The results from our Multidose Gene Therapy Clinical Trial were presented on Saturday 30 May at a special event for the CF community. To ensure that as many people from the community were able to see the announcement, and to overcome the barrier of cross-infection, the presentation was live-streamed for people to watch at an event in Edinburgh as well as over the internet.
This was arranged because we were keen that the CF community were the first people to hear the results. The community has generously supported this trial for many years, and we are pleased that we were able to provide this preview.
The results have been submitted to a medical journal and are currently undergoing peer review. Papers under submission are subject to a media embargo before they are published, and therefore we are unable to share the results publically again at present.
As is usual practice for scientific research, we will announce the results as soon as they are published in full in a journal.
The event on 30 May was recorded, and we will make this recording available as soon as we can.
We know that this delay is frustrating, and we thank you for your patience.
27 days 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.
7 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