SAN DIEGO — Bluebird Bio (BLUE) presented data Wednesday that for the first time links its gene therapy for sickle cell disease to improved clinical outcomes for patients. It’s part of a plan to speed up a regulatory filing for the one-time treatment called LentiGlobin.

In many ways, Bluebird is following the successful sickle cell disease regulatory playbook written by Global Blood Therapeutics (GBT). Both companies are seeking to take advantage of flexibility at the Food and Drug Administration and a desire to see new sickle cell treatments reach the market.

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  • Adam I know you don’t care about investors’ daily predicaments but can me and my Gernies please get a Gern update? I read all your past posts to better understand the risks associated with the drug but I’m still a believer that this belongs in a discussion of competing with Luscpatercept and Rituximab for the MDS market. And you have to admit that their drug’s mechanism is at least interesting in its more general applications to other blood diseases should they be able to commercialize an MDS drug.


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