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How many patients will be enrolled in the first human clinical trial for the new lipid nanoparticle-based treatment for cystic fibrosis by end of 2024?
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Clinical trial registries and official announcements
Story
New Lipid Nanoparticles and Prime Editing Correct CFTR F508del for Cystic Fibrosis
Jul 10, 2024, 12:02 PM
Scientists have developed new lipid nanoparticle designs that enable targeted in vivo lung editing in mice, potentially leading to new treatment options for cystic fibrosis. Recent advancements in prime editing, including the systematic application of six recent prime editing developments, have allowed for the efficient and functional correction of the CFTR F508del mutation, the predominant cause of cystic fibrosis, in primary airway cells from CF patients. This breakthrough, reported in Nature Biomedical Engineering, suggests the potential for a one-time prime editing treatment for cystic fibrosis that could be as effective as existing daily therapies.
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