Our Story and Mission

Our Story and Mission

A beacon for patients living with severe rare genetic diseases

Founded in 2018, 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. 

We have advanced nizubaglustat into the clinic and have successfully completed a Phase 1 first-in-human trial in healthy volunteers.  These results have been published in the peer-reviewed journal, Molecular Genetics and Metabolism.

As we continue to develop nizubaglustat, we are also strengthening our partnership with academics to expand our pipeline into treating other severe and rare metabolic diseases.