iECURE, inc. Email Format

Biotechnology ResearchPennsylvania, United States11-50 Employees

iECURE is a clinical-stage biotechnology company focused on genome editing to treat neurometabolic disorders. It uses a variant-agnostic, in vivo gene insertion approach intended to restore function by inserting a functional copy of a missing or dysfunctional gene, with the goal of durable expression and potential long-term benefit. The company is developing a pipeline of genetic medicines led by ECUR-506 for neonatal-onset ornithine transcarbamylase deficiency and is based in Pennsylvania, United States, with headquarters in Blue Bell. Within the biotechnology research industry, iECURE positions itself at the intersection of gene editing and liver-related genetic disorders with significant unmet needs. Its target customers include patients affected by severe neurometabolic or liver disorders and clinicians seeking disease-modifying therapies; a recent update from the company notes partial restoration of OTC enzyme activity in the first patient dosed in the OTC-HOPE study.

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