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.
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.
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.
Information for Patients on Our Clinical Trial
Consortium Members' Internal Website
Medical Futures Innovation Award 2011