California; Motor Neuron Biologist. 

Mark Rich, MD, PhD, Associate Professor in the Department of Neuroscience, Cell Biology, and Physiology at Wright State University; Neuromuscular Junction Biology. 

Kathryn Swoboda, MD, Associate Professor of Neurology and Pediatrics at the University of Utah School of Medicine, and Director of the Pediatric Motor Disorders Clinic at Primary Children’s Medical Center in Salt Lake City, Utah; Child Neuromuscular Disorders. 

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