Partnership boosts cystic fibrosis therapy bid

Tuesday 7th August 2018, 9:00am

Experts at the University of Edinburgh are teaming up with leading figures from industry to advance development of a gene therapy for cystic fibrosis.


The UK Cystic Fibrosis Gene Therapy Consortium - a collaboration between Imperial College London and the Universities of Oxford and Edinburgh -  will join with Boehringer Ingelheim and Oxford Biomedica to develop a new viral vector-based therapy. 

The partnership builds on pioneering research carried out by the Consortium including clinical trials, which have shown encouraging results.



Cystic fibrosis is an inherited condition caused by mutations in a gene called CFTR. The disease causes thick, sticky mucus to build up in the airways and digestive tract. People affected by the condition are more prone to lung diseases and have a significantly reduced life expectancy.

Gene therapy aims to fix the faulty gene by delivering a functioning version into a patientís lung cells. For the past 17 years, the Gene Therapy Consortium has been working to establish whether gene therapy can become a clinically viable option for patients with cystic fibrosis.

Under the new partnership, researchers will share their expertise to develop an inhaled treatment that can be taken forward into clinical trials. It is hoped that this approach will help people with cystic fibrosis live longer and significantly improve their quality of life.

"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 enable the viral vector system to progress into clinical trials."

Dr Chris Boyd, Gene Therapy Consortium member, Group Leader, MRC Institute of Genetics and Molecular Medicine

"The Gene Therapy Consortium is excited about beginning what we hope will prove to be a productivepartnership with these world class organisations."

Dr Gerry McLachlan, Gene Therapy Consortium member, Group Leader, The Roslin Institute