This page shows the latest beta-thalassemia news and features for those working in and with pharma, biotech and healthcare.
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
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
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.
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,
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
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).
More from news
Approximately 9 fully matching, plus 24 partially matching documents found.
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
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
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 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
with sickle cell disease or beta thalassemia with FDA Fast Track Designation.
More from intelligence
Approximately 0 fully matching, plus 8 partially matching documents found.
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.
The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia.
More from PMHub
Approximately 0 fully matching, plus 2 partially matching documents found.
We’re a specialist health consultancy that supports companies, healthcare professionals and patients to work hand in hand with a common...