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...
treatment News
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 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...
Breadth of presentations, including one oral and two flash talks, showcase Blueprint Medicines’ leadership role in advancing care for patients with systemic mastocytosis CAMBRIDGE, Mass. — Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the...
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...
FORT WASHINGTON, Pennsylvania and INGELHEIM, Germany – Boehringer Ingelheim and Vitae Pharmaceuticals, Inc., announced today that they have entered into a significant worldwide collaboration to research and develop beta-secretase (BACE) inhibitors for the treatment of Alzheimer’s disease. Current therapies for Alzheimer’s disease can improve symptoms, but do not affect the...