Big deal raises big hopes for treating sickle cell disease and Beta-thalassemia

2014 is getting off to a very good start for CIRM-funded companies. Today Sangamo BioSciences announced that it has reached agreement on a global collaboration with Biogen Idec, a move that could speed up work to develop cures for both sickle cell disease and Beta-thalassemia.

This follows hard-on-the-heels of a deal announced earlier this week between Capricor and Janssen Biotech for a stem cell treatment for heart attacks. That approach recently got approval to move into a Phase 2 clinical trial, which we are funding.

In this latest collaboration Biogen Idec will give Sangamo $20 million upfront with the possibility of an additional $300 million if the company’s research hits certain milestones in development, sales and royalties.

Last May Sangamo got a $6.3 million award from CIRM to develop a method of correcting faulty genes that lead to sickle cell disease and beta-thalassemia This approach involves removing bone marrow from people with these conditions, fixing the genetic defect in the blood-forming stem cells., then reintroducing the modified stem cells into the patient where they are expected to produce new healthy red blood cells.

Ellen Feigal, M.D., Senior VP for Research and Development at the stem cell agency said:

“This is great news. It’s great news for the companies involved, for the stem cell field in general and of course for patients whose lives could benefit from this collaboration. It’s also important because it shows how important the funding we provide is in helping companies like Sangamo get their research to a point where big pharmaceutical companies stand up and take notice, and invest.” 

In a news release announcing the collaboration Sangamo’s President and CEO, Edward Lanphier, said:

“This alliance is further validation of our ZFP platform as a transformative technology and accelerates our goal of developing a novel class of therapeutics which has the potential to revolutionize the treatment of genetic diseases.”

He was even nicer in an email to us saying:

“Thank you for ALL of your support over the past several years. We wouldn’t be where we are today without you.” 

The two big deals this week are likely to be hot topics for conversation at next week’s Alliance for Regenerative Medicine’s State of the Industry briefing in San Francisco. That’s a gathering for many of the key players in the regenerative medicine field that focuses on the recent advances in the field and the outlook for the coming year.

And so far, the coming year looks like being a pretty good one for the field.

kevin mccormack