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Azafaros is a clinical stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, a compound library from Leiden University, and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer patients and their families new treatment options. The company’s lead clinical-staged program is AZ-3102 a small molecule, orally available, brain penetrant azasugar, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann-Pick disease type C (NP-C). By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them.

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Patient and Trial Information

Gisela Linthorst, Head of Patient Engagement

Latest News

NPC Community Meeting with Azafaros hosted by NNPDF & NTSAD
February 8, 2023  |  8:00 pm EST


Updates from Azafaros

Azafaros receives FDA IND clearance and Fast Track Designation for its lead asset AZ-3102 for treatment of Niemann-Pick disease type C (NPC). Read complete announcement.

Azafaros also announces organizational changes within the company. Read press release.



Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease type C

Read complete announcement.



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