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IECURE


iECURE is a clinical-stage rare disease company advancing in vivo targeted gene insertion therapies designed to address the underlying genetic cause of devastating liver disorders. We focus on severe, life-threatening conditions with significant unmet need, particularly in pediatric populations. Our variant-agnostic approach is designed to insert a functional copy of a gene directly into a patient’s cells, with the goal of enabling durable gene expression and the potential for long-term therapeutic benefit. iECURE’s pipeline is led by ECUR-506, an investigational therapy in clinical development for neonatal-onset ornithine transcarbamylase (OTC) deficiency. The company is headquartered in Blue Bell, Pennsylvania, and has been recognized as a Philadelphia Business Journal “Best Place to Work” and a Fierce Biotech “Fierce 15” company.

Therapeutic Developer


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