NTSAD is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies in the following diseases: Tay-Sachs, GM-1, Sandhoff, and Canavan.
Basic research and translational studies should generate strong preliminary data to enable future major funding by other third parties. Projects may be in such areas as drug delivery to the brain, new animal models designed to facilitate translational research and drug discovery, assay development for drug screening, substrate reduction, stem cells, molecular chaperones, gene therapy, and biomarkers, as well as exploring other novel therapeutic strategies.
In addition to soliciting proposals for novel research projects, we strongly encourage applicants to submit proposals that address one or more of the following: understanding disease progression and natural history as well as unmet needs from a patient perspective across the severity spectrum of any given indication, development of patient registries, translational biomarkers with clinical utility, measurable and clinically meaningful efficacy endpoints for clinical trials , clinical research networks, and newborn screening.
The pre-applications will be reviewed by sub-committees of NTSAD’s Scientific Advisory Committee and Corporate Advisory Council. Selected applications will be invited to submit full applications for further peer review and funding consideration. Grant awards are based on proposal evaluation by a Research Evaluation Subcommittee of NTSAD’s Scientific Advisory Committee. The grant awards will be announced in April. The funding period begins approximately on July 1, 2017, pending the Institution’s agreement to NTSAD’s Research Grant Policies.
Download the 2016-2017 RFP Guidelines and other important dates here.
The deadline for pre-application proposals is 5:00 p.m (EST), Wednesday, January 4, 2017.