OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency
https://iecure.com/wp-content/uploads/ASHG_iEcure_72x36_poster.pdf
Initial Findings from the First Four Patients Enrolled in OTC-HOPE Clinical Trial: No Hyperammonemic Events in First Participant to Complete 24-week Study
https://iecure.com/wp-content/uploads/ICIEM-poster_web.pdf
Initial clinical results from OTC-HOPE, the first in vivo, liver directed, AAV-mediated gene insertion study in neonatal OTC deficiency: complete clinical response observed in first male infant to receive ECUR-506 and complete 24-week Phase 1/2 study
https://iecure.com/wp-content/uploads/iECURE_ASGCT_Poster-852_Final.pdf