We wanted to update you on the Multidose Gene Therapy Trial. This was a complex trial requiring extensive data analysis, but we now have the results, which have been submitted for peer review.
We wanted to share our findings with the CF community as soon as possible, so in collaboration with the CF Trust, we will be holding an open meeting to present the data and address questions.
This will be held in London on Saturday May 30th (more details), and will be live-streamed for those unable to attend; we are also looking into the possibility of a simultaneous conference link to Edinburgh. For cross-infection reasons, we request that CF patients do not attend the venues.
To allow us to book the appropriately sized venues, it would be very helpful if those wishing to attend in person could indicate London or Edinburgh by following the hyperlink to the CF Trust website and filling in your details.
Further details of the venue and exact timing will be posted shortly.
Shortly after the meeting, we will let all trial participants know whether they received the active gene therapy or the placebo and to provide contact details for any questions arising.
Many thanks once again to all of you for your invaluable help!
Best wishes, Eric Alton on behalf of the UK CF Gene Therapy Consortium
14 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.
6 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