There have never been as many new CF drugs in development as there are today. In the second plenary at NACFC, Dr. George Retsch-Bogart outlined the progress we've made, the road ahead and the changes needed to make it all possible. Read on for my key takeaways.
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The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.
The Cystic Fibrosis Foundation awarded nearly $700,000 to Calibr, the drug discovery and development arm of Scripps Research, to screen for a compound that could be used to treat Burkholderia cepacia complex infections.
As Congress weighs budget appropriations, we look at long-term funding for the National Institutes of Health. We decided to sit down with the Foundation's senior vice president of research affairs to learn more about why the work being done at NIH is so important in the search for a cure for CF and other serious, rare diseases.
The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.
Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
The Cystic Fibrosis Foundation awarded Talee Bio Inc. up to $4.5 million to develop methods for delivering potential gene therapy treatments to the lungs.