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. With Professors Manju Kurian, Paul Gissen and Ahad Rahim, he has co-founded a gene therapy company “Bloomsbury Genetic Therapies” which is progressing four gene therapy programmes to the clinic. They are gene therapy for Ornithine Transcarbamylase Deficiency, Infantile Neuronaxonal Dystrophy, Niemann Pick C and Dopamine Transporter Deficiency Syndrome.
He and his team have received funding from the UK Medical Research Council Developmental Pathway Funding Scheme and LifeArc for translational projects to treat Dravet Syndrome and Dopamine Transporter Deficiency Syndrome. Collaborating with Prof Tristan McKay (Manchester Metropolitan University) he obtained a European Research Council starter grant of €1.5 million from the European Research Council to develop the means to quantify signalling pathways in diseased organs and tumours continually and non-invasively. Working alongside the teams of Professors Andy Baker (Edinburgh), Stu Nickin (Glasgow), John McVey (University of Surrey) and Alan Parker (Cardiff) he elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells. He has several long-standing international collaborations including Drs Jerry Chan (Duke-NUS Graduate Medical School Singapore and Prof Patrick Arbuthnot (University of Witwatersrand, South Africa) where he holds an honorary chair.
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.