Drug Discovery Funding Opportunities

Cure SMA has invested more than $20 million in drug discovery since 2000. Our pre-clinical drug discovery program invests funding in innovative and promising projects that will lead directly to creating viable drugs for treating SMA.

Current RFP

Cure SMA invites proposals for pre-clinical drug discovery programs for Spinal Muscular Atrophy (SMA), under a competitive review by our Translational Advisory Committee (TAC). 

Questions? Email research@curesma.org.

Program Overview

We anticipate funding two programs to advance innovative therapeutic candidates in SMA, either biologics or small molecules. Proposals should involve obtaining proof-of-principle data or progressing specific aspects of ongoing preclinical programs. Non-SMN directed approaches are highly encouraged. We also welcome SMN-enhancing approaches with significant advantage over current options or with different mechanisms of action allowing for combination use.

Scientific Priorities 

Awards are intended to provide exploratory seed funding for early-stage program or to provide gap funding for ongoing programs. This first area focuses on proof-of-concept studies for innovative new approaches or mechanisms of actions for SMA therapeutics. Projects should involve obtaining proof-of-concept data in disease relevant animal or cellular models, which would justify further investment in the approach. In particular, Cure SMA has an interest in non-SMN based approaches.

The second area focuses on funding of ongoing SMA drug programs. Funded studies could include a subset of the following: 1) assessment of pharmacology / biodistribution, 2) animal model testing, 3) in vitro ADME data (hCYP inhibition, microsomal stability, etc.), 4) in vitro screening of safety (Ames, hERG, etc.), 7) animal toxicology studies, 8) new delivery methods, 9) optimizing manufacturing processes, or 10) IND preparation.

Application Process and Instructions for Letter of Intent (LOI)

A letter of intent is due by February 25, 2018. Letters of Intent (LOI) should be submitted by email to Jackie@curesma.org. The full application form will be provided upon acceptance of the Letter of Intent, which will occur by March 3, 2018. Full proposals are due April 2, 2018.

Please provide no more than a 2-page project summary (11 point font or larger) for the LOI. Include the following:

  1. brief outline of scientific strategy,
  2. brief summary of the preliminary data, including existing resources / assays,
  3. overall project goals and timeline, 
  4. specific aims for Cure SMA funding, and 
  5. names & affiliations of personnel, with contact information for PI.

The LOI is solely intended to assess alignment of priorities between applicants and Cure SMA for submission of a full proposal.

Review Criteria and Process 

Proposals will be peer-reviewed by the Cure SMA Translational Advisory Committee (TAC). They will be scored based on:

  • scientific strategy, 
  • experiment plan, 
  • impact of Cure SMA funding on the overall program,
  • investigator experience and environment, and
  • budget.

TAC members will review proposals under a non-disclosure agreement to ensure data confidentiality.


RFP Release Date: February 5, 2018
Letter of Intent Deadline: February 26, 2018
Invitation for Full Proposal: March 3, 2018
Proposal Due Date: April 2, 2018 (midnight ET)
Award Notification: May 1, 2018

Cure SMA Contact

Jackie Glascock, Ph.D.
Scientific Program Manager
925 Busse Road, Elk Grove Village, IL 60007
(800) 886-1762

Past Funding

See more information about the drug discovery programs that we have funded:

Translational Advisory Council

Christine Brideau, PhD, Vice President, In Vitro Biology, WuXi AppTec, Preclinical Drug Development 

Arthur Burghes, PhD, Professor, The Ohio State University, SMA Biology  

Peter Grootenhuis, PhD, Senior Director, Vertex Pharmaceuticals, Medicinal Chemistry  

Peter Hodder, PhD,  Executive Director of Discovery Research, Amgen Inc., Assay Development and High-Throughput Screening 

Jim Inglese, PhD, Head, Laboratory of Assay Development & Screening Technology (ADST), National Center for Advancing Translational Sciences, Assay Development and High-Throughput Screening   

Joseph W. Lewcock, PhD, Head of Biology Discovery, Denali Therapeutics, Preclinical Drug Development

Brian Pollok, PhD, Prinicipal, Rapidan BioAdvisors, Assay Development

Timothy Reilly, PhD, DABT, Director, Bristol Myers Squibb, Toxicology

Lee Rubin, PhD, Director of Translational Medicine, Harvard Stem Cell Institute, Neuronal Assay Development and High-Throughput Screening 

Jasbir Singh, PhD, President, Jasin Discovery Solutions, Medicinal Chemist 

Charlotte Sumner, MD, Associate Professor, Johns Hopkins University, SMA Biology/Neurology  

Michael Vellard, PhD, Vice President of Research, Ultragenyx Pharmaceutical, Preclinical Drug Development 

Chien-Ping Ko, PhD, Professor, University of Southern California, SMA Biology/Neurobiology

Dione Kobayashi, PhD, Executive Scientist and Rare Disease Drug Developer, Myonexus Therapeutics, Inc., Preclinical Drug Development 

© 2019 Cure SMA | Cure SMA is recognized as tax-exempt under section 501(c)(3) of the Internal Revenue Code. EIN: 36-3320440. | Privacy Policy

Powered by Blackbaud
nonprofit software