On 9 January 2020, orphan designation EU/3/19/2242 was granted by the European Commission to Vertex Pharmaceuticals (Ireland) Limited, Ireland, for autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene (CTX001) for the treatment of sickle cell disease.
The sponsor’s address was updated in January 2022.
autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
Treatment of sickle cell disease
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Documents related to this orphan designation evaluation
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: