Boehringer Ingelheim forms pact to develop gene therapy for cystic fibrosis

Boehringer Ingelheim announced Monday a collaboration with Oxford BioMedica and the UK Cystic Fibrosis Gene Therapy Consortium (GTC) to develop a gene therapy for patients with cystic fibrosis. The parties will focus on the use of a replication-deficient lentiviral vector in an inhaled formulation to introduce a healthy copy of the CFTR gene into the cells of the lung.

The partnership aims to leverage Oxford BioMedica's experience in manufacturing lentiviral vector-based therapies and Boehringer Ingelheim's capabilities in drug discovery and the clinical development of therapeutic agents. As part of the collaboration, Boehringer Ingelheim has received an option to license the exclusive global rights to develop, manufacture, register and commercialise the lentiviral vector-based gene therapy. During the option period, Boehringer Ingelheim will finance all work, although further financial terms of the agreement were not disclosed.

The GTC, which comprises Imperial College London and the Universities of Oxford and Edinburgh, has undertaken a Phase IIb study of gene therapy for cystic fibrosis, which met its main goal. Results reported in 2015 showed that patients who received the gene therapy had a significant, if modest, benefit in lung function compared with those administered placebo.

According to the parties, the approach developed by the GTC has "demonstrated high gene transfer efficiency and offers the possibility of repeated administration to maintain the therapeutic effect." Eric Alton, coordinator of the GTC, said the collaboration "provides [cystic fibrosis] patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status."

Boehringer Ingelheim indicated that the partnership is part of its Respiratory Therapeutic Area and Research Beyond Borders (RBB), which is one of the pillars of the drugmaker's R&D strategy.

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