Success Story

iECURE Gets Early FDA Designation

Penn spinout iECURE launched with a $50 million Series A led by Versant Ventures and Orbimed Advisors with a mission to develop its proprietary mutation agnostic in vivo gene editing approach.  The Series A proceeds will be used to advance a slate of gene insertion programs for liver disorders  discovered by 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 significant boost from the Food and Drug Administration (FDA) in September 2021 when GTP-506, its lead product candidate, was awarded orphan drug designation on the heels of having previously received rare pediatric disease designation in late August of that same year. 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.

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