Orphan Technologies is committed to reduce the burden of patients suffering from homocystinuria. OT-58, our lead drug development candidate, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases.
Dedicated to Patients
We put patients first. While innovative research and development is our backbone, we take the time to listen to what the patients and medical community are telling us. Basic research advances the field but our focus is on bringing therapeutics to patients that improve patients’ lives as quickly as possible.