iECURE announces FDA clearance of IND application for ECUR-506

iECURE announced clearance of its Investigational New Drug application from the U.S. Food and Drug Administration for ECUR-506 in Ornithine Transcarbamylase deficiency. ECUR-506 will be evaluated in the OTC-HOPE study in newborn males with genetically confirmed neonatal onset OTC deficiency. The OTC-HOPE study was previously cleared to begin in the United Kingdom by the Medicines & Healthcare Products Regulatory Agency and Australia by the Therapeutic Goods Administration. The OTC-HOPE study is a Phase 1/2 first-in-human study in newborn males with genetically confirmed neonatal onset OTC deficiency. It is designed primarily to assess the safety and tolerability of ECUR-506 following intravenous administration of a single dose. Secondary objectives are to assess the pharmacokinetics and efficacy of ECUR-506. In addition, exploratory endpoints will assess disease-specific biologic markers, developmental milestones and quality of life. The ECUR-506 program is the first in vivo gene insertion program to be cleared in the U.S. for study in infants, and it represents the first time that the ARCUS nuclease has been used to provide an in vivo insertion point for a functional gene in the clinic. iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences.