European grant for gene therapy for rare disease

Sanquin Research and SanaGen B.V., both housed on the Health & Innovation District Amsterdam (Hid), will be developing a new gene therapy for the rare disease alpha-1-antitrypsin deficiency (AATD). A serious disease for which a solution is desperately needed. They are awarded a grant of 1.3 million euro from Eurostars, in a consortium with the Swiss company InSphero. 

Patients who make too little or malformed alpha-1 antitrypsin, a protein that is produced by the liver, can develop severe lung or liver problems. Treatment now consists mainly of symptom management. Gene therapy might offer a solution for these patients. Jan Voorberg of Sanquin Research: “We look forward to working together with InSphero and SanaGen on this project and we hope that our unique approach will allow us to develop a novel therapy for patients with this chronic disorder”.

Eurostars is part of the European Partnership on Innovative SMEs. The partnership is co-funded by the European Union through Horizon Europe. (Project NOCAAVAAT)