Clinical trials of an inhaled gene therapy for cystic fibrosis have 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 gene, the defect underlying the disease, carried on a lentiviral vector designed to deliver it to airway epithelial cells. LENTICLAIR 1, a Phase I open-label trial and a Phase II placebo-controlled trial, are planned to evaluate the drug's safety and efficacy. Ten clinical centers in the United Kingdom, Spain, Italy, the Netherlands, and France will conduct the trial.
The study, led by Eric Alton of Imperial College London, is expected to involve approximately 36 adult patients with any genetic variant of cystic fibrosis. Each patient will receive one of three doses of the drug to determine the optimal dose for further testing. The 24-week LENTICLAIR 1 trial is expected to end in April 2027, after which long-term follow-up will continue as part of the LENTICLAIR-ON trial.
