- Biogen Idec is teaming up with a non-profit organization, Fondazione Telethon, and a research institutution, Ospedale San Raffaele, in Italy to develop gene therapies to address hemophilia A and B.
- More than 10 years ago, Biogen decided to leave gene therapy research because it did not seem to be yielding results; however, new delivery technologies and compelling human clinical trial data have made this Cambridge-based company reconsider—and reinvest.
- The Italian non-profit organization has teamed up with the research institution to form a joint venture, which will receive $5 million upfront to delve into research.
The basic idea that underlies gene therapy is using a modified virus or some other type of vector to deliver healthy genes into a cell, thereby replacing faulty genes. Unfortunately, despite the apparent logic of that theory, ineffective delivery technologies and safety concerns led to billions of wasted R&D dollars. The result: Not one gene therapy technique has been approved by the FDA.
Now some good news: In 2012, UniQure, a gene therapy that treats a rare metabolic disorder, received approval in the EU. In addition, bluebird bio has generated early-stage clinical trial results that are encouraging.
Biogen is refocusing its attention in the field of gene therapy and other therapeutic areas as well, including sickle cell anemia (as part of a partnership with Sangamo Biosciences). Hemophilia is considered a "sensible" target for gene therapy, and a worthwhile investment for Biogen.