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IECURE Secures FDA Approval for ECUR-506's Investigational New Drug Application, Launching OTC-HOPE Trial for Neonatal Onset Ornithine Transcarbamylase Deficiency Treatment in the U.S.

Friday, April 05, 2024

IECURE, a pioneering gene editing company dedicated to addressing liver disorders with significant unmet medical needs, has announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for ECUR-506 in Ornithine Transcarbamylase (OTC) deficiency. This milestone allows the company to proceed with its groundbreaking OTC-HOPE study, which was previously approved in the United Kingdom and Australia.

CEO Joe Truitt expressed enthusiasm about the global reach of the trial, stating, "With this IND clearance, we are now activating sites in three countries, facilitating families' access to this landmark clinical trial." The trial aims to evaluate ECUR-506 in newborn males with genetically confirmed neonatal onset OTC deficiency, welcoming participants from around the world.

The Phase 1/2 OTC-HOPE study primarily focuses on assessing the safety and tolerability of ECUR-506 following intravenous administration of a single dose in newborn males with OTC deficiency. Secondary objectives include evaluating its pharmacokinetics and efficacy, while exploratory endpoints will delve into disease-specific markers, developmental milestones, and quality of life.

Dr. Gabriel M. Cohn, Chief Medical Officer, emphasized the urgent need for treatment options for newborns with severe OTC deficiency, stating, "ECUR-506 represents hope for potentially enabling children to produce functional OTC enzyme in the long term without the need for transplantation."

The development of ECUR-506 marks a significant milestone in gene editing, as it represents the first in vivo gene insertion program to be cleared in the U.S. for study in infants. Dr. James M. Wilson, whose laboratory developed ECUR-506, expressed optimism about its potential to bring meaningful improvements to infants with neonatal onset OTC deficiency and their families.

ECUR-506 utilizes a unique approach to gene editing, involving the delivery of two adeno-associated virus (AAV) capsids carrying different payloads. The program employs the ARCUS® nuclease to target gene editing and facilitate the insertion of the functional OTC gene. This innovative strategy offers a potential pathway to long-term expression of a healthy gene.

The OTC-HOPE study represents a critical step forward in addressing the urgent medical needs of newborns with severe OTC deficiency. It offers hope for a novel therapeutic approach that could significantly impact the lives of affected individuals and their families.



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