Biogen Idec forges a $320M gene editing deal aimed at curing inherited blood disorders

Shares of Sangamo BioSciences picked up steam early this morning with the news that Biogen Idec ($BIIB) is anteing up $20 million and promising up to $300 million more in milestones to partner with the biotech on new therapies for two inherited blood disorders.

Biogen R&D chief Douglas Williams

The deal brings a major league player to the genome editing game, which has been gaining new attention with some significant new investments in the field. In this particular partnership Richmond, CA-based Sangamo will use its zinc finger nuclease platform to work up new therapies for sickle cell disease and beta-thalassemia.

In this instance, the technology can be used to address "the abnormal structure or underproduction of hemoglobin," either by knocking out a key regulator of gene expression or inserting a corrective gene to substitute for the defective one causing the disease.

By midmorning, shares of Sangamo ($SGMO) jumped 25%. Sangamo is responsible for early R&D work and is hanging on to U.S. co-promotion rights for both indications.

"Our collaboration with Sangamo is expected to help us expand our capabilities to develop treatments for people with serious, inherited hematologic conditions," said Biogen R&D chief Douglas Williams in a statement. "Building upon emerging science related to fetal hemoglobin regulation, we intend to develop Sangamo's novel gene-editing technology to create a single approach that has the potential to functionally cure both sickle cell disease and beta-thalassemia."

Biogen Idec doesn't do a lot of deals, but under CEO George Scangos the company has remained committed to staying on the cutting edge of some particular fields in R&D. The recent approval of Tecfidera and some recent deals have helped it build a reputation as one of the leaders in multiple sclerosis. 

- here's the release

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