BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced $1.7 million in new research funding to drive progress on its Path to a Cure. The awards, to seven academic institutions and two companies, will support focused research into key scientific challenges associated with developing therapies to address the underlying cause of disease for all people with CF, regardless of their underlying mutation.
“There has been an explosion of scientific progress in novel technologies with the potential to benefit all people with CF, yet significant additional research will be required to move these advances out of the lab and safely to patients,” said William Skach, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “These awards reflect important investments into the foundational research that is needed to advance curative therapies for all people with cystic fibrosis.”
These research awards, which encompass preclinical and laboratory research, include:
- Up to $343,000 to Splice Bio to advance a platform capable of modifying gene therapy delivery vehicles, called vectors, to better target specific cells in the lungs of people with CF. Splice Bio will collaborate with the Cystic Fibrosis Foundation Therapeutics Lab on this research.
- Funding to Rewrite Therapeutics to demonstrate preclinical proof-of-concept for a genome editing treatment for CF.
- Dr. Adam Feinberg, Carnegie Mellon University: Further develop and test a platform capable of overcoming the innate immune defenses in the lungs to successfully deliver a potential new genetic-based therapy to the airway epithelium, the tissue that lines the airways.
- Dr. Tom Harris, University of Alabama at Birmingham: Explore a novel approach to modulate CFTR expression, increasing abundance of CFTR mRNA which could boost the efficacy of potential nonsense mutation readthrough therapies, as well as existing therapies.
- Dr. Tamas Hegedus, Office for Research Groups Attached to Universities and Other Institutions, Eötvös Loránd Research Network and Dr. John Hunt, Columbia University: Studies to deepen current understanding of both the mechanism of action of ivacaftor (Kalydeco®) and the structure and function of cystic fibrosis transmembrane conduction regulator (CFTR) protein, respectively, with the goal of identifying new therapeutic targets and improving future therapies.
- Dr. Darrell Kotton, Boston University School of Medicine/Boston Medical Center and Dr. George Lukacs, McGill University: Grants to develop scientific tools, including a new method to generate specialized lung cells that express high levels of CFTR in the airways and improved models to study extremely rare CFTR mutations.
Previously in 2020, the Foundation funded six research grants and five pilot and feasibility studies focused specifically on nonsense mutations.
About the Path to a Cure
The CF Foundation launched its $500 million Path to a Cure initiative in October 2019. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF. Each approach requires a different set of scientific tools and knowledge, leading the Foundation to bring together researchers and industry leaders from a range of disciplines to advance multiple areas of research in parallel. Innovators who are interested in pursuing programs in cystic fibrosis can learn about our specific funding opportunities on cff.org.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.