-- 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
Oral Presentation:
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
Abstract: 549
Session Name: 801. Gene Therapy and Transfer I
Date:
Session Time:
Oral Presentation Time:
Location: Moscone Center,
Poster Presentation:
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
Abstract: 4797
Session Name: 801. Gene Therapy and Transfer: Poster III
Date:
Session Time:
Location: Moscone Center,
Investor Conference Call and Webcast Information
bluebird
bio will host a conference call and webcast on
About the
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
Forward-Looking Statements
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.
Investor Relations:
bluebird bio, Inc
Richard E. T.
Smith, Ph.D., 339-499-9382
or
Media Contact:
Pure
Communications, Inc.
Dan Budwick, 973-271-6085