Policy & Regulation
Bluebird bio Presents Updated Data of LentiGlobin Gene Therapy in Patients with Severe Sickle Cell Disease
21 June 2018 - - Cambridge, Massachusetts-based gene therapies specialist bluebird bio, Inc. (NASDAQ: BLUE) has discussed new interim data from the ongoing HGB-206 Phase 1 multicenter clinical study of LentiGlobin investigational gene therapy in patients with severe sickle cell disease (SCD) at the Congress of the European Hematology Association, the company said.
The study was divided into groups A, B, and C. Group A was treated under the original study protocol. Group B was treated under an amended study protocol that included a refined drug product (DP) manufacturing process intended to increase DP vector copy number as well as changes to improve engraftment of gene-modified stem cells.
Both group A and B had DP made from stem cells collected using bone marrow harvest.
Group C was also treated under the amended study protocol, but received LentiGlobin gene therapy made from stem cells collected from peripheral blood after mobilization with plerixafor rather than via bone marrow harvest.
In group C, all patients with ≥ 3 months follow-up were consistently producing ≥ 30% anti-sickling HbAT87Q. The first group C patient was generating a normal total hemoglobin of 14.2 g/dL with over 60% anti-sickling HbAT87Qat 6 months.
With its lentiviral-based gene therapies, T cell immunotherapy expertise, and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer.
The company has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina; and Zug, Switzerland.
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