On 12 December 2017, orphan designation (EU/3/17/1950) was granted by the European Commission to MeiraGTx UK II Limited, United Kingdom, for adenovirus associated viral vector serotype 8 containing the human AIPL1 gene for the treatment of Leber's congenital amaurosis.
The sponsorship was transferred to MeiraGTx B.V., The Netherlands in May 2019.
Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene
|Disease / condition||
Treatment of Leber's congenital amaurosis
|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;