- First patient with severe sickle cell disease treated with gene therapy remains free of clinical symptoms 21 months after receiving LentiGlobin Drug Product -
- Ongoing transfusion independence and sustained production of HbAT87Q in patients with transfusion-dependent β-thalassemia -
- Company to host event at ASH with live webcast,
The data from the HGB-205 study were highlighted today in a poster
presentation by Marina Cavazzana, M.D., Ph.D., lead investigator of the
HGB-205 study conducted in
“We believe the enduring responses seen in this study - in the patients
with TDT as well as the patient with SCD - demonstrate the continued
promise of LentiGlobin gene therapy in both of these patient
populations. We have seen nearly three years of transfusion independence
in TDT in certain patients, providing important data on the long-term
safety and durability of this therapy,” said
Abstract #2311: Update from the HGB-205 Phase 1/2 Clinical Study of LentiGlobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies
HGB-205 is an ongoing, open-label, single-center Phase 1/2 study
designed to evaluate the safety and efficacy of LentiGlobin drug product
in the treatment of patients with TDT and severe SCD. Four patients with
TDT and one patient with severe SCD have undergone infusion with
LentiGlobin drug product in this study as of
Key Results as of
- Three patients with TDT and β0/βE genotype have remained free of transfusions since shortly after receiving LentiGlobin treatment. Patient 1201 has been free of transfusions for 33.1 months with total hemoglobin of 10.9 g/dL, of which 7.7 g/dL was HbAT87Q. Patient 1202 has been free of transfusions for 29.9 months with total hemoglobin of 13.5 g/dL, of which 10.1 g/dL was HbAT87Q. In addition, this patient has been able to stop iron chelation. Patient 1206 has been free of transfusions for 11.5 months with total hemoglobin of 11.3 g/dL, of which 8.6 g/dL was HbAT87Q.
- Patient 1203 with TDT and homozygousity for the severe β+ mutation IVS1-110 has been free of transfusions for 11.6 months (since approximately 3 months after receiving LentiGlobin treatment) with total hemoglobin of 8.3 g/dL, of which 6.7 g/dL was HbAT87Q.
- Patient 1204 with severe SCD at 21-month post-drug infusion was producing 48% HbAT87Q – well above the 30 percent threshold of anti-sickling Hb that may potentially achieve a disease-modifying clinical effect.
Prior to drug product infusion, Patient 1204 required monthly blood
transfusions after failure of hydroxyurea treatment to help control
his SCD symptoms; he has not received
RBCtransfusions since shortly after LentiGlobin infusion. Since infusion, this patient has had no hospitalizations or acute SCD-related events.
- No LentiGlobin-related adverse events have been observed for the patients with either TDT or SCD; the adverse events observed are generally consistent with myeloablative conditioning.
- All five treated patients successfully engrafted and insertional site analyses demonstrate highly polyclonal reconstitution without clonal dominance.
“These data show a stable clinical and biological effect in patients with TDT or severe SCD who have received a one-time treatment with LentiGlobin,” said Professor Cavazzana. “We are now seeing the benefit of gene therapy with LentiGlobin beyond two years in TDT in certain patients, and clinical benefit continues to be realized in the patient with severe SCD after almost 24 months of follow-up. We are encouraged by these results and the potential benefit treatment with LentiGlobin can have on patients living with these debilitating diseases and without an HLA compatible sibling donor.”
bluebird bio will host a live webcast at
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™ BB305 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 megaTALs/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 severe sickle cell disease.
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, risks that the preliminary positive results from our prior and
ongoing clinical trials of LentiGlobin, including HGB-205, will not
continue or be repeated in our ongoing or planned clinical trials of
LentiGlobin, the risks that the changes we have made in the LentiGlobin
manufacturing process or the HGB-206 clinical trial protocol 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 quarterly report on Form 10-Q, as well as discussions of
potential risks, uncertainties, and other important factors in our
subsequent filings with the
About AP-HP: AP-HP -
About the Imagine Institute: As the leading
European center for research, care and teaching in genetic diseases, the
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
bluebird bio, Inc.
Elizabeth Pingpank, 617-914-8736
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