iECURE to Present Initial Data from Phase 1/2 OTC-HOPE Clinical Trial in Ornithine Transcarbamylase Deficiency at ACMG Annual Clinical Genetics Meeting
iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced that initial data from the Phase 1/2 OTC-HOPE clinical trial evaluating in vivo gene editing candidate ECUR-506 in neonatal onset ornithine transcarbamylase (OTC) deficiency will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting taking place March 18-22, 2025 in Los Angeles.
iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency
iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders with significant unmet need, today reported preliminary findings from the first infant dosed in the ongoing OTC-HOPE Phase 1/2 study of ECUR-506, the company’s clinical candidate designed to treat neonatal onset ornithine transcarbamylase (OTC) deficiency. Treatment with ECUR-506 was generally well tolerated in this infant with no significant clinical safety concerns apart from asymptomatic transaminitis at four weeks. The asymptomatic transaminitis was managed with immunosuppressive therapy and resolved within four weeks. Twelve weeks after a single dose of ECUR-506, ammonia scavenger medication was discontinued and mean daily protein intake was increased to age-appropriate levels. Protein liberalization was well tolerated, and the subsequent mean ammonia level remained within normal limits and was reduced compared to the mean pretreatment level.
iECURE Receives FDA Fast Track Designation for ECUR-506 for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ECUR-506, the company’s in vivo gene insertion program designed to treat neonatal onset Ornithine Transcarbamylase (OTC) deficiency.
iECURE Announces FDA Clearance of Investigational New Drug Application for ECUR-506 to Initiate OTC-HOPE Trial for Treatment of Neonatal Onset Ornithine Transcarbamylase Deficiency in the U.S.
iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for ECUR-506 in Ornithine Transcarbamylase (OTC) 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 (MHRA) and Australia by the Therapeutic Goods Administration (TGA).
iECURE Secures Approval from U.K. Medicines & Healthcare Products Regulatory Agency to Expand the OTC-HOPE Study of ECUR-506
iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today approval from the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) of the company’s Clinical Trial Authorisation application (CTA) to expand the OTC-HOPE study into the U.K. The OTC-HOPE study is investigating ECUR-506, an investigational gene editing-based therapy, for the treatment of Ornithine Transcarbamylase (OTC) deficiency in infants. The CTA approval by the MHRA follows the previous approval to begin the OTC-HOPE study by the Australian Therapeutic Goods Administration (TGA).
