Accelerating the Discovery of Nonsense Mutation Therapies for CF

Funding Agency:
Cystic Fibrosis Foundation

To better understand the obstacles, limitations, and challenges of developing a permanent cure for cystic fibrosis, the Cystic Fibrosis Foundation held a workshop in January 2019 focused on identifying the scientific gaps and challenges to develop a therapy for nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. As an outcome of the workshop, the CF Foundation is pleased to announce a request for applications (RFA) to identify and support highly meritorious proposals that will accelerate the discovery of novel therapies for nonsense mutations.

To bring new technologies and expertise to the CF field, investigators without experience in CF research are encouraged to apply.

There are two program options:

  • Research Grant: Up to $250,000 per year plus 12% indirect costs for up to two years
  • Pilot and Feasibility Award: Up to $75,000 per year plus 12% indirect costs for up to two years

Deadline: Oct. 24, 2019

Agency Website

Areas of Interest

Successful proposals will focus on strategies, tools, technologies, and model systems that will advance the development of nonsense mutation-directed therapies for CF. These include, but are not limited to, studies aimed at the following:

  • Improving our understanding of the pathways that regulate translation termination, which includes identifying, characterizing, and validating potential targets to promote nonsense mutation suppression
  • Evaluating the potential impact of CFTR messenger RNA (mRNA) sequence variability or cell type on the relative efficacy of a nonsense mutation therapy
  • Improving the definition of the nonsense-mediated decay (NMD) pathway and the mechanism that destabilizes CFTR mRNAs that carry premature termination codons (PTCs)
  • Evaluating the impact on efficacy and safety of inhibiting NMD globally and in a CFTR mRNA-specific manner
  • Understanding the mechanisms governing readthrough agents, antisense oligonucleotides, and other candidate therapeutics to overcome NMD and PTC mutations
  • Developing and optimizing approaches to up-regulate CFTR mRNA synthesis
  • Improving translation of novel therapeutic strategies by developing in vitro reporter systems with enhanced capabilities to monitor PTC readthrough and/or NMD inhibition
  • Developing and characterizing new in vivo reporter models that can evaluate longitudinal efficacy of a potential readthrough agent or NMD inhibitor

Eligibility Requirements

U.S. residents and applicants from outside the U.S. are welcome to apply. International applicants and institutions are required to submit additional information in accordance with U.S. anti-terrorism restrictions.

Funding Type



Junior Faculty


Medical - Basic Science
Medical - Clinical Science
Medical - Translational

External Deadline

October 24, 2019