Our Story and Mission

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. Nizubaglustat has successfully completed a Phase 1 first-in-human trial in healthy volunteers, with results published in the peer-reviewed journal Molecular Genetics and Metabolism. Building on the positive outcomes of our Phase 2 (RAINBOW) study, we have now advanced nizubaglustat into a Phase 3 development program with two pivotal trials (NAVIGATE) — one in NPC and one in GM1/GM2 gangliosidoses. 

As we advance nizubaglustat in the clinic, we continue to expand our pipeline in collaboration with leading academics, with the aim of developing disease-modifying therapeutics for other severe and underserved metabolic conditions. By applying our knowledge, network, and courage, Azafaros challenges traditional development pathways to accelerate the delivery of new medicines to patients. 

The company is backed by leading healthcare investors, including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.