OT-58

Overview

OT-58 is a modified recombinant enzyme replacement therapy in development for patients suffering from the rare disease classical homocystinuria. OT-58 is designed to help patients reduce their homocysteine levels and restore a normal lifestyle.

OT-58 Development
Orphan Technologies has conducted rigorous preclinical evaluation or OT-58 with extremely promising results demonstrating the potential to reduce homocysteine levels. For a list of scientific publications on OT-58, please click here.

CLINICAL DEVELOPMENT

Orphan Technologies plans to begin testing OT-58 in patients in 2018.

In addition, Orphan Technologies is currently conducting the first ever Natural History Study in patients with homocystinuria. For more information on the Natural History Study, please click here (link to NHS section above) or to join the NHS, please email info@orphantechnologies.com

Natural History Study

In addition, Orphan Technologies is currently conducting the first ever Natural History Study in patients with homocystinuria to establish an understanding of homocystinuria disease progression and inform future clinical studies of potential new therapies for HCU. Learn more.

How OT-58 Works

OT-58 is designed to address the underlying cause of classical homocystinuria which is a deficiency in the naturally occurring enzyme cystathionine beta synthase (CBS). Reduced or total lack of CBS activity results in the inability to metabolize, or process, homocysteine. This can lead to dangerous elevations of homocysteine. OT-58, is a modified recombinant enzyme replacement therapy that may dramatically decrease tissue and plasma levels of homocysteine, and as a result may prevent, delay, and reverse clinical abnormalities, and reduce dietary restrictions