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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 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). .
Latest news
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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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bluebird bio posts modest Q4 revenue growth as it continues investigation into troubled LentiGlobin
In addition to the clinical holds, bluebird has also temporarily paused marketing of the gene therapy in Europe, where it is currently marketed as Zynteglo for the treatment of beta thalassaemia. ... CALD mid-year and the planned US filings for
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bluebird bio temporarily suspends trials of sickle cell gene therapy LentiGlobin
Company will also suspend marketing of beta-thalassemia gene therapy Zynteglo. bluebird bio has announced that it will temporarily place phase 1/2 and phase 3 trials of its sickle cell ... Although no cases of haematologic malignancy have been reported
BMS, Acceleron’s Reblozyl shows new improvements for beta thalassemia patients
FDA lifts clinical hold on bluebird bio’s LentiGlobin studies
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Approximately 7 fully matching, plus 21 partially matching documents found.
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.
The trials and tribulations of rare opportunities
Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019
Bluebird: on a mission to ‘recode’ the DNA of healthcare
Vertex: a vision of treating all CF patients worldwide draws closer
2019: CRISPR and therapeutic gene editing comes of age
More from intelligence
Approximately 0 fully matching, plus 7 partially matching documents found.
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