MAKANDA – Blue Sky Vineyard will have food, music and auctions to raise awareness and funds for Tay-Sachs, a genetic neurological disease, from 10 a.m. to 7 p.m. Saturday. This will be the fourth year the winery will host the Tay-Sachs Benefit Concert in honor of 4-year-old Elise Rochman. To...
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CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded based on the review of all available data that the benefit-risk balance of medicinal products containing ZYNTEGLO™ (betibeglogene autotemcel gene therapy) remains favorable. As of today, bluebird bio has...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for sickle cell disease (SCD) gene therapy (bb1111) for adult and pediatric patients with SCD,...
CAMBRIDGE, Mass. – bluebird bio, Inc. (NASDAQ: BLUE) today announced the company has completed the tax-free spin-off of its oncology programs and portfolio into 2seventy bio, Inc., an independent, publicly-traded company. bluebird bio will continue its work focused on severe genetic disease, with three near-term opportunities to bring transformative gene...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including updated results from the pivotal Phase 2/3 Starbeam study (ALD-102) and the long-term follow-up study LTF-304, as well...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) announced today business and program updates across its severe genetic disease portfolio including a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111), the company’s decision to withdraw...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) announced today that based on the analyses completed to date, it is very unlikely the Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) reported in its Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for sickle cell disease (SCD) (bb1111)...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen...
CAMBRIDGE, Mass. – bluebird bio, Inc. (NASDAQ:BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2020 and shared recent operational progress. “For over ten years, bluebird bio has been pioneering gene therapies for patients with rare diseases and cancer, and our...