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Bluebird bio Presents New Data for LentiGlobin Gene Therapy for Sickle Cell Disease at 24th European Hematology Association Congress.

M2 PHARMA-June 18, 2019-Bluebird bio Presents New Data for LentiGlobin Gene Therapy for Sickle Cell Disease at 24th European Hematology Association Congress

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- US-based bluebird bio, Inc. (NASDAQ: BLUE) has presented new data from patients in Group C of its ongoing Phase 1/2 HGB-206 study of the company's investigational LentiGlobin gene therapy for sickle cell disease at the 24th European Hematology Association Congress in Amsterdam, the Netherlands, the company said.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the ?-globin gene that leads to the production of abnormal sickle hemoglobin, causing red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events.

For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complications, such as acute chest syndrome, stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD adds functional copies of a modified form of the ?-globin gene (?A-T87Q-globin gene) into a patient's own hematopoietic (blood) stem cells. Once patients have the ?A-T87Q-globin gene, they have the potential to make functional RBCs, with the goal of reducing sickled RBCs, hemolysis, and other complications.

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD that includes three treatment cohorts: Groups A, B and C. As of March 7, 2019, 25 patients were enrolled and a total of 13 patients had been treated with LentiGlobin in Group C, with a median post-treatment follow-up of nine months (1.0 15.2 months).

Eight of the 13 treated patients in Group C had at least six months of follow-up at the time of the data cutoff. In these patients, production of gene therapy-derived hemoglobin (HbAT87Q) ranged from 4.5-8.8 g/dL and total unsupported hemoglobin levels ranged from 10.2-15.0 g/dL at the last study visit.

The median concentration of HbAT87Q continued to increase, accounting for >=50 % of total Hb in patients with at least 12 months of follow up.

No ACS or serious vaso-occlusive crisis was reported in patients in Group C at up to 15 months post-treatment with LentiGlobin. In an exploratory analysis, key markers of hemolysis, including reticulocyte counts, lactate dehydrogenase and total bilirubin concentration, trended toward normal levels.

As of the data cutoff date, the safety data from all patients in HGB-206 are reflective of underlying SCD, the known side effects of hematopoietic stem cell collection and myeloablative conditioning.

There have been no serious adverse events related to LentiGlobin for SCD. One mild, non-serious event of hot flush was reported that the investigator considered to be related to LentiGlobin for SCD; it occurred and resolved on the day of drug product infusion and did not require treatment.

Established tools, including high-performance liquid chromatography, are used to measure the amount of HbAT87Q in a blood sample. In order to detect HbAT87Q and HbS protein expression at a cellular level, bluebird bio has utilised a new, exploratory assay to demonstrate the pancellular expression of HbAT87Q in patients treated with LentiGlobin.

The assay enables detection of HbAT87Q and HbS protein expression at a cellular level. Results from this assay showed that in samples from five patients who were at least nine months post-treatment, on average, at least 70 % of each patient's RBCs expressed HbAT87Q.

LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bio's clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The US Food and Drug Administration granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we're developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully.

Beyond our labs, we're working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. We're putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent ?-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

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Publication:M2 Pharma
Date:Jun 18, 2019
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