Biogen Partners to Develop Gene Therapies for Hemophilia

Biogen Idec is teaming up with Fondazione Telethon and Ospedale San Raffaele to develop gene therapies for hemophilia A and B.

The worldwide collaboration will combine San Raffaele-Telethon Institute for Gene Therapy’s (TIGET) extensive expertise in creating new gene therapy strategies and developing them from bench to bedside with Biogen’s deep understanding of hematology to potentially treat the underlying cause of hemophilia A and B.

Under the collaboration, TIGET will receive a $5 million upfront payment for the rights to the programs and their development. Additionally, TIGET is eligible to receive undisclosed milestone payments subject to the achievement of certain development goals. Biogen will be responsible for the research costs for both the hemophilia A program and the hemophilia B program, and will have the option to exercise worldwide development and commercialization rights for each program following initial clinical proof of concept trials.

TIGET has developed a lentiviral gene transfer vector, engineered to deliver versions of defective genes responsible for causing certain diseases into the patients’ cells. The approach has shown promise in studies 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 TIGET’s strategy for treating hemophilia, the lentiviral vector is administered directly into the body and targets liver cells. If safe and effective, this approach could provide long-term, stable therapeutic benefit in the future for hemophilia patients.

“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 hemophilia B. 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, always keeping in mind the safety and benefit of the patients,” said Luigi Naldini, director of TIGET.

Over the last few years, gene therapy has become a popular field. In 2012, UniQure won European approval of the first gene therapy treatment for lipoprotein lipase deficiency, a rare metabolic disorder. Bluebird Bio, which partnered with Celgene, provided promising early results using gene therapy for beta-thalassemia. Biogen’s return to gene therapy started last January when it struck a deal with Sangamo Biosciences. In September, Biogen said that the deal with Sangamo was the first in a broad effort in gene therapy.

“This alliance confirms again how the excellent scientific research we are funding can lead to exciting new therapeutic approaches,” said the general manager of Telethon, Francesca Pasinelli. “We can say that we have pioneered a model whereby the charity organization acts not only as a funding agency, but plays a primary role in managing the development of research to ensure that each step of the process leads to the ultimate goal, which is to provide accessible therapy to patients.”

Source: Biogen Idec

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