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.

The Wave 1 Clinical Trial (CF gene delivered by liposomes) is going well. We were able to recruit all the patients needed from CF Centres across the UK.

Approximately half the patients have received all the 12 doses of gene therapy or placebo given at monthly intervals. The remaining will be completing their dosing over the next few months, with the final dose in the final patient due around June.

There will then be a period of data analysis, and we hope to present the findings at the North American CF Conference in October.

There have been 3 safety reviews by an independent external committee, with no unexpected problems noted to date. The trial is double-blind so we, of course, have no knowledge about any efficacy. The project was awarded by the Efficacy and Mechanism Evaluation (EME) Programme and is funded and managed by the National Institute for Health Research (NIHR) on behalf of the MRC-NIHR partnership.

Professor Eric Alton, February 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

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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
Milestones
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
GL67A/pGM169
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

 

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

 

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