Success Story

iECURE Gets Early FDA Designation

iECURE launched with a $50 million Series A and a mission to solve the “holy grail” of gene editing with an in vivo approach that is mutation agnostic and is using the funds for a slate of gene insertion programs for liver disorders out of Penn’s Gene Therapy Program led by Jim Wilson, M.D., Ph.D., Director, Gene Therapy Program, Rose H. Weiss Professor and Director, Orphan Disease Center, Professor of Medicine and Pediatrics, Penn Medicine.   The company received a boost from the Food and Drug Administration (FDA) for its lead product candidate which was awarded orphan drug designation for its experimental therapy, GTP-506, on the heels of the drug receiving rare pediatric disease designation in late August. GTP-506 is under development by iEcure as a potential treatment for ornithine transcarbamylase (OTC) deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death in children.  Versant Ventures provided the Series A funding for the company along with OrbiMed Advisors which will allow iECURE access to up to 13 programs out of Wilson’s Gene Therapy Program.   

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