BETHESDA, Md. -- Today the Cystic Fibrosis Foundation announced an investment of up to $14 million in 4D Molecular Therapeutics (4DMT) to develop a customized vehicle to deliver a healthy cystic fibrosis transmembrane conductance regulator (CFTR) gene into the lung cells of people with cystic fibrosis (CF). The delivery of genetic-based therapies is one of the key hurdles to developing an effective therapy for the more than 1,700 different mutations that cause CF, including nonsense and rare mutations.
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 approaches, including gene therapy and gene editing, is critical to finding a cure.
“Today's announcement reaffirms our commitment to invest aggressively to draw the best scientific minds and technology into CF and deliver the next generation of transformative therapies for people with CF,” said Michael P. Boyle, MD, President and Chief Executive Officer. “Despite decades of progress in gene therapy, delivery to the lung remains a key challenge and one we are committed to solving.”
4DMT's gene delivery vehicle, known as 4D-710, is a customized adeno-associated virus (AAV) vector designed to deliver a healthy CFTR gene specifically to cells in the lungs of people with CF. The vehicle was selected by 4DMT from their proprietary library of over one billion different AAVs based on its particular ability to target cells in the lung. One of the challenges that needs to be addressed is countering the lungs' aggressive protection against foreign intruders -- including vehicles designed to deliver a healthy CFTR gene.
With this funding, 4DMT will explore whether an inhaled version of 4D-710 will restore CFTR protein levels to improve lung function. This includes developing manufacturing processes and assessing the safety and tolerability of this potential new CF treatment. If successful, 4DMT plans to advance 4D-710 into early stage clinical trials for CF.
The Foundation previously awarded 4DMT more than $3 million in funding to expand its preclinical development of viral gene delivery in the lung.
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, driving progress toward our goal to make CF stand for Cure Found. Innovators who are interested in pursuing programs in cystic fibrosis can learn about our specific funding opportunities here.
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.