On 4 June 2020, orphan designation EU/3/20/2287 was granted by the European Commission to Raremoon Consulting Limited, United Kingdom, for adeno-associated virus serotype 9 containing the human ASPA gene (also known as BBP-812) for the treatment of Canavan disease.
The sponsorship was transferred to Raremoon Consulting Esp S.L., Spain, in March 2021.
Adeno-associated virus serotype 9 containing the human ASPA gene
|Disease / condition||
Treatment of Canavan disease
|Date of first decision||
|EU designation 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.