The UK Cystic Fibrosis Gene Therapy Consortium

Welcome to the UK CFGTC website. Our aim is to develop gene therapy for Cystic Fibrosis (CF).
For more information on Cystic Fibrosis please click here.


The UKCFGTC is based at three of the UK's leading Universities, Edinburgh, Oxford and Imperial College London.

Gene Therapy Multi-Dose Clinical Trial Update.

As you will know we had hoped to announce the outcome of the Multidose Gene Therapy Trial at the upcoming North American CF Conference (NACFC).

The UK CF Gene Therapy Consortium (GTC) only received the first batch of quality-controlled raw data a few days ago, representing perhaps less than half the total information that we will need to look through.

We now have thousands of pieces of data to analyse and interpret, something that is just not plausible to do in a credible way in a few days. The CF community, and the GTC, have spent a considerable time reaching this stage, and it would be unthinkable to rush through this to try and produce a conclusion that may not stand up to later in-depth scrutiny.

We will, therefore, not be presenting at the NACFC and I, with several members of the GTC, will remain in the UK to push forward the data analysis as rapidly as possible so that we can publish the data as soon as possible. We are obviously disappointed by the delay, and will update the website as soon as we have further information.

Professor Eric Alton (On behalf of the GTC), 6 October 2014


The UKCFGTC is based at three of the UK's leading Universities, Edinburgh, Oxford and Imperial College London.

Wave II Update.

The Wave 2 product (CF gene delivered by a virus) that the Consortium has developed in parallel with Wave 1 over the last decade is now progressing rapidly.

We have chosen the final form of the virus that we will take into CF patients, have learnt how to make the quantities that will be needed for the first clinical trial, and have undertaken preliminary safety studies which have not shown any unexpected problems.

The next stage is to embark on a formal toxicology programme, aiming to be in a first clinical trial in 2017.

Professor Eric Alton, February 2014




Research Highlights

Our Clinical Trial Programme

About the Consortium

Site map


Contact Details
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Events & Fundraising
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The CF Trust


Information for Patients on Our Clinical Trial
Latest Clinical Trial News
Contact Details
Patient Identification Centres
Frequently Asked Questions


Consortium Members' Internal Website
About Us
How the Consortium Works
Profiles of the Strategy Group
Community Review
Prof David Porteous OBE
Oxford University Gene Medicine
Centre for Molecular Medicine, Edinburgh
The Roslin Institute
Dep of Gene Therapy, Imperial


Our Research
Optimisation of pDNA
Aerosol Delivery
Viral Vectors Development
Assay Development
Core Facilities
Consortium Patents


Clinical Programme
Clinical Team
Tracking Study
The Run-in Study
Single Dose Clinical Trial
Multi Dose Clinical Trial


Cystic Fibrosis
What is CF?
History of CF
Discovery of the CFTR Gene
What Causes CF?
CFTR Protein Structure
Cystic Fibrosis Links


Gene Therapy
Introduction to CF Gene Therapy
Why use Gene Therapy for CF?
Other CF Gene Therapy Groups
Examples of Successful Gene Therapy
Gene Therapy Links


Publications in Journals
Conference Posters & Presentations
JustGeneTherapy Lecture, Sept 2012
Book Chapters
Open Access Publications
Latest Publications


Medical Futures Innovation Award 2011
Introductory Video
What is a Lentivirus
Production of Lentiviruses
Progress to Date
Photo Gallery