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Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
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The Cystic Fibrosis Foundation is aggressively pursuing potential treatments for people with CF who have nonsense and rare mutations who will not benefit from drugs known as modulators, which correct the malfunctioning cystic fibrosis transmembrane conductance regulator (
Through efficient study design, optimized clinical trial execution, and high-quality data, the Therapeutics Development Network (TDN) helps speed the delivery of new and better therapies to people with cystic fibrosis.
Our corporate champions are an important part of our progress — with corporate supporters by our side, we continue to advance our goal — to make CF stand for Cure Found.
Some genetic diseases, such as cystic fibrosis, are caused by mutations in a single gene. A gene contains DNA “letters” that spell out the instructions to make a specific protein. When the protein isn't made correctly, it can lead to a cascade of problems.
Working alongside the CF community, the Foundation has fostered the development of more than a dozen CF treatments — an unprecedented number in a short span of time — and helped add decades of life for people with CF. And while therapies such as Trikafta® have had an incredible impact on the lives of those with CF, there are still many people with this disease who do not benefit from existing treatments. Our goal is to cure cystic fibrosis.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.