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Simon Waddington

University College London UCL

Simon Waddington is Professor of Gene Therapy at UCL. He leads a team developing translational gene therapy for childhood inherited genetic diseases including neurodegenerative diseases, inherited epilepsy and metabolic diseases. He and his team have more than one hundred and sixty peer reviewed publications in journals including Nature, Cell, Nature Medicine, Science Translational Medicine, Nature Immunology, Nature Communications, Cell Metabolism and Blood.


With Professors Manju Kurian, Paul Gissen and Ahad Rahim, he has co-founded a gene therapy company “Bloomsbury Genetic Therapies” to progress four gene therapy programmes to the clinic, namely Ornithine Transcarbamylase Deficiency, Infantile Neuronaxonal Dystrophy, Niemann Pick C and Dopamine Transporter Deficiency Syndrome.

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