On 4 July 2014, orphan designation (EU/3/14/1290) was granted by the European Commission to NightstaRx Ltd., United Kingdom, for adeno-associated viral vector serotype 2 containing the human REP1 gene for the treatment of choroideremia.
The sponsor’s address was updated in September 2018.
The sponsorship was transferred to Nightstar Europa Limited, Ireland, in February 2019.
Adeno-associated viral vector serotype 2 containing the human REP1 gene
|Disease / condition||
Treatment of choroideraemia
|Date of decision||
|Orphan decision number||
Review of designation
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;