Vertex 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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Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
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.
The first regulatory submission for a CRISPR-based therapy has been validated by the European Medicines Agency, to treat sickle cell disease and transfusion-dependent beta thalassemia.
Winners of ISPE's Facility of the Year Awards (FOYA) 2022 include Janssen, Takeda, CRISPR Therapeutics, Catalent and Iovance Biotherapeutics.
Adam Pearson, Senior Oncology Analyst at GlobalData, comments on the development of CRISPR-based therapies and outlines potential opportunities and challenges in the space.