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Research announcement – February 2018

We are very excited to share the latest update on the proposed clinical trial for GM2 Gangliosides (Tay-Sachs and Sandhoff disease).

At the end of last year, The University of Cambridge research team along with Daniel Lewi from The Cure & Action for Tay-Sachs (CATS) Foundation met with the European Medicines Agency (EMA) to seek European designation as an Orphan Medicinal Product.

Orphan Medicinal Products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union. The EU introduced new legislation in 2000 with the aim of providing incentives for the development of medicines for rare diseases (so-called orphan medicinal products).

We are delighted to announce that we were granted European designation as an Orphan Medicinal Product by the EMA. In addition, the medicine and procedure being developed by Professor Cox and his team has been recognised as meeting the criteria as an Advanced Therapy Medicinal Product (ATMP) by the EMA.

These are both significant milestones which have been achieved and means we are on schedule to start our clinical trial for GM2 Gangliosides at the end of 2018 / start of 2019.

The work has been further enhanced by being given Orphan Drug Designation by the FDA in the United States. This shows that although the clinical trial being planned by the team at the University of Cambridge will initially focus on European patients, it has the potential to have a massive impact on the wider Tay-Sachs and Sandhoff community.

The next steps are vital. Over the coming months we will begin planning how the trial will be implemented and we will start deciding on both the outcome goals we want to measure and the recruitment criteria.

We hope you join us in being excited by this news. We understand that for some families this treatment will come too late. As the founders of The CATS Foundation our daughter Amelie sadly lost her battle to Tay-Sachs at the age of 8 in December 2017. Like many of you, we are dedicated to this fight and we will not stop until there is a treatment available for all the children affected by Tay-Sachs and Sandhoff disease which gives our children a brighter future.

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

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