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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