This award is intended for exceptional investigators who have made significant, impactful contributions to their field of research. Academic programs supported through the Path to a Cure: Pioneer Award will focus on two core strategies to address the underlying cause of cystic fibrosis:
Restoring CFTR protein
Successfully restoring CFTR protein production when none is present would enable chloride transport in and out cells, increasing fluid at the cell surface, and reducing or thinning the thick, sticky mucus that affects the lungs and other organs in people with CF. Areas of interest include approaches that target premature termination codons (PTC) mutations and other rare mutations — including transfer RNA therapy, readthrough agents, nonsense-mediated decay inhibitors, and short nucleotide therapies — in addition to mutation-agnostic treatments that could help all people with CF, such as messenger RNA therapy.
Targeting the Root Cause of Disease
Genetic-based therapies, which include both gene replacement and gene editing approaches, are considered the most promising strategy to cure CF, as they have the potential to address the root genetic cause of disease. Numerous challenges must be addressed to advance genetic-based therapies to the clinic, including but not limited to the following:
- Identify and target the appropriate cells in affected organs
- Develop new and improved vehicles and vectors with the appropriate biochemical properties to navigate through CF mucus and/or the vasculature
- Selectively and efficiently edit the genome for numerous mutations
- Avoid or mitigate the immune response to enable repetitive dosing
Applicants may propose an amount necessary to complete the proposed research, plus an additional 12 percent of indirect costs for up to three years.
Policies and Guidelines
Please review the 2023 Policies and Guidelines for complete submission information.
Letter of Intent: Rolling
Full Application: Rolling
Applications must be submitted online at awards.cff.org.
For More Information
Those who are interested in any funding programs offered by the Cystic Fibrosis 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 at email@example.com.
Please Direct Inquiries to:
Cystic Fibrosis Foundation
4550 Montgomery Ave.
Suite 1100 N
Bethesda, MD 20814