Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2022 on request of the Sponsor.
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
Treatment of sickle cell disease
|Orphan designation status||
|EU designation number||
|Date of designation||
|September 2022||Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.|
|April 2022||The sponsorship was transferred to PPD Bulgaria EOOD, Bulgaria, in April 2022.|
Documents related to this orphan designation evaluation
EU/3/20/2356: Public summary of opinion on orphan designation - Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene for the treatment of sickle cell disease (PDF/189.71 KB)
First published: 15/02/2021
Last updated: 16/09/2022
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: