Exciting collaboration on cystic fibrosis gene therapy

Boehringer Ingelheim has announced its collaboration with Oxford BioMedica, Imperial Innovations, and the UK Cystic Fibrosis Gene Therapy Consortium of Imperial College London and the Universities of Oxford and Edinburgh.

Currently, Vertex’s Kalydeco and Orkambi are used to treat the faulty CFTR protein that causes cystic fibrosis, but it can only slow the progress of the disease, and Orkambi is currently only licensed for patients with two copies of the F508del mutation. The new collaboration will work on replacing the faulty mutated gene with a healthy gene inserted directly into the lungs in a lentiviral vector. If they succeed in making an inhalable gene therapy, it could potentially provide a cure for the disease, and BI will have exclusive global rights to develop and commercialise it.

Professor Eric Alton, co-ordinator of the UK Cystic Fibrosis Gene Therapy Consortium at Imperial College, said: “The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems. We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years.”