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.
For more information, please visit azafaros.com.
Patient and Trial Information
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