The OTC-Hope Study, building hope one gene at a time logo.


OTC-HOPE study – a first-in-human study of a gene editing therapy for baby boys less than seven months of age with ornithine transcarbamylase (OTC) deficiency.

Baby boy that is resting his face on his folded hands, laying on a white blanket.

For additional information on this and other OTC deficiency clinical research studies, please visit If you are a healthcare provider and would like to learn more about this study, please email

More information on this study will be coming. Check back soon.


Please use this form to contact us if you would like to receive updates on the OTC-HOPE study.*

Baby boy's tiny four fingers wrapped around an adult index finger.