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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 (CF). 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.
In a strong show of support for the CF community, the U.S. Senate Appropriations Committee recently expressed the importance of innovative cystic fibrosis research in a spending bill, which is part of the federal budget under consideration right now in Congress.
The Cystic Fibrosis Foundation has awarded up to $3 million to Kinnear Pharmaceuticals to conduct preclinical testing of a broad-spectrum anti-infective that has the potential to treat multi-drug resistant Pseudomonas and other infections in people with cystic fibrosis.
Corbus Pharmaceuticals reported promising results Thursday from an early stage clinical study of a potential anti-inflammatory drug for people with cystic fibrosis.
Based in Lexington, Mass., the Cystic Fibrosis Foundation Therapeutics Lab conducts groundbreaking CF research to help rapidly advance new therapies.
Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all. By understanding how the protein is made, scientists have been able to develop treatments that target the protein and restore its function.
Two-thirds of people with cystic fibrosis in the U.S. experience debt, food insecurity, or housing issues.
The journey to end cystic fibrosis isn't a straight line. It is an evolving map with many paths and unique challenges. It requires an ambitious research agenda to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure.
Results show that people on Trikafta® enrolled in the six-week study were able to safely stop taking one of the two common CF medications without negatively affecting their health.