CAMBRIDGE, Mass.--(BUSINESS WIRE)--
bluebird bio, Inc. (BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today released initial positive clinical data from its HGB-205 clinical study of its LentiGlobin BB305 product candidate in beta-thalassemia major subjects at the 19th Annual Congress of the European Hematology Association (EHA) in Milan, Italy.
“We are gratified that the improvements we introduced into the BB305 lentiviral vector design and manufacturing process appear to have translated into clinical results that we believe support the potential for our LentiGlobin BB305 gene therapy to transform the lives of patients with beta-thalassemia major,” stated David Davidson, M.D., bluebird bio’s Chief Medical Officer. “We are encouraged by the early and high-level production of corrected betaAT87Q-globin and the rapid onset of transfusion independence in these initial subjects, as well as the absence of any gene therapy related adverse events. We look forward to providing additional data from this study and our ongoing multi-center Northstar Study later this year.”
The principal investigator of the HGB-205 Study, Marina Cavazzana, M.D delivered an oral presentation at the EHA Congress entitled “Improving gene therapy for beta-thalassemia major: initial results from Study HGB-205” on June 14, 2014 at 04:15 pm CET (10:15 am EDT). The presentation included data from the prior LG001 Clinical Study and the ongoing HGB-205 Study.
Summary of the clinical data presented at EHA were:
LG001 Clinical Study
Clinical update provided on two subjects treated in the prior LG001 Study (subjects 3 and 4) using the prior lentiviral HPV569 product candidate
Subject 3 remains blood transfusion independent 72 months after being transplanted with the lentiviral HPV569 product candidate
Subjects 3 and 4 are producing 2.7 g/dL and 0.4 g/dL of therapeutic betaA-T87Q-globin post-transplant, respectively
No drug product related adverse events were reported in the LG001 Study.
HGB-205 Clinical Study
Clinical data were presented on two subjects (subjects 1 and 2), both with beta-thalassemia major and the Beta E/Beta 0 genotype who were treated using the new lentiviral vector BB305
At 4.5 months following autologous transplant subject 1 had a total hemoglobin of 10.1 g/dL of which 6.6 g/dL was therapeutic betaAT87Q-globin, and at 2 months post-transplant subject 2 had a total hemoglobin of 11.6 g/dL of which 4.2 g/dL was betaAT87Q-globin
Subjects 1 and 2 received their last blood transfusion on day 10 and 12, respectively, post-transplant and both subjects remain blood transfusion independent
Vector copy number in the drug product for subjects 1 and 2 were 1.5 and 2.1, respectively; multiple times higher than the drug product vector copy numbers reported in the prior LG001 Study (VCN 0.6 and 0.3 for Subjects 3 and 4, respectively)
No drug product related adverse events were reported, and the integration site analysis performed on subject 1 at the 3-month time point showed polyclonal reconstitution.
We anticipate reporting additional data from the HGB-205 Study and from our ongoing Northstar Study in late 2014. |
