Update from Professor Eric Alton - September 2018

Over the last few months, the UK Cystic Fibrosis Gene Therapy Consortium (GTC), which includes the NHLI (Eric Alton, Jane Davies, Uta Griesenbach and Tracy Harman), and the Universities of Oxford and Edinburgh, have made progress in a number of areas.

The GTC has built on experience gained with a non-viral approach to transferring genes into the airways, successfully used in a major Phase 2B cystic fibrosis trial which met its primary outcome, to develop a new viral vector-based product. The latter recently received £2.75M funding from the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) for translation into the clinic.

To catalyse this translation the GTC has now joined forces with two world class organisations in a major collaboration. The partnership will include Boehringer Ingelheim, an internationally renowned ‘big pharma’ company with substantial expertise in bringing products through to patients in the respiratory field, and Oxford BioMedica who are the acknowledged leaders in the field of manufacturing the viral vector. The three partners are coming together to translate this product into clinical trials, and if successful, into routine clinical practice. The GTC believes that this partnership provides cystic fibrosis (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. Imperial College London's technology commercialisation company, Imperial Innovations, is acting as the lead on the technology transfer for the partnership.

In parallel the GTC were successful in a £6.4M bid for a Wellcome Portfolio award focused on using the lentiviral vector as a platform technology for diseases other than CF. Five initial disease targets have been identified including α1-antitrypsin deficiency, surfactant protein B deficiency, interstitial lung diseases, haemophilias and pandemic flu. The GTC has also gained funding to assess the use of the lentivirus platform for the treatment of alveolar proteinosis (with Cliff Morgan at the Brompton) and thrombotic thrombocytopaenic purpura (with Tom McKinnon and Mark Islan at Imperial).

Finally, Imperial (Eric Alton and Uta Griesenbach) were successful in a £5M Higher Education Innovation Fund bid, partnered with UCL and King’s College London, to increase collaboration across London in the field of Advanced Therapy Medicinal Products (ATMP’s, i.e. gene and cell therapies). The aim is to reduce duplication and competition across the capital and to enhance further the already significant portfolio of ATMP studies being delivered both with industry and academically.