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.
The sponsor’s address was updated in July 2021.
Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene
Treatment of Leber's congenital amaurosis
|Date of designation||
|Orphan designation status||
|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.