Request for Proposals for Pre-Clinical Drug Projects in Spinal Muscular Atrophy
Families of SMA (FSMA) invites proposals for pre-clinical drug discovery programs for Spinal Muscular Atrophy (SMA), under a competitive review by our Translational Advisory Committee (TAC).
Proposals should involve innovative methods of advancing novel therapeutic candidates for SMA, either biologics or small molecules. Novel methods of boosting SMN levels and non-SMN based approaches are both welcome. We anticipate granting awards for two SMA projects focused either on obtaining proof-of-principle data for early-stage exploratory projects or on specific aspects of ongoing preclinical drug programs. Awards will be for one year with maximum funding of $200,000 (including 8% overhead).
Program Stage: We invite proposals encompassing any stage of pre-clinical drug development from early discovery to IND submission to the FDA.
Scientific Focus: Projects should be focused in two areas. The first area of focus is on validating innovative new approaches or mechanisms of actions for potential SMA drugs. Projects will involve obtaining proof-of-concept data in disease relevant animal or cellular models, which would justify further investment in the approach. Non-SMA based approaches would be well suited for this focus area. FSMA anticipates a 2013 drug discovery RFP to enable programs of larger scope with validated mechanisms of action.
The second focus is for specific parts of ongoing SMA drug programs, based on already validated scientific strategies. 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, or 8) IND preparation.
Selection Process: All proposals will be peer-reviewed by the FSMA Translational Advisory Committee (TAC). They will be scored based on 1) the scientific strategy / feasibility of the proposal, 2) quality of the methods / research plan, 3) impact of FSMA funding on the overall project, 4) investigator experience, environment / resources, and 5) appropriateness of budget. All members of the TAC will review proposals under a confidentiality agreement with FSMA to ensure the privacy of applicant ideas and data.
Application Process: A letter of intent is due by July 20, 2012. Letters of Intent should be submitted by email to Jill@FSMA.org. Instructions for the Letters of Intent can be found in Appendix 1* below. The full application form will be provided upon acceptance of the Letter of Intent, which will occur by July 31, 2012. Full proposals are due August 31, 2012.
Proposals will be accepted from independent investigators at public or private institutions, for-profit or nonprofit organizations, and foreign institutions. Applying to this RFP will not preclude an application for a FSMA Basic Research Grant, also with a deadline of August 31, 2012, given it is for a distinct project.
Overall Timeline:
RFP Release Date: June 28, 2012
Letter of Intent Deadline*: July 20, 2012
Invitation for Full Proposal: July 31, 2012
Proposal Due Date: August 31, 2012 (midnight ET)
Award Notification: December 2012
Appendix 1: Instructions for Letter of Intent.
Please provide no more than a 2-page project summary (11 point font or larger). Include the following: 1) brief outline of scientific strategy & rationale, 2) brief summary of the preliminary data, including existing resources / assays, 3) overall project goals and timeline, 4) specific aims for FSMA 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 FSMA for submission of a full proposal.
Please contact Jill Jarecki, Ph.D at Jill@fsma.org for more information about this RFP.
