Biogen Idec, Fondazione Telethon and Ospedale San Raffaele have entered into a worldwide collaboration to jointly develop gene therapies for the treatment of both hemophilia A and B.
The agreement will combine San Raffaele - Telethon Institute for Gene Therapy’s (TIGET) extensive expertise in creating new gene therapy strategies and developing them from the bench to bedside with Biogen Idec’s deep understanding of hematology to potentially treat the underlying causes of hemophilia A and B.
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TIGET will receive an upfront payment of $5 million for the rights to the programs and their development, and is eligible to receive further payments subject to the achievement of certain development milestones.
In addition, Biogen Idec will support the research costs for the two programs; one in hemophilia A and one in hemophilia B. Biogen Idec will have the option to exercise worldwide development and commercialization rights for each program following the initial clinical proof of concept trials.
The collaboration centers on TIGET’s advanced lentiviral gene transfer technology.
Lentiviral vectors are engineered viruses used to deliver working versions of defective genes responsible for causing certain diseases into the patients’ cells.
This approach has shown promise in clinical trials for the treatment of some immune-hematologic and neurodegenerative diseases by gene transfer into hematopoietic stem cells harvested from the patients, treated with the vector and then re-infused into the body.
In the TIGET strategy for treating hemophilia, the lentiviral vector is directly administered into the body and targets liver cells.
If proved safe and effective, this approach may in the future provide long-term, stable therapeutic benefit in people affected by hemophilia.
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