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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 maintains its focus on gene therapy opportunities during structural changes
The company intends to increase its focus on near-term catalysts – including anticipated FDA approvals for beta-thalassemia and cerebral adrenoleukodystrophy gene therapies. ... Indeed, if approved, betibeglogene autotemcel (beti-cel) for
Latest news
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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
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Vertex expands further into cell and genetic therapies with a deal worth up to $1.2bn
and sickle cell disease and beta thalassemia. ... In the same month, the company extended its collaboration with CRISP Therapeutics for the development, manufacturing and commercialisation of CTX001 in sickle cell disease and beta thalassemia.
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Gene therapy company bluebird bio quits Europe
While the company has not announced how much it asked for Lenti-D, earlier this year, it withdrew its beta-thalassemia therapy from Germany after the government offered $790, 000 upfront,
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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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Approximately 9 fully matching, plus 24 partially matching documents found.
Latest Intelligence
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Who needs to be trusted anyway?
Already there are a score or more approved (BluebirdBio’s Zynteglo for beta thalassemia among the latest), and with a further 3, 633 and counting in development, we are about to
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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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Approximately 0 fully matching, plus 8 partially matching documents found.
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The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia
Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. ... Earlier this year, the US Food and Drug Administration (FDA) approved the first potentially curative gene therapy to treat beta-thalassemia.
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Fishawack Health
The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia.
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Approximately 0 fully matching, plus 2 partially matching documents found.
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