COLLABORATIVE PROGRAM TO ACCELERATE THERAPEUTICS DEVELOPMENT FOR SPINAL MUSCULAR ATROPHY (SMA) RELEASE DATE: April 8, 2003 NOTICE: NOT-NS-03-012 RFP Available: NIH-NINDS-RFP-03-03 National Institute of Neurological Disorders and Stroke (NINDS) The National Institute of Neurological Disorders and Stroke (NINDS) has the stated mission to reduce the burden of neurological disease a burden borne by every age group, by every segment of society, by people all over the world. To support this mission, NINDS: o Conducts, fosters, coordinates, and guides research on the causes, prevention, diagnosis, and treatment of neurological disorders and stroke, and supports basic research in related scientific areas. o Provides grants-in-aid to public and private institutions and individuals in fields related to its areas of interest, including research project, program project, and research center grants. Provides individual and institutional fellowships to increase scientific expertise in neurological fields. o Operates a program of contracts for the funding of research and research support efforts in selected areas of institute need. o Conducts a diversified program of intramural and collaborative research in its own laboratories, branches, and clinics. o Collects and disseminates research information related to neurological disorders. NINDS has targeted Spinal Muscular Atrophy (SMA) for a pilot program in accelerated therapeutics development. SMA is a devastating neurological disease of childhood for which no effective treatment is currently available. SMA causes degeneration of motor neurons in the spinal cord and brainstem, leading to muscle weakness and, in severe forms of the disease, death. It is the most severe of the common genetic neurological diseases and is the leading heritable cause of infant death. SMA is an ideal candidate for targeted therapeutics development. It is caused by the loss of function of a known gene, SMN1. Thus, there are tractable strategies for developing treatments based on replacement of the lost function. Multiple therapeutic avenues are available, including drug development, gene therapy and stem cell therapy. NINDS' research goal for Spinal Muscular Atrophy is to efficiently translate a mechanistic understanding of SMA into a testable therapy suitable for clinical trials. The NINDS strategy for achieving its research goal is to fund parallel therapeutic pathways in a rapid milestone-driven process that will identify a promising therapeutic candidate. This project will require a high level of scientific expertise and experience on the part of the contractor. Furthermore, efficient progress will involve an unusual degree of acceleration and integration of multiple research projects, and thus success will also require that the contractor use creativity and flexibility in applying that scientific expertise. It is anticipated that one award may be made for a period of four years in September 2003. Request for Proposals (RFP) NIH-NINDS-03-03 will ONLY be AVAILABLE ELECTRONICALLY at either one of the following websites: http://www.FedBizOpps.gov/ or http://www.ninds.nih.gov/funding/funding_announcements/RFP_all.htm, 15 or more calendar days after the issuance of this synopsis. OFFERORS ARE RESPONSIBLE FOR ROUTINELY CHECKING EITHER WEBSITE FOR ANY POSSIBLE SOLICITATION AMENDMENTS THAT MAY BE ISSUED. NO INDIVIDUAL NOTIFICATION OF ANY AMENDMENTS WILL BE PROVIDED. All responsible sources may submit a proposal, which shall be considered by the agency. Refer to numbered note 26.**** INQUIRIES Inquiries may be directed to: Patricia S. Denney, Contracting Officer Contracts Management Branch National Institute of Neurological Disorders and Stroke Neuroscience Center, Room 3287 6001 Executive Boulevard, MSC 9531 Bethesda, MD 20892-9531 Tel: (301) 496-1813 Fax: (301) 402-4225 Email: PD22N@NIH.GOV

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