On 19 October 2020, orphan designation EU/3/20/2346 was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated virus serotype hu68 containing the human GLB1 gene (also known as PBGM01) for the treatment of GM1 gangliosidosis.
Adeno-associated virus serotype hu68 containing the human GLB1 gene
|Disease / condition||
Treatment of GM1 gangliosidosis
|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.