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Rare Disease Collaboration: Biogen Idec And Hemophilia Gene Therapy

January 30, 2015



On January 29th, Biogen Idec and San Raffaele – Telethon Institute for Gene Therapy (TIGET) announce a global collaboration to develop gene therapies for Hemophilia A and B. Biogen Idec will contribute their understanding of hematology with TIGET’s expertise in creating new gene therapies.  

Biogen Idec, an international biotechnology company founded in 1978, is developing therapies for neurodegenerative diseases, hematologic conditions, and autoimmune disorders.  The company currently has 2 FDA approved orphan drugs for the treatment of Hemophilia A and B.

Biogen Idec’s Approved FDA ODD Products For Hemophilia

Row Num Generic Name/ Trade Name FDA ODD Approval Date Indication
1 Antihemophilic factor (recombinant), Fc fusion protein / Eloctate 06.06.14 Hemophilia A
2 Coagulation factor IX (recombinant), Fc fusion protein/  Alprolix 03.28.14 Hemophilia B


The collaboration takes advantage of TIGET’s lentiviral gene therapy technology:

“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.”

Please reference the Press Release for the terms of the collaboration.

Hemophilia B, which affects approximately 4,000 people in the United States and 28,000 worldwide, is an inherited sex-linked blood clotting disorder affecting mostly males. It is caused by defects in the Factor IX gene. Hemophilia A, the most common type of Hemophilia:

•   Occurs in approximately 1 in 5,000 male births

•   Effects approximately 16,000 patients in the US

•   Effects approximately 142,000 patients worldwide.

Other players in the Hemophilia Gene Therapy field are:

•   Gene therapy start-up (2013) Dimension Therapeutics is developing novel Adeno-Associated Virus (AAV) gene therapy (reference Blog Post)

•   Gene therapy start-up (2013) Spark Therapeutics is collaborating with Pfizer to develop AAV gene therapy for Hemophilia B (reference Blog Post)

•   uniQure is developing AAV gene therapy for Hemophilia B.

Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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