CRISPR 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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Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
If approved, Vertex’s Casgevy (exagamglogene autotemcel) would be the first gene-editing medicine authorised in the European Union.
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.
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.