Our Story and Mission

Our Story and Mission

A beacon for patients living with severe rare genetic diseases

Founded in 2018 with a deep understanding of rare genetic disease mechanisms 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. Our lead clinical-staged program is AZ-3102, a highly differentiated, orally available, small molecule with the potential to treat GM1 and GM2 Gangliosidosis and other metabolic disorders. By applying our 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.

Leveraging the know-how of our team and partners, we have advanced AZ-3102 into the clinic and have successfully completed a Phase 1 First-in-Human trial in healthy volunteers. As we continue to develop AZ-3102, we will also expand our pipeline into other severe rare metabolic diseases.

Team of Azafaros