Research announcement – November 2018

The latest update on the proposed clinical trial for GM2 Gangliosides (Tay-Sachs and Sandhoff disease) is below.

The UK gene therapy team at the University of Cambridge is making progress with plans for a clinical trial safely to test the effects of the selected rAAV-based gene transfer system for GM2 gangliosidosis (Tay-Sachs & Sandhoff diseases) in young babies and children.

Funding has been secured for this new stage of development and progression in the laboratory continues – also with the full support of the UK Medical Research Council. However, there are currently difficulties worldwide in the making of effective rAAV vectors that can be proved to be completely safe for human use. As with other colleagues in the field, we have experienced great disappointment due to unwanted effects that would be dangerous in humans and proved to be disastrous in non-human primates as well as other test systems. The costs of manufacturing clinical grade gene therapy vectors have also greatly increased.

With increasing regulatory requirements for the safe development of gene therapy medicinal products and other biologically active substances, it is likely that there will be an additional year of pre-clinical validation experiments required before the trial can move forward into human clinical trials. It is expected that, should progress be made on the lines predicted and the final regulatory approval be granted, the trial will begin recruiting sometime in 2021.

We realise that this extended period places a huge burden of hope and frustration on everyone in the community but we will update the European Tay-Sachs and Sandhoff Charity Consortium (ETSCC) members and share all the news as the programme development continues and as would be expected as part of our joint engagement.

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