BI 3720931

BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy developed for the treatment of cystic fibrosis (CF). It is designed to deliver a functional copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene directly into airway epithelial cells via inhalation. This approach aims to restore CFTR function in patients with CF, including those who are not eligible for or do not benefit from existing CFTR modulator therapies. The therapy uses a replication-deficient lentiviral vector and is administered as a single dose by inhaler. The primary goal is long-term improvement in lung function and disease modification regardless of the underlying CFTR mutation type. Development involves Boehringer Ingelheim, Oxford Biomedica (OXB), and the UK Cystic Fibrosis Gene Therapy Consortium[1][2][6][7].

Brand names

Lenticlair

Other names

Cystic fibrosis gene therapy - Boehringer Ingelheim/Oxford BioMedica/the UK Cystic Fibrosis Gene Therapy Consortium