BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced that it awarded funding to enGene Inc. to develop a customized vehicle to deliver genetic-based therapies, such as gene therapy and gene editing, into the lung cells of people with cystic fibrosis. Delivering genetic-based therapies to the lungs is a key hurdle to developing effective treatments for all people with CF, including individuals with two nonsense and rare mutations.
“Today's announcement reflects yet another milestone on our path to delivering transformative therapies for people with CF,” said William Skach, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “This is a very early but important step in advancing a viable way to produce healthy CFTR genes in the lungs.”
This industry award is part of the CF Foundation's $500 million Path to a Cure challenge to accelerate treatments for the underlying cause of CF and develop a cure. Current therapies have significantly improved the lives of many people living with CF, but there are still people who do not have an effective treatment for their CF mutations. Developing a way of delivering genetic-based therapies to the lungs is critical to finding treatments and a cure for all people with CF.
How It Works
enGene is developing a non-viral approach with the potential to deliver a healthy CFTR gene
specifically to the lung cells of people with CF that would allow functional CFTR protein to be made. Traditional gene therapy approaches use harmless viruses, called vectors, to deliver healthy genes into cells. However, viral vectors are particularly susceptible to aggressive defense mechanisms in the lungs that protect against foreign intruders.
enGene is creating small, dense structures of DNA that are combined with their delivery vehicle and coated with molecules to make them more effective at penetrating mucus. The CF Foundation funding will be used to advance preclinical testing of these therapies.
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 the Path to a Cure page 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.