The Consortium are pleased to announce the publication of the results from our multi dose gene therapy clinical trial in Lancet Respiratory Medicine.
One hundred and thirty six patients aged 12 and above were randomly assigned to either 5ml of nebulised pGM169/GL67A (gene therapy) or saline (placebo) at monthly intervals over 1 year. Lung function was evaluated using a common clinical measure FEV1.
The clinical trial reached its primary endpoint with patients who received therapy having a significant, if modest benefit in lung function compared with those receiving a placebo. After a year of treatment, in the 62 patients who received the gene therapy, FEV1 was 3.7% greater compared to placebo.
The trial is the first ever to show that repeated doses of a gene therapy can have a meaningful effect on the disease and change the lung function of patients.
More details here.
3 months ago
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.
11 months ago
The Consortium groups are based in three of the leading UK universities. Prior to the formation of the Consortium each group had conducted at least one clinical trial for gene therapy. Therefore, our researchers have been at the forefront of this field for over 20 years.Learn More
Our research is focussed on the development of gene therapies for CF. Taking research ideas from the lab to the clinic is beyond the reach of most academic groups. The Consortium is different as we have aligned our different groups to focus on their key talents to ensure that we continue to progress.Learn More
Since 2004 UKCFGTC members have published over 100 papers in peer reviewed journals and have presented many posters and presentations at international conferences. Where possible we will provide pdfs of publications. However, most of our conference posters and presentations are available.Learn More