iECURE Receives FDA Fast Track Designation for ECUR-506 for the Treatment of 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 Secures Approval to Expand the OTC-HOPE Study of ECUR-506
iECURE Secures Clearance from TGA for Trial Application for ECUR-506
iECURE Appoints Gabriel M. Cohn, M.D., as Chief Medical Officer
Jim Wilson-founded biotech iECURE has wrapped a $65M Series A extension round to get its lead candidate — a gene replacement therapy for a rare inherited liver disease known as ornithine transcarbamylase deficiency, or OTC — into the clinic.
PHILADELPHIA--(BUSINESS WIRE)--iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, today announced that data presented at the International Conference on Ureagenesis Defects and Allied Conditions 2022 by its research collaborators led by James Wilson, MD, PhD, at the University of Pennsylvania’s Gene Therapy Program (GTP), showed stable insertion of the therapeutic gene one year post-dosing in newborn and infant macaques and potential signs of efficacy in mouse Ornithine Transcarbamylase (OTC) Deficiency knock out models.
PHILADELPHIA--(BUSINESS WIRE)--iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate GTP-506, an investigational product, for the treatment of Ornithine Transcarbamylase (OTC) deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death in a pediatric population.
PHILADELPHIA--(BUSINESS WIRE)--iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death, in a pediatric population.
iECURE, a gene editing company focused on mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, and Center for Breakthrough Medicines (CBM), have entered into a collaboration wherein CBM will produce and supply good manufacturing practices (GMP)-grade adeno-associated virus (AAV) for use in iECURE’s future clinical studies.