- Drug product vector copy number (DP VCN) and percentage of
lentiviral vector positive cells (LVV+) for initial 7 drug product lots
- Initial results show that the three patients treated to date have achieved in vivo VCN and HbAT87Q production as good as or better than patients achieving transfusion independence in
- First patient treated in
- Safety profile to date consistent with autologous transplantation –
- Company to hold conference call and webcast today,
“Northstar-2 is our first study to utilize our improved LentiGlobin drug
product manufacturing process to increase the drug product vector copy
number and percent of cells transduced. The first patient treated in
this study exemplifies the promise of gene therapy, discontinuing blood
transfusions approximately a month after treatment and achieving a
normal level of total hemoglobin production at six months
“Although early, these data add to the growing body of clinical evidence
that indicate that LentiGlobin may offer a transformative benefit for
patients with TDT,” said
A Phase 3 Study to Evaluate Safety and Efficacy of LentiGlobin Gene
Therapy for Transfusion-Dependent β-thalassemia in Patients with non-β0/β0
|Patient 1||Patient 2||Patient 3|
|DP VCN in each drug product lot (copies/diploid genome)||2.9||2.4||3.2, 2.4|
|LVV+ cells||77%||53%||77%, 82%|
|CD34+ cell dose (x106/kg)||7.0||13.6||8.1|
|HbAT87Q (g/dl; at last follow-up)||9.5||1.6||4.6|
|Total hemoglobin||13.3||Not reported||Not reported|
|Days since last transfusion||140||Not reported||Not reported|
|Follow-up||6 months||3 months||2 months|
- Follow-up on Patients 2 and 3 was not sufficient for total hemoglobin or days since last transfusion to be clinically relevant.
- The safety profile to date appears consistent with autologous transplantation. No Grade 3 or higher drug-product related adverse events have been observed.
bluebird bio will host a live webcast at
The target enrollment of the study is 15 adult and adolescent patients
and 8 pediatric patients. The study’s primary endpoint is the proportion
of treated subjects who meet the definition of "transfusion
independence," defined as total hemoglobin levels of at least 9g/dL
without any red blood cell (
Transfusion-dependent β-thalassemia (TDT), also called β-thalassemia major or Cooley’s anemia, is an inherited blood disease that can be fatal within the first few years of life if not treated.
Despite advances in the supportive conventional management of the disease, which consists of frequent and lifelong blood transfusions and iron chelation therapy, there is still a significant unmet medical need, including the risk for significant morbidity and early mortality. Currently, the only advanced treatment option for TDT is allogeneic hematopoietic stem cell transplant (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft vs. host disease and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.
About bluebird bio, Inc.
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. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia, and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s research, development, manufacturing and regulatory approval plans for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia, and whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with transfusion-dependent ß-thalassemia. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing, planned or expanded clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing process will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU, the risk of a delay in the enrollment of patients in our clinical studies, and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the
Source: bluebird bio, Inc.