Freeline Therapeutics’ Founder and Chief Scientific Officer is honoured by the European Society for Gene and Cell Therapy with the 2016 Outstanding Achievement Award

21st Oct 2016 By admin

London, 21 October 2016 – Freeline Therapeutics (“Freeline”), a biopharmaceutical company focused on the development of curative gene therapies for bleeding and other debilitating disorders, today announced that The European Society for Gene and Cell Therapy (EGCST) has recognised the Company’s Founder and Chief Scientific Officer (CSO), Professor Amit Nathwani, with the Outstanding Achievement Award.

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 pioneering work on gene therapy for patients with haemophilia and other bleeding disorders.  He will present a plenary lecture at the ESGCT Annual Meeting at 5pm on October 21 2016.

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

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.

Dr Jan Thirkettle, Chief Executive Officer of Freeline, said:

“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.”


Professor Amit Nathwani, Chief Scientific Officer of Freeline, added:

“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.”

Freeline, based at the UCL Medical School, Royal Free Hospital, London, was launched in December 2015 with a £25 million Series A financing from Syncona LLP. The Company’s next-generation AAV gene therapy platform, developed by Professor Amit Nathwani, Founder and Chief Scientific Officer of Freeline as well as Professor of Haematology at University College London, builds on the successful haemophilia B phase I/II trial conducted by UCL in collaboration with St. Jude Children’s Research Hospital.

– Ends –

Further information:

Freeline Therapeutics

Susan Hill, Chief Business Officer


+44 (0)20 7611 2031
JW Communications

Julia Wilson

+44 (0) 781 8430877



Notes for Editors:


About Freeline Therapeutics

Freeline Therapeutics is a biopharmaceutical company based at the Royal Free Hospital, London, focused on the development of gene therapies for bleeding and other disorders. Gene therapy has the potential to transform lives for people with severe diseases by providing a long-lasting, safe and reliable source of enzymes to the blood. The Company’s human adapted AAV vector platform is being utilised to advance therapies through the clinic and its shareholders include Syncona LLP and University College London.  For further information please visit the Company’s website at:


About Syncona

Syncona LLP was founded in 2012 and operates as a healthcare investment company, taking an active role in identifying, developing and funding technologies with the potential to significantly impact the healthcare market of the future.  Syncona can take the long view when necessary, able to concentrate investment into opportunities as technology is validated. Syncona is a subsidiary of the Wellcome Trust. For further information please visit the Company’s website at:


About UCL (University College London)

Founded in 1826, UCL was the first English university established after Oxford and Cambridge, the first to admit students regardless of race, class, religion or gender, and the first to provide systematic teaching of law, architecture and medicine. We are among the world’s top universities, as reflected by performance in a range of international rankings and tables. UCL currently has over 35,000 students from 150 countries and more than 11,000 staff. Our annual income is more than £1 billion.


About Haemophilia B

Haemophilia B, also called Factor IX (FIX) deficiency, is a genetic disorder caused by missing or defective Factor IX that can result in dangerously low levels of the essential clotting protein. The World Federation of Haemophilia identified over 28,000 males suffering worldwide from haemophilia B in their Annual Global Survey 2013. For those with severe disease marked by FIX levels of less than one percent of normal, scrapes and bumps are medical emergencies. Painful episodes of spontaneous bleeding can result in crippling joint damage early in life and an increased risk of potentially fatal bleeding within the brain.

Current treatment involves replacement therapy where concentrates of clotting factor are administered intravenously on an on-demand or prophylactic basis. This preventive therapy is usually initiated in patients at a young age and may need to be continued for life. It is hampered by the frequency of administration of up to 3 times per week as well as the high associated costs.

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