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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.

Meanwhile Bluebird is proposing a similar five-year instalment plan for its 1.57m beta thalassemia therapy Zynteglo.

Bluebird puts €1.57m price on gene therapy Zynteglo

US biotech pioneering outcomes-based payment in Europe. Bluebird Bio’s gene therapy treatment for beta thalassemia Zynteglo is to be priced at 1.575m ($1.76m) in Europe, where the ... As the therapy is only currently licensed for use in beta
A first for Bluebird and Europe as gene therapy Zynteglo approved

That’s because Bluebird believes it can offer a compelling cost-effectiveness case for its treatment in beta thalassemia and sickle cell cases versus conventional stem cell treatment. ... beta thalassemia patients live in these four count.
Novartis secures US approval - and payers’ blessing – for $2m gene therapy Zolgensma

Another gene therapy pioneer, with a similar payment model looks likely to beat Novartis to the European market, however - Bluebird Bio and its beta thalassemia treatment.
Europe responding to gene therapy challenge, but picture remains fragmented

its gene therapies, starting with its soon-to-be-approved beta thalassemia therapy Zynteglo.
Post-merger Celgene to lead pharma growth

Celgene also has five potential blockbusters in its late-stage pipeline, including MS treatment ozanimod and myelodysplastic syndromes/beta thalassemia candidate luspatercept, which have both recently been filed.

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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
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
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
Vertex: a vision of treating all CF patients worldwide draws closer

with sickle cell disease or beta thalassemia with FDA Fast Track Designation.
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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