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.
Bluebird has settled on a clinical trial plan that uses hemoglobin response as a primary endpoint and surrogate marker for reduction in the frequency of sickle “crises.” Also known by the shorthand vaso-occlusive crises, or VOCs, these are the searingly painful and potentially life-threatening exacerbations of the disease that occur when misshapen red blood cells clump inside blood vessels.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
About the Author Reprints
To submit a correction request, please visit our Contact Us page.
