Deal to aid development of cystic fibrosis gene therapy
Researchers and commercial partners agree licensing to advance work on treatment for life-threatening lung condition.
Development of a potential treatment for cystic fibrosis, a rare, progressive, life-threatening disease that affects 70,000 people worldwide, is to be supported in a collaborative agreement.
The UK Respiratory Gene Therapy Consortium (GTC), led by Imperial College London, includes scientists from the University of Edinburgh's Institute of Genetics and Cancer and the Roslin Institute among the partners in a licensing agreement with pharmaceutical company Boehringer Ingelheim.
The academic organisations in the collaboration – Imperial College London, University of Oxford and University of Edinburgh – have agreed to license intellectual property and know-how towards development of the gene therapy product.
Under the arrangement, which follows more than two decades of research within the GTC, Boehringer Ingelheim will support development of the potential therapy through clinical trials in people, before it potentially becomes available for use in patients.
The gene therapy under development is designed for inhalation, and uses a modified virus to deliver a healthy version of a gene known as CFTR into the relevant cells in patients. This can correct for defective or absent protein resulting from mutations in the CFTR gene, which causes cystic fibrosis.
University of Edinburgh Principal Investigators Dr Chris Boyd and Dr Gerry McLachlan have been GTC members since its inception and serve on its Strategy Group. Both were involved in the pre-clinical work culminating in the trial of a non-viral gene therapy formulation for cystic fibrosis. Their efforts since then have been focused on contributing to the development of the lentivirus-based vector system. Dr Boyd leads the Gene Therapy Group in the University’s Centre for Genomic and Experimental Medicine, which has investigated the distribution of lentiviral insertion sites in lung-derived cells.
The decision by Boehringer Ingelheim to exercise intellectual property options concerning the GTC's lentiviral vector for CF gene therapy is a significant milestone which will greatly facilitate the product's further development. It is a vindication of how successful the collaboration between Imperial, Oxford and Edinburgh has been in its ability to work together towards this translational goal for many years. We look forward to continuing to work alongside BI and Oxford Biomedica as we progress towards clinical studies.
Boehringer Ingelheim announcement (external)