MRC/NIHR Funding Secures UK CFGTC Clinical Trial & Research Programme

We are very pleased today to annouce that the UK CF Gene Therapy Consortium has secured funding to enable both our Multi-Dose GL67A/pGM169 clinical trial and our Wave II Lentiviral Research Programme to continue.

MRC/NIHR Efficacy and Mechanism Evaluation programme: Clinical Trial Funding.

The Consortium trial will be funded by a £3.1 million grant from the National Institute for Health Research (NIHR) and the Medical Research Council (MRC) through the Efficacy and Mechanism Evaluation programme.

Patients will receive the treatment by inhaling aerosolised molecules of the plasmid pGM169 (developed by Oxford Gene Medicine) complexed with the lipid GL67A. Half the participants will receive the real treatment and half a placebo in a double-blind study. Patients aged 12 and over at Royal Brompton Hospital, London and Western General and Royal Hospital for Sick Children in Edinburgh, will receive one dose a month for one year.

Over 30 patients have already received a single dose of the formulation and by delivering multiple doses over the course of a year, we aim to determine whether the therapy can improve symptoms for CF patients.

Dr Deborah Gill, University of Oxford:

"No one has ever done a gene therapy study like this in cystic fibrosis before. It's a worldwide first in terms of the length of the study, the number of patients involved and the number of doses of gene therapy. By giving the therapy over a whole year, we will have the best chance yet of seeing an improvement in patients."

Andrew Lansley, Secretary of State for Health:

"We want to create a world-class NHS where patients benefit from the latest and best treatments. This funding provided by the Government, via the MRC and the NIHR, will enable pioneering studies demonstrating how science in the lab can be translated into gene therapy for patients with cystic fibrosis. This work is another example of the Government's continued commitment to reducing the burden of genetic disease and follows recent recommendations from the Human Genomics Strategy Group, which I have welcomed, which provides a strategic vision for the adoption of genomic technologies across healthcare to diagnose and treat genetic conditions."

David Willets, Minister for Universities and Science:

"This £3.1 million investment will deliver a groundbreaking clinical trial in cystic fibrosis gene therapy, which is an important and promising area of health research. It is an excellent example of collaborative working between Government, the research base and a leading health charity."

Professor Eric Alton Interviewed on BBC Radio 4's Today Programme.

Developmental Pathway Funding Scheme

In addition to the funding for the clinical trial, the Consortium has also been awarded a further £1.2 million to fund the development of our Wave II Lentiviral Research Programme. This grant has been awarded by the MRC DPFS Scheme.

The aim of this research is to use a modified virus to replace the defective CFTR gene in the human airways. Typically this type of viral vector can be designed in such a way to potentially allow far more efficient gene transfer than our existing clinical product, thus reducing the requirement for repeat administrations.

Dr Wendy Ewart, Deputy Chief Executive of the MRC::

"Gene therapy represents one of the most promising treatment avenues for cystic fibrosis and many other devastating conditions that currently have no effective cure. By investing more than £4m in this exciting research, the MRC reaffirms its commitment to driving the translation of laboratory and clinical science into new treatments for human disease. The investment will also strengthen further our significant collaborative links with the NIHR, which make this research possible."

Dr Deborah Gill:

"The new virus delivery approach has never been tried before but it could be more efficient. It is specifically designed to deliver the gene therapy to the lungs, but it will take several more years of development before it gets to the point of human trials."

David Willets:

"The £1.2 million investment from the Medical Research Council to support work in more effective delivery methods will help bring improvements in treatment and benefits for patients."


Other Resources

MRC Press Release

UK Cystic Fibrosis Gene Therapy Consortium Press Release

Oxford University Press Release


Introductory Videos
Medical Futures Innovation Award 2011
Twitter Feed
About Us
Contact Us
Lab Events
Environemental Policy
About this Site

Google Site Search

Site Feedback Form

All Site Images



How the Consortium works/FAQs

Consortium Website

Centre for Molecular Medicine, Edinburgh
The Roslin Institute
Dep of Gene Therapy, Imperial



The Run-in Study

Single Dose Clinical Trial

Multi Dose Clinical Trial


Our Research

Non-viral Vector Development

Aerosol Mediated Gene Delivery

Viral Vector Development

Taqman Core Facility

Cystic Fibrosis

History of CF

Discovery of the CFTR Gene

CFTR Protein Structure

CFTR Function

CF Links


Gene Therapy

Introduction to Gene Therapy

Other CF Gene Therapy Groups

Why use Gene Therapy for CF

Target Cells for CF Gene Therapy

Barriers for CF Gene Therapy

Clinical Trials

Gene Therapy Successes

Gene Therapy Links




Papers in Journals

Conference Posters & Presentations

Book Chapters

D.Phil Theses



Gene Therapy Seminars


Directions & Venue