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beta-thalassemia
This page shows the latest beta-thalassemia news and features for those working in and with pharma, biotech and healthcare.
bluebird bio’s beti-cel accepted for priority review by FDA
The tough journey to approval for beta-thalassemia gene therapy (beti-cel) in the US may be coming to an end, with approval possible by 2022. ... The one-off gene therapy is for adult, adolescent and paediatric patients with beta-thalassemia across all
Latest news
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bluebird bio to restart marketing of Zynteglo in EU after previous suspension
beta-thalassemia gene therapy. ... We are pleased to resume offering Zynteglo to patients living with transfusion-dependent beta-thalassemia, offering the potential to live free from transfusions which is evidenced by our clinical studies where
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bluebird bio presents new data for beti-cel in beta thalassemia at EHA2021
bluebird bio has presented new data for its gene therapy betibeglogene autotemcel (beti-cel) at the European Hematology Association 2021 virtual congress (EHA) in transfusion dependent beta thalassemia (TDT). .
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BMS, Acceleron’s Reblozyl shows new improvements for beta thalassemia patients
Bristol Myers Squibb (BMS) and Acceleron have revealed the first data from a phase 2 study of its erythroid maturation agent Reblozyl in adults with non-transfusion dependent (NTD) beta thalassemia. ... Rebloyzl is the first erythroid maturation agent to
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FDA lifts clinical hold on bluebird bio’s LentiGlobin studies
The US Food and Drug Administration (FDA) has lifted the clinical holds on numerous studies evaluating its gene therapy LentiGlobin for sickle cell disease (SCD) and beta-thalassemia. ... We look forward to resuming our clinical programmes and continuing
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bluebird bio says AML case "very unlikely" to be related to gene therapy LentiGlobin
the FDA that we believe these results support lifting the clinical holds on our beta-thalassemia and sickle cell disease programmes,” he added. ... bluebird bio said that, based on the available data, it has initiated discussions with regulators to
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Latest Intelligence
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The trials and tribulations of rare opportunities
Revenue projection in the year 2025 is $1.259bn,” he continued. Ringheim cited Celgene’s beta thalassemia candidate Sotatercept, which is expected to achieve sales of $458m by 2023, as a
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Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019
LentiGlobin (betibeglogene darolentivec), an Orphan Drug and Breakthrough Therapy from Bluebird Bio, corrects the defect causing beta thalassemia, a blood disorder that causes life-threatening anaemia. ... The initial studies for LentiGlobin in
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Bluebird: on a mission to ‘recode’ the DNA of healthcare
Bluebird Bio is just weeks away from gaining its first ever approval for its first ever product, Zynteglo, a groundbreaking gene therapy treatment for beta thalassemia. ... Increasingly factored into this equation is competition from companies following
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Vertex: a vision of treating all CF patients worldwide draws closer
with sickle cell disease or beta thalassemia with FDA Fast Track Designation.
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2019: CRISPR and therapeutic gene editing comes of age
beta thalassemia – to become the first CRISPR drug to start testing in Europe. ... The FDA placed the CTX001 programmed in beta thalassemia and a planned trial in sickle cell disease on a clinical hold shortly afterwards, but relaxed that last October.
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