Inhalable gene therapy for CF
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An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe.
Cystic fibrosis (CF) is caused by defects in the CFTR gene, which causes a buildup of thick sticky mucus in the lungs and digestive system. It causes lung infections and gradually affects the ability to breathe. The new lentiviral vector-based gene therapy works by inserting a functioning copy of the CFTR gene in the DNA of the epithelial cells in a patient’s airway.
At present, some people with CF respond well to relatively new treatments known as CFTR modulator medicines, which work by targeting the related CFTR protein. However, these are not an option for around 10-15% of patients.
The aim of the new treatment being trialled, known as BI 3720931, is to improve lung function and reduce exacerbations (flare-ups, often leading to hospitalisation), for people with CF irrespective of their mutation type – including those who genetically cannot benefit from other CF therapies.
