On 18 November 2016, orphan designation (EU/3/16/1772) was granted by the European Commission to Audentes Therapeutics UK Limited, United Kingdom, for adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (also known as AT342) for the treatment of Crigler-Najjar syndrome.
The sponsorship was transferred to Audentes Therapeutics Netherlands B.V., Netherlands in May 2019.
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
|Disease / condition||
Treatment of Crigler-Najjar syndrome
|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;