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Cambridge team secure £14M to develop novel therapy for patients with Haemophilia

02 February 2017
A team of researchers from Cambridge have secured a £14M investment to develop a novel drug therapy that aims to help patients with haemophilia throughout the world.

Dr Trevor Baglin (Cambridge University Hospitals) and Professor Jim Huntington (Cambridge Institute for Medical Research) have received the funding – from Medicxi, Touchstone Innovations and Cambridge Enterprise – into the University of Cambridge spin-out company ApcinteX Limited, to develop a drug they predict will produce normal blood clotting in patients with haemophilia, a genetic disorder that causes uncontrolled bleeding.

Dr Trevor Baglin, honorary consultant haematologist at Cambridge University Hospitals Dr Baglin and Professor Huntington developed the hypothesis based on the clinical observation that patients with severe haemophilia who by chance also inherit a common genetic mutation associated with thrombosis (factor V Leiden) have less severe bleeding. By ‘turning down’ the activity of a natural anticoagulant pathway, it is predicted that the drug will restore the balance between coagulation – the process that clots the blood – and anticoagulation in a person with haemophilia.

A protein that 'turns down' the activity of the natural anticoagulant protein C pathway was engineered in Professor Huntington's laboratory and they were able to show that the clotting capacity of haemophilia blood was returned to normal when they added the novel protein.

Haemophilia is the result of a deficiency in proteins required for normal blood clotting, for example factor VIII in haemophilia A and factor IX in haemophilia B, and affects around 400,000 individuals in the world.

Currently, the standard treatment is administration of the missing clotting factor. However, this requires regular intravenous infusions and is not completely effective. In addition, about one quarter of patients develop an inhibitory antibody to the administered clotting factor which renders further treatment ineffective.

The new drug will not cause anti-clotting antibodies to form and it could be given every few weeks by simple injection under the skin instead of by frequent intravenous infusion. 

Dr Baglin commented: "Bearing in mind that the majority of people in the world with haemophilia have no access to effective therapy, a stable, easily administered, long-acting, drug that can be used in all patients, regardless of the type of haemophilia, could bring treatment to a great deal many more people who suffer from haemophilia."

Notes to editors

Cambridge University Hospitals (CUH)

Cambridge University Hospitals (CUH) is one of the largest and best known hospitals in the country. As well as delivering care through Addenbrooke’s and the Rosie, it is also:

a leading national centre for specialist treatment for rare or complex conditions
a government-designated biomedical research centre
one of only five academic health science centres in the UK
a university teaching hospital with a worldwide reputation
a partner in the development of the Cambridge Biomedical Campus

CUH’s vision is to be one of the best academic healthcare organisations in the world.

Cambridge Enterprise - www.enterprise.cam.ac.uk

A wholly owned subsidiary of the University of Cambridge, Cambridge Enterprise Limited is responsible for the commercialisation of University intellectual property. It provides access to early stage capital through the Cambridge Enterprise Seed Funds, University of Cambridge Enterprise Funds and Cambridge Enterprise Venture Partners, and offers business planning, mentoring and related programmes. Activities include management and licensing of intellectual property and patents, proof of concept funding and support for University staff and research groups wishing to provide expert advice or facilities to public and private sector organisations. For more information, please visit: www.enterprise.cam.ac.uk

Touchstone Innovations – www.touchstoneinnovations.com

Touchstone Innovations plc (formerly Imperial Innovations Group plc or just “Innovations”) creates, builds and invests in pioneering technology companies and licensing opportunities developed from outstanding scientific research from the 'Golden Triangle', the geographical region broadly bounded by London, Cambridge and Oxford.

This area has an unrivalled cluster of outstanding academic research and technology businesses, and is home to four of the world’s top 10 universities, as well as leading research institutions, the cream of the UK’s science and technology businesses and many of its leading investors.

Innovations supports scientists and entrepreneurs in the commercialisation of their ideas through protecting and licensing out intellectual property (through its Technology Transfer subsidiary, Imperial Innovations), by leading the formation of new companies, by recruiting high calibre management teams and by providing investment and encouraging co-investment. Innovations remains an active investor over the life of its portfolio companies, with the majority of Innovations’ investment going into businesses in which it is already a shareholder.

Since becoming a public company in 2006, Innovations has raised more than £440 million of equity from investors, which has enabled it to invest in some of the most exciting spin-outs to come out of UK academic research. In addition, the Group has agreed £80.0 million in loan facilities from the European Investment Bank (EIB).

Between Innovations’ admission to AIM (August 2006) and 31 July 2016, Innovations has invested a total of £306.7 million across its portfolio companies, which have collectively raised investment of £1.5 billion.

Medixci - www.medicxi.com

Medicxi is a life sciences focused venture capital firm, recently spun out of Index Ventures. One of Europe's largest dedicated life sciences VC firms, Medicxi has offices in London, Jersey and Geneva. Past and existing portfolio companies include for example GenMab (Copenhagen: GEN.CO), PanGenetics (acquired by Abbott), Minerva Neurosciences (NasdaqNERV), Egalet (NasdaqEGLT), Molecular Partners (Swiss: MOLN. SW), Versartis (NasdaqVSAR), Levicept, XO1 (acquired by J&J), MorphogenIX, and Gadeta

For more information on our investments, please visit us at Medicxi and connect with us on Twitter and LinkedIn