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.
Scancell initiates SCOPE trial's iSCIB1+ cohort
Bio-Thera Solutions' BAT8006 phase II Study receives US FDA IND approval
Poseida Therapeutics names new chief medical officer
Biocytogen and ABL Bio collaborate on development of new bispecific antibody-drug conjugates
BioVaxys Technology announces non-brokered private placement
Senhwa Biosciences doses first subject in phase II study of Silmitasertib
Antennova completes first dosing cohort in Phase one study of ATN-031
M8 Pharmaceuticals collaborates with SERB Pharmaceuticals
BerGenBio ASA starts Phase 2a portion of BGBC016 clinical study of bemcentinib
Phanes Therapeutics receives FDA Fast Track designation for PT886