Boehringer halts inhaled gene therapy cystic fibrosis trial

Boehringer Ingelheim has stopped a phase 1/2 first-in-human trial for an inhaled lentiviral vector-based gene therapy in adults with cystic fibrosis who are ineligible for CFTR modulators.

The LENTICLAIR 1 trial started early last year as part of an ongoing collaboration between Boehringer, the UK Respiratory Gene Therapy Consortium and Oxford Biomedica. The therapy uses Oxford’s proprietary lentiviral vector manufacturing technology. According to ClinicalTrial.gov, the study — which had enrolled five participants — has been stopped early and will not start again. Participants are no longer being examined or treated. A Boehringer spokesperson told media outlets that the trial did not produce the clinical data needed to support the continued development of BI 3720931.

It was hoped that BI 3720931 could potentially address unmet needs in cystic fibrosis by inserting a functional copy of the CFTR gene in the DNA of airway epithelial cells. The aim was to improve lung function and reduce exacerbations for people with CF irrespective of their mutations, including those who genetically could not benefit from CFTR modulators. While CFTR modulators have provided treatment options for some patients, there remains a significant unmet need for 10-15% of people with CF due to mutation type or modulator intolerance.