Clinical trials of inhaled gene therapy for cystic fibrosis have been launched in European countries, according to a press release from Imperial College London. The experimental drug BI 3720931, developed by Boehringer Ingelheim, is a working version of the CFTR protein gene, the defect of which underlies the disease, on a lentiviral vector that should deliver it to the epithelial cells of the respiratory tract. It is planned that the LENTICLAIR 1 trials of the first open and second placebo-controlled phases, during which the safety and efficacy of the drug will be assessed, will be conducted in ten clinical centers in the UK, Spain, Italy, the Netherlands and France.
The trial, led by Eric Alton of Imperial College London, is expected to involve around 36 adult patients with any genetic variant of cystic fibrosis. Each will receive one of three doses of the drug to determine which is best for further testing. The 24-week LENTICLAIR 1 trial is expected to end in April 2027, after which long-term follow-up will be continued in the LENTICLAIR-ON trial.