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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.
Pseudomonas aeruginosa are strains of bacteria that are widely found in the environment. Pseudomonas is a major cause of lung infections in people with cystic fibrosis. The bacteria thrive in moist environments and equipment, such as humidifiers and catheters in hospital wards, and in kitchens, bathrooms, pools, hot tubs and sinks.
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.
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.
The Drug Development Pipeline features an extensive list of drugs that are in development or approved for treating cystic fibrosis. For a drug or program to be shown on the pipeline, it must meet certain conditions.
This competition provides one year of free lab and office space at Bakar Labs at the University of California Berkeley to awardees with emerging genetic medicines delivery technology that may be applied to CF.
Theratyping matches therapies, or medications, to specific types of mutations. The primary goal of theratyping is to identify which mutations respond to certain CFTR modulators, thereby helping people with rare CFTR mutations gain access to already approved modulators quickly and safely.