As someone who does not qualify for Trikafta®, I took particular interest in the first plenary of the North American Cystic Fibrosis Conference, which discussed potential treatments for people with nonsense and rare mutations. I learned that researchers are exploring a variety of options, and some early research has shown promising results.
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The Cystic Fibrosis Foundation is providing up to $5 million to develop a method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF that is unlikely to trigger an immune system response.
Carbon Biosciences is the first company to publicly launch from the Foundation’s collaboration with Longwood Fund and the first to work alongside CF Foundation researchers at the Foundation’s therapeutics lab
Clarametyx’s novel therapy aims to disrupt bacterial biofilms, one of the primary causes of antibiotic resistance, thereby potentially increasing the effectiveness of existing treatments in fighting a wide range of bacterial infections, including those commonly affecting people with CF.
Prime Medicine’s prime editing technology has the potential to bring genetic therapies to all people with cystic fibrosis, ultimately paving the way to a cure.
The funding will help support research into the development of potential new modulator therapies for people with cystic fibrosis with an F508del mutation.
Funding will support a Phase 2b trial of phage therapy for chronic Pseudomonas aeruginosa lung infections.
Since 2011, the Foundation has awarded $2.5 million across 35 different projects to improve cystic fibrosis newborn screening across the country.