Small-molecule CFTR 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 the Path to a Cure (PTAC) initiative in 2019.
There are three program options:
|Award Type||Funding Level|
|PTAC Research Grant||Up to $150,000 per year plus 12% indirect costs for up to two years|
|PTAC Pilot and Feasibility Award||Up to $50,000 per year plus 12% indirect costs for up to two years|
|PTAC Postdoctoral Fellowship Award||Up to $75,000 in 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:
- Path to a Cure (PTAC) - Research Grant Policies and Guidelines
- Path to a Cure (PTAC) - Pilot and Feasibility Award Policies and Guidelines
- Path to a Cure (PTAC) - Postdoctoral Research Fellowship Award Policies and Guidelines
Deadline for all applications: May 11, 2023
Applications must be submitted online at awards.cff.org by 5 p.m. ET.
For More Information
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 (GMCA) office at firstname.lastname@example.org.
Please Direct Inquiries to:
Cystic Fibrosis Foundation
4550 Montgomery Ave.
Suite 1100 N
Bethesda, MD 20814