We are developing new treatment options to help patients with rare metabolic disorders.
Azafaros was founded in 2018 by a team of experienced industry professionals and scientists aspiring to address rare genetic metabolic disorders through a pipeline of oral small molecules with disease-modifying potential. Based on discoveries from Leiden University and Amsterdam UMC, Azafaros’s proprietary lead compound AZ-3102 will initially address inherited life-threatening lysosomal storage diseases for which there are no effective therapies today.
This orally available azasugar compound interferes with the metabolism of glycolipids and uniquely affects several key disease pathways through a dual mode of action. Leveraging the know-how of its team and partners in orphan drug development, the company is advancing its lead program toward first-in-man studies while further expanding its product pipeline into other rare metabolic diseases through its drug discovery efforts.