All Casgevy articles
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NewsNICE recommends gene therapy for severe sickle cell disease
The committee’s approval of the one-time gene therapy means the healthcare inequalities for patients with this genetic blood disorder can begin to be re-addressed.
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ArticleRegulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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NewsCRISPR technologies fuelling haematological innovations
Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
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NewsVertex secures European approval for CRISPR cell therapy
Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
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NewsCHMP issues positive option for first gene-editing medicine
If approved, Vertex’s Casgevy (exagamglogene autotemcel) would be the first gene-editing medicine authorised in the European Union.
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NewsFDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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NewsFirst CRISPR-based gene-editing therapy authorised
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a world-first regulatory authorisation to a medicine based on Nobel Prize-winning technology.