02 February 2015
ByAppeared in BioNews 788
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.