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)...
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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...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell...
SAN MATEO, Calif. — Bluejay Therapeutics, Inc., a private clinical-stage biopharmaceutical company focused on viral and liver diseases, today announced the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to BJT-778 for the treatment of Chronic Hepatitis Delta Virus (HDV) infection. BJT-778 is a high-potency, fully human immunoglobulin...
NEW YORK – Quest Diagnostics subsidiary Blueprint Genetics said on Thursday that it has partnered with the Helsinki Biobank and Helsinki University Hospital for a research initiative called Rare3k that aims to develop algorithms to accelerate the identification of patients who could benefit from genetic testing for certain rare diseases....
Cambridge, Mass. — Blueprint Medicines Corporation (Nasdaq: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, announced that, effective April 1, 2023, the Compensation Committee of Blueprint Medicines’ Board of Directors granted non-qualified stock options to purchase an aggregate of 5,933 shares of its...
CAMBRIDGE, Mass. — Blueprint Medicines Corporation announced the European Commission has approved AYVAKYT® (avapritinib) for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment. AYVAKYT is the first and only approved therapy for people living with ISM in Europe. Systemic mastocytosis...
SAN DIEGO – Bluestar Genomics announces it received U.S. Food and Drug Administration (FDA) Breakthrough Device designation for its proprietary noninvasive pancreatic cancer detection test in patients with new-onset diabetes. The FDA’s Breakthrough Device program accelerates the development of medical devices that have the potential to effectively treat or diagnose...
Ingelheim am Rhein, Germany – Boehringer Ingelheim announced today that the FIBRONEER™-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function.1 Based on these results, Boehringer Ingelheim will submit...