New Partnership to Develop Gene Therapy for Cystic Fibrosis
Centre for Genomic and Experimental medicine researchers are teaming up with leading figures from industry to advance development of a gene therapy for cystic fibrosis: August 2018
Cystic fibrosis (CF) is an inherited condition caused by mutations in the CFTR gene, causing a build-up of thick, sticky mucus in the lungs and digestive tracks. People affected by the condition are more prone to lung diseases and have a significantly reduced life expectancy (see https://www.nhs.uk/conditions/cystic-fibrosis/).
The UK CF Gene Therapy Consortium (GTC) brings together researchers from the University of Edinburgh, Imperial College London and the University of Oxford. At the University of Edinburgh, the consortium’s principal investigators are Chris Boyd at the Centre for Genomic and Experimental Medicine within the MRC Institute of Genetics and Molecular Medicine and Gerry McLachlan at The Roslin Institute.
The aim of this new partnership is to establish whether gene therapy can become a clinically viable option for patients with CF. This form of treatment needs new copies of the CFTR gene to be introduced into the cells lining the lung, which is hard to achieve because these cells have evolved to keep external molecules out. The CFTR gene has to be carried past these defences, and one way to do this involves inserting the gene inside a viral vector - a virus which has been engineered to deliver genes efficiently into lung cells.
The GTC have been working for over a decade with a Japanese biotechnology company (DNAVEC, now called ID Pharma), and have developed a viral vector carrying CFTR to a stage where it can now undergo formal pre-clinical testing and larger-scale manufacturing. To further this, the GTC is joining forces with two world class organisations in a major collaboration; Boehringer Ingelheim - an internationall research-driven pharmaceutical company with substantial expertise in bringing products through to patients, and Oxford BioMedica who are the acknowledged leaders in the field of manufacturing this type of virus.
This partnership brings together the expertise and support needed to facilitate our aim of realising viral vector-based CF gene therapy. We are delighted that we are now in a position to carry out the crucial pre-clinical work that is required to potentially enable the viral vector system to progress into clinical trials. My team has been studying the pattern of genomic integration of the viral vector. It is important that we have a good understanding of this phenomenon in lung cells before proceeding to the clinic, so this work will be taken forward in larger scale studies by the partnership as part of its preclinical programme.
This partnership provides the potential 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. The collaboration has the greatest chance of realising a new therapeutic pathway for CF patients that will add to the improvements already being seen with small molecule treatments.
The Gene Therapy Consortium is excited about beginning what we hope will prove to be a productive partnership with these world class organisations.
The project has so far benefited from support from Just Gene Therapy, Flutterby FUNdraisers, the MRC Developmental Pathway Funding Scheme and the Cystic Fibrosis Trust. The GTC has also recently received funding from the Health Innovation Challenge Fund, a collaboration between the Wellcome Trust and the Department of Health and Social Care, to undertake the next steps.