Lenticlair-ON

Lenticlair-ON (BI 372385) is an investigational lentiviral gene therapy developed by Boehringer Ingelheim in collaboration with Oxford BioMedica and the UK Cystic Fibrosis Gene Therapy Consortium. It is designed for the treatment of cystic fibrosis (CF), a genetic disorder caused by mutations in the CFTR gene. The therapy utilizes a replication-deficient, HIV-1-based lentiviral vector pseudotyped with the fusion (F) and hemagglutinin-neuraminidase (HN) proteins from the Sendai virus to efficiently transduce airway epithelial cells. Delivered via inhalation, Lenticlair-ON aims to provide a functional copy of the CFTR gene to the respiratory tract, thereby restoring chloride channel function. Unlike non-integrating viral vectors, the lentiviral platform allows for integration into the host cell genome, which may offer the potential for long-term therapeutic effect and stable expression of the CFTR protein, potentially reducing the frequency of administration.

Brand names

Lenticlair-ONLenticlair

Other names

BI-372385BI372385BI 372385rHlV-CFTR-F/HN