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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.
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.
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.
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.
Once your
Clinical trials are critical to developing new treatments for CF. So, how exactly does an experimental drug become an approved therapy?
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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.