UCL Cancer Institute


UCL scientist receives outstanding achievement award

10 November 2016

The European Society for Gene and Cell Therapy (ESGCT) have awarded Professor Amit Nathwani, Founder and Chief Scientific Officer of Freeline Therapeutics and Professor of Haematology at UCL, with the 2016 Outstanding Achievement Award for his pioneering work on gene therapy.

Professor Amit Nathwani…

The ESGCT seeks to honour one eminent scientist each year who has made pioneering research success and a significant and lasting contribution to the advancement of the field. The winner is competitively selected on the basis of quality and impact of their research career. Professor Nathwani was the first to show successful correction of bleeding diathesis in patients with severe haemophilia B using a distinct gene transfer approach and he has continued this leading work on gene therapy for patients with haemophilia and other bleeding disorders. 

As well as being the Founder and CSO of Freeline and Professor of Haematology at University College London (UCL) Cancer Institute, Professor Nathwani is the Director of the Katharine Dormandy Haemophilia Centre at the Royal Free Hospital, Senior NIHR Investigator and Consultant Haematologist at UCLH.

Professor Nathwani started his pioneering work on gene therapy for haemophilia B in 1997 with Dr Arthur Nienhuis at St Jude Children’s Research Hospital. In 2001 he returned to University College London as a Senior Lecturer in Haematology and a Consultant to the National Blood Services in the UK. Professor Nathwani developed a cutting-edge gene therapy programme for inherited bleeding disorders using adeno associated viral (AAV) vectors, encompassing basic vector design, scale-up and purification of novel AAV serotypes and conduct of preclinical studies to support proof-of-concept Phase I/II clinical trials. In 2010, together with collaborators at St Jude Children’s Research Hospital, Professor Nathwani conducted the first haemophilia B phase I/II trial to clearly demonstrate the durable potential of gene therapy to treat haemophilia and other bleeding disorders. The results of the study, published in the New England Journal of Medicine, demonstrated that all ten treated haemophilia B patients showed safe, sustained expression of blood clotting Factor IX from a single treatment.

“I am honoured to receive such a prestigious award and it serves as recognition of our research in this field of haemophilia gene therapy over the past 19 years. It is an exciting time in gene therapy, where we are seeing potentially life-changing therapies for patients. I want to thank all my colleagues and collaborators who have worked with me over the years and I look forward to continued advances in the field.” says Professor Nathwani.

Dr Jan Thirkettle, Chief Executive Officer of Freeline, says: “Over the years Amit and his team at UCL has amassed a profound record of scientific achievement, innovation, and pioneering work using a distinct gene transfer approach for patients with haemophilia and other bleeding disorders. I am delighted that he has been selected to receive the Outstanding Achievement Award from the EGCST; an honour that Amit richly deserves.”

Freeline Therapeutics (Freeline), is a biopharmaceutical company focused on the development of curative gene therapies for bleeding and other debilitating disorders.

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