Path to a Cure Postdoctoral Research Fellowship Award

This program supports research that will accelerate the discovery and development of therapies to restore cystic fibrosis transmembrane conductance regulator (CFTR) function in all patients with cystic fibrosis. 4 min read In this article Small-moleculeCFTR modulators that directly target the mutant protein are powerful therapies that will likely benefit 90 percent of people with cystic fibrosis in the near future. However, there is still a significant unmet need for people with CFTR mutations who either do not respond to modulators, do not generate sufficient quantities of protein for correction, or block protein synthesis (i.e., premature stop codon mutations, splice mutations, insertion/deletion mutations, etc.). To ensure all people with CF have access to effective CFTR-directed therapies, the Cystic Fibrosis Foundation announced thePath to a Cure (PTAC) initiative in 2019. Award Types

There are three program options: Award Type PTAC Research Grant

Up to $150,000 per year plus 12% indirect costs for up to threeyears PTAC Pilot and Feasibility Award

Up to $50,000 per year plus 12% indirect costs for up to twoyears PTAC Postdoctoral Research Fellowship Award

Up to $75,000in the first year, and up to $76,000 in the second year. Indirect costs are not allowable.

Focus of Proposals

Projects supported through the PTAC initiative should focus on foundational concepts, strategies, novel tools and methods, and/or technologies that have the potential to inform or ultimately translate into novel therapies to restore CFTR protein function or fix/replace the defective CFTR gene: Identifying, characterizing, and validating potential targets to promote nonsense mutation suppression, which includes understanding the pathways and mechanisms that regulate translation termination and nonsense mediated decay (NMD) Developing novel means of repairing and/or replacing the mutant CFTR gene Characterization of cellular targets for CFTR correction, including airway progenitor cells and other affected epithelial tissues (biliary tract, GI tract, pancreas) Developing and optimizing the chemistry and formulation of nucleic acid delivery vehicles, both viral and non-viral, that can target disease relevant cells and tissues Comprehensive evaluation of the role of various pulmonary cell types in CF disease pathogenesis (i.e. ciliated cells, club cells, ionocytes, etc.) and the region of the lung that is necessary to target with a genetic-based therapy to prevent, halt, or reverse disease Methods to overcome barriers that limit delivery of genetic therapies to disease relevant cells and tissues Cell, tissue, and animal models to discover and develop nucleic acid delivery vehicles and/or methods of restoring or repairing CFTR Tools and assays to pre-clinically evaluate the efficacy and safety of restoring functional CFTR through gene repair, gene replacement, small molecules, or any other novel method General Guidelines and Eligibility

Please see individual policies and guidelines for additional guidelines and eligibility criteria. Eligibility requirements vary by award type. Policies and Guidelines

Please review the appropriate Policies and Guidelines for complete submission information: Deadline for all applications: May 2, 2024 Those who are interested in any funding programs offered by the CF Foundation can get further information, or discuss the potential relevance of their studies or research, by contacting the Grants and Contracts Management and Administration (GCMA) office atgrants@cff.org . Please Direct Inquiries to: GCMA Office Cystic Fibrosis Foundation 4550 Montgomery Ave. Suite 1100 N Bethesda, MD 20814 800-FIGHT-CF 301-841-2614 Share this article Topics Topics

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Information for Applicants and Awardees

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PTAC Research Grant Policies and Guidelines

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PTAC Pilot and Feasibility Award Policies and Guidelines

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PTAC Postdoctoral Research Fellowship Award Policies and Guidelines

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