Richmond, CA-based Sangamo and Biogen have struck a deal to co-develop a group of drugs to treat hemoglobinopathies, inherited blood diseases—like beta-thalassemia and sickle cell disease—that come either from the underproduction, or abnormal structure of hemoglobin. Sangamo will get $20 million in cash up front, and stands to receive up to another $300 million in milestone payments if things break right. It'd also get a royalty stream on any marketed drugs to come out of the deal.
Sangamo will handle all the research and development through the first proof of concept trial for a drug candidate in beta-thalassemia. Both companies will team up on the pre-clinical work leading to an investigational new drug application regarding a drug to treat sickle cell disease. Sangamo will then hand off the clinical development lead to Biogen, which will get worldwide rights to the drugs, though Sangamo has an option to co-promote them in the U.S. Biogen will reimburse Sangamo for its internal and external R&D costs related to the programs.
Shares of Sangamo shot up about 15 percent in pre-market trading,
The drugs in the partnership will be built off of Sangamo's approach, which is to use so-called zinc finger proteins to modify genes. Those zinc finger proteins are tied to molecular tools like nucleases and then used to target a specific gene tied to a specific disease, and alter, remove, or replace it.
To date, Sangamo's approach hasn't yet led to a marketed drug. Its most advanced product candidate is an HIV therapy in Phase II clinical trials. The deal is, however, Sangamo's most substantial partnership to date. In 2012, the company teamed up with Shire to research potential treatments for hemophilia A, hemophilia B, and Huntington's Disease. Sangamo got $13 million in cash up front as part of that transaction.
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