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Families of SMA Request for Proposals (RFP) for Pre-clinical Drug Projects in Spinal Muscular Atrophy October 1, 2010 Families of SMA (FSMA) invites proposals for pre-clinical drug discovery programs in SMA, under a competitive review by our Translational Advisory Committee (TAC). Please click here to view TAC member profiles. Suggested guidelines are provided within the RFP, although specifics will likely vary based on project details.
We anticipate awards of one or two milestone-driven projects encompassing some aspect of pre-clinical drug development in SMA, from assay development to IND submission. The award amount is limited to a maximum of $500,000 per year, and funding will be allocated through milestones. Projects can be multi-year in scope with typical project duration of two-to-three years. All programs should have a focused, well-defined, goal-oriented plan for advancement through pre-clinical drug development.
Program Overview: Proposals should focus on innovative methods of identifying novel therapeutic candidates for SMA, either biologics or small molecules. Some priority will be given to strategies that enhance SMN levels or that bypass the need for greater SMN in motor neurons, although other therapeutic approaches with demonstrated proof-of-concept in relevant cellular or animal models of SMA will be favorably considered (see scientific strategy section within for more details). Multi-site projects are encouraged to achieve the diverse expertise required for drug programs.
Program Stage: We invite proposals encompassing the following stages of pre-clinical drug development (projects focused on sub-stages of those listed below are welcome if budget is appropriate, e.g., projects focused solely on novel assay development):
1) Assay Development, High-Throughput Screening, to Validated Hit,
2) Medicinal Chemistry / Drug Optimization, Leading to Clinical Candidate Nomination,
3) IND-Enabling Studies on Identified Clinical Candidates.
Please click here to view the RFP, which provides additional information on preferred scientific strategies, suggested preliminary data, the application process, and the selection process. Overall timeline for this RFP is as follows:
RFP Release Date: October 1, 2010
Letter of Intent Deadline: November 1, 2010 (midnight ET)
Invitation for Full Proposal: December 15, 2010
Proposal Due Date: February 1, 2011 (midnight ET)
Award Notification: July 2011
Please contact Jill Jarecki, Ph.D at Jill@fsma.org for more information about this RFP.
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