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Development of gene therapy gives hope to cystic fibrosis community

Project contact
Dr Sarah Trewick
Business Development Manager College of Medicine and Veterinary Medicine Institute of Genetics and Cancer, Usher Institute

Cystic fibrosis (CF) is a common genetic disease affecting around 70,000 people worldwide. It is caused by a defective or absent CFTR protein that results from mutations in the CFTR gene. This causes the build-up of thick sticky mucus in the lungs which become repeatedly infected and inflamed, leading to respiratory failure.

While the median age of those who died from cystic fibrosis in 2019 was 31, half of those living with cystic fibrosis today are expected to live into their forties, thanks to earlier diagnosis and ongoing developments in care and treatments.

In 2001, research groups from Imperial College London and the Universities of Oxford and Edinburgh, including the CF Gene Therapy Group in the Edinburgh Centre for Genomic and Experimental Medicine, formed UK Cystic Fibrosis Gene Therapy Consortium (GTC) to share expertise and funding with a common goal of making gene therapy for CF patients a clinical reality.

In 2018, the German biopharmaceutical company Boehringer Ingelheim formed a partnership with the GTC, the IP Group and Oxford Biomedica. Then in 2021, Boehringer Ingelheim exercised options arising from an agreement facilitated by the GTC and the IP Group to develop a new lentiviral vector-based gene therapy for patients with cystic fibrosis.

The lentiviral gene therapy product is made of virus particles modified to contain neither infectious material nor having replicative capacity. Instead, the virus particles only use the virus' ability to get into cells and to release a gene therapy construct which reconstitutes the function of the damaged CFTR gene in transduced airway cells.

University of Edinburgh researchers Dr Chris Boyd and Dr Gerry McLachlan are members of the GTC and serve on its Strategy Group.

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.

Dr McLachlan leads a group in the Roslin Institute, which has focused on evaluating the delivery and efficacy of the viral vector system in large animal models.

Both were also involved in the pre-clinical work culminating in the successful Phase IIb trial of a previous non-viral gene therapy formulation for cystic fibrosis performed before Boehringer Ingelheim joined the program. Phase IIb trials are specifically designed to assess how well a therapy works. Their efforts since then have focused on contributing to the development of the lentivirus-based vector system.

Find out how you can work with the University’s world-class inflammation expertise and collaborate on further research into treatments for people with cystic fibrosis.

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