On 24 April 2015, orphan designation (EU/3/15/1482) was granted by the European Commission to Horama SAS, France, for adeno-associated viral vector serotype 5 containing the human CHM gene for the treatment of choroideremia.
In November 2016 Horama SAS changed name to Horama SA.
The sponsorship was transferred to Inserm-Transfert SA, France, in October 2017.
Adeno-associated viral vector serotype 5 containing the human CHM gene
|Disease / condition||
Treatment of choroideremia
|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:
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.