Azafaros

Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced industry experts in rare diseases, Azafaros is working on building a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. Our lead clinical-stage program is nizubaglustat, an orally available, brain-penetrant small molecule with a unique dual mode of action which has the potential to treat GM1 and GM2 gangliosidoses, Niemann-Pick disease type C, and other metabolic disorders.