β-thalassaemia
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News
ACCESS TO INNOVATION | UK Greenlights Gene-Editing Therapy Casgevy for β-Thalassaemia Treatment
This development opens new treatment avenues for an estimated 460 patients aged 12 and above in England who rely on regular transfusions due to β-thalassaemia, leveraging innovative CRISPR gene-editing technology.…
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BREAKING | Vertex and CRISPR’s Gene-editing Therapy Casgevy Receives Early FDA Approval for β-Thalassaemia Treatment
Just one month prior, Casgevy made history as the first CRISPR gene editing therapy to enter the US market when it was approved for sickle cell disease. Now, the FDA…
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IRON CONTROL | Disc Medicine And Mabwell Sign Deal For Licence To Antibodies Portfolio
Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023. MWTX-003 has the potential to address a wide range of hematologic disorders including…
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HSCT | Briquilimab Granted Orphan Drug Designation By The European Commission
Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency…
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CLINICAL UPDATE | Graphite’s Hopes For Sickle Cell Cure Blunted After First Patient Dosed Experiences Serious Event
The company cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) as part of a phase 1/2 trial for SCD. The event is likely to…
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BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and…
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POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells
This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…
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PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
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FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
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