Orphan Technologies

Orphan Technologies is dedicated to reducing the burden on patients with homocystinuria. Their lead drug development candidate, OT-58, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disorder characterized by debilitating cardiovascular, skeletal, neurological, and ophthalmic complications. OT-58 aims to reduce homocysteine levels through a targeted mechanism of action and may have therapeutic applications in other diseases.