On 13 November 2020, orphan designation EU/3/20/2356 was granted by the European Commission to Clinical Technology Centre (Ireland) Limited, Ireland, for autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene (also known as ARU-1801) for the treatment of sickle cell disease.
Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene
|Disease / condition||
Treatment of sickle cell disease
|Date of first decision||
|EU designation number||
Sponsor's contact details
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.