A beacon for patients living with severe rare genetic diseases
Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms. Building on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and Amsterdam UMC, Azafaros is led by a team of highly experienced industry experts dedicated to bringing transformative therapies to patients and families affected by severe and rare diseases.
Our lead program, nizubaglustat, is an orally available, brain-penetrant small molecule with a unique dual mode of action and the potential to treat GM1 and GM2 gangliosidoses, Niemann-Pick type C disease (NPC), and other metabolic disorders... read more
A team of highly experienced industry experts
Azafaros is supported by a syndicate of leading Dutch and Swiss investors. Our clinical candidate, nizubaglustat, is an orally available azasugar designed to treat the central nervous system and to interfere with the metabolism of glycosphingolipids affecting key disease pathways through a unique dual mode of action.
