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Research announcement – January 2017

We are pleased to bring you the latest update to the research into a potential treatment for Tay-Sachs and Sandhoff disease being undertaken at the University of Cambridge and Addenbrooke’s Hospital.

Since our last update, the team have sought hard to understand the problems that they encountered with the human-type vectors which were supplied to administer the healthy genes. Although this has taken a long time to complete, we are delighted to announce that they have resolved the issues, and now work with a different manufacturing team and one much more experienced with making vectors used in clinical trials. The group has been awarded a grant extension by the Medical Research Council to continue with the project in Cambridge and thankfully, with all the uncertainty, the team there is very much a team – and still ‘in there’!

The main question everyone will have is: what does this mean for us? Unfortunately, the necessary re-testing of new vectors imposed a delay to the work before it could move on to the second milestone in the project, which is getting ‘Regulatory Approval’ for the vectors and the design of the clinical trial. However, now that clinical testing for the working vector is not only nearing completion, but has also been successful we are planning on approaching the various regulatory bodies in early 2017. Why is this important? Well, without their support and backing the project will not be able to continue to the next stage which is starting a clinical trial in children with Tay-Sachs and Sandhoff disease. There is real hope that if everything goes to plan over the coming months we would be in a position to start the third milestone of the project which is recruitment in 2018.

The members of the European Tay-Sachs & Sandhoff Disease Charity Consortium (ETSCC)

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