CONFRONTING DEVASTATION.
Patients living with devastating rare diseases need lasting, potentially curative therapies. At iECURE, we are advancing innovative genetic therapies designed to address the underlying cause of neurometabolic disorders, with the goal of delivering lasting benefit for patients and families.
FROM SCIENTIFIC INSIGHT TO CLINICAL PROGRESS.
Our in vivo targeted gene insertion approach builds on decades of scientific discovery and is designed to restore the function of a missing or dysfunctional gene, regardless of variant.
REALIZING THE PROMISE OF GENOME EDITING IN THE LIVER.
Many rare genetic disorders arise from metabolic pathways centered in the liver and often present early in life. Our initial programs focus on such disorders where treatment options are limited and long-term disease control remains a significant challenge.
A TEAM ADVANCING GENETIC MEDICINE WITH PURPOSE.
From foundational research to clinical development, we have built a team and platform dedicated to advancing genome editing–based investigational therapies for rare neurometabolic disorders.
iECURE to Present Preliminary Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at the ASGCT and SIMD Annual Meetings
/in news, press release/by eallisoniECURE Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of ECUR-506 Ahead of Planned BLA Submission
/in news, press release/by eallisoniECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
/in news, press release/by David Anderson