-- Two abstracts accepted for presentation
-- Data to be presented from the Northstar Study and from the HGB-205 Study
-- Company to host investor call on
“We are encouraged by the data generated so far from the ongoing
LentiGlobin phase 1/2 studies. In the HGB-205 trial, the two subjects
with beta-thalassemia previously presented at the 19th Annual
“In the Northstar Study, at the time of the abstract submission, one subject had received LentiGlobin BB305 Drug Product. In this subject we have observed a monthly increase in beta-T87Q-globin levels following infusion, reaching 1.8g/dL at month 3, and we are encouraged by the trajectory of the beta-T87Q-globin increase to date. At ASH, we plan to provide additional follow-up data on this subject, and early data on at least two additional subjects treated in the Northstar Study, including a subject with the beta0/beta0 genotype of beta-thalassemia,” continued Dr. Davidson.
The accepted abstracts are listed below and are now available online on
the ASH conference web site: www.hematology.org/Annual-Meeting.
Information contained in the abstracts reflect data available as of
Title: Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral BetaA-T87Q-Globin Vector (LentiGlobin BB305 Drug Product). A. Thompson, M.D. et al
Session Name: 801. Gene Therapy and Transfer I
Oral Presentation Time:
Location: Moscone Center,
Title: Study HGB-205: Outcomes of Gene Therapy for Hemoglobinopathies via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral BetaA-T87Q-Globin Vector (Lentiglobin BB305 Drug Product). Marina Cavazzana, M.D. Ph.D et al
Session Name: 801. Gene Therapy and Transfer: Poster III
Location: Moscone Center,
Investor Conference Call and Webcast Information
bluebird bio will host a conference call and webcast on
The phase 1/2 study is designed to evaluate the feasibility, safety and efficacy of LentiGlobin (BB305) drug product in the treatment of subjects with beta-thalassemia major. The study is designed to enroll up to fifteen subjects. Subjects will be evaluated for safety and efficacy post-transplant.
For more information on the Northstar Study, please visit www.northstarstudy.com or clinicaltrials.gov using identifier NCT01745120.
About the HGB-205 Study
The phase 1/2 study is designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of subjects with beta-thalassemia major and severe sickle cell disease. The study is designed to enroll up to seven subjects. Subjects will be followed to evaluate safety and transfusion requirements post-transplant. In sickle cell disease patients only, efficacy will also be measured based on the number of vaso-occlusive crises or acute chest syndrome events.
For more information on the HGB-205 Study, please visit clinicaltrials.gov using identifier NCT02151526.
About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases. bluebird bio has two clinical-stage programs in development. The most advanced product candidate, Lenti-D, is in a recently-initiated phase 2/3 study, the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder affecting young boys. The next most advanced product candidate, LentiGlobin, is currently in two phase 1/2 studies, one in the US (the Northstar Study) and one in
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in collaboration with
bluebird bio has operations in
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the potential efficacy and safety of the Company’s LentiGlobin BB305 product candidate, the Company’s plans with respect to LentiGlobin and its other product candidates and anticipated clinical and business milestones and announcements. In addition, it should be noted that the data for LentiGlobin announced from the
Availability of other information about bluebird bio
Investors and others should note that we communicate with our investors and the public using our company website (www.bluebirdbio.com), our investor relations website (http://www.bluebirdbio.com/investor-splash.html), including but not limited to investor presentations and FAQs,
Source: bluebird bio, Inc.
bluebird bio, Inc
Richard E. T. Smith, Ph.D., 339-499-9382
Pure Communications, Inc.
Dan Budwick, 973-271-6085