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Biogen makes moves in gene therapy

02 February 2015

By Dr Lucy Freem

Appeared in BioNews 788

Biogen Idec has made an investment deal to develop gene therapy for haemophilia A and B.

The biotechnology company is partnering with the San Raffaele-Telethon Institute for Gene Therapies (TIGET) - a joint venture of Italy's Fondazione Telethon and Ospedale San Raffaele - which has already made inroads into the development of a haemophilia B gene therapy. Biogen Idec is investing US $5 million in addition to two years of research support for lentiviral vector-based gene therapies.

'People with haemophilia often require life-long treatment to control dangerous bleeding, but there is hope that gene therapy could one day lead to a single-dose, lasting therapy,' said Olivier Danos, Biogen Idec's senior vice president of gene therapy.

Haemophilia is a rare genetic disease. A mutation in any one of several genes causes impairment in the network of proteins that allow blood to clot, meaning it cannot do so properly. As a result, people with haemophilia experience prolonged bleeding episodes after bruising or minor injury that may cause pain, joint damage and, at their worst, life-threatening haemorrhages.

Lentiviral vector technology uses engineered, partially disabled viruses to insert genes into the genome of a living cell. The technology is well established in the laboratory, and has shown promise in clinical trials for the treatment of some immune and neurodegenerative diseases. In the case of haemophilia treatment, potential vectors will be designed to carry healthy copies of clotting protein genes into the liver cells of haemophilia patients. The hope is that this will allow the patient's body to produce the normal clotting protein, stopping the symptoms of haemophilia.

'We at TIGET have worked for several years to develop a new vector design that upon administration into the blood stringently targets expression of its genetic cargo to the hepatocytes, the main cell type of the liver. This was crucial to establish long-term expression of the therapeutic gene and obtain proof-of-principle of its therapeutic benefit in experimental models of haemophilia B,' said Luigi Naldini, director of TIGET.

'We are now delighted to collaborate with Biogen Idec to expand our hemophilia gene therapy program also to hemophilia A and to advance both programs towards clinical testing', he added.

The most common types of haemophilia, types A and B, are sex-linked and appear more commonly in men than women. Approximately 142,000 people worldwide are currently diagnosed with haemophilia A and approximately 28,000 people are currently diagnosed with haemophilia B worldwide.


16 February 2015 - by Arit Udoh 
Sanofi has signed a partnership deal worth potentially US $845 million with Voyager Therapeutics, a comparatively small biotech firm, making it the latest major pharmaceutical firms to commit financially to gene therapy research...

15 December 2014 - by Sean Byrne 
Pfizer has entered into a deal with biotech company Spark Therapeutics to conduct research into gene therapy for haemophilia B...
01 December 2014 - by Arit Udoh 
Glybera, the first gene therapy to go on sale in Europe, is set to cost €1.1m (£870,000) per patient, making it the world's most expensive drug...
11 November 2013 - by Dr Naqash Raja 
The Children's Hospital of Philadelphia has invested US $50 million in a biotech start-up in an effort to commercialise gene therapy...
19 December 2011 - by Dr Zara Mahmoud 
Scientists have successfully used gene therapy to alleviate the symptoms of the blood disease haemophilia B in six human volunteers, raising hope for a potential cure. The study has been hailed as a landmark trial for gene therapy...
11 July 2011 - by Dr Caroline Hirst 
Scientists have, for the first time, successfully treated a blood disorder by repairing errors in the DNA of a living animal. Researchers from The Children’s Hospital of Philadelphia, together with California-based Sangamo BioSciences, have applied an innovative genome editing technique to treat haemophilia B, which affects around one in 30,000 boys and men...

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