IECURE Granted Expansion Approval by U.K. MHRA for OTC-HOPE Study with ECUR-506
Thursday, March 07, 2024
IECURE, Inc., a pioneering gene editing company focused on addressing liver disorders with significant unmet medical needs, has announced that it has received approval from the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) for its Clinical Trial Authorisation application (CTA) to expand the OTC-HOPE study into the U.K. This study is centered on ECUR-506, an investigational gene editing therapy designed to treat Ornithine Transcarbamylase (OTC) deficiency in infants, and it was previously approved by the Australian Therapeutic Goods Administration (TGA).
Joe Truitt, Chief Executive Officer of iECURE, expressed confidence in the ECUR-506 program following the MHRA and TGA approvals, stating, "ECUR-506 marks a significant milestone as the first clinical meganuclease-based in vivo gene insertion program. As we advance towards the first dosing in humans, we are encouraged by the support shown by regulatory agencies." Truitt added that they are actively engaging with other regulatory bodies to expand the study into additional regions and preparing sites to enroll patients.
The OTC-HOPE study, a Phase 1/2 trial, aims to evaluate ECUR-506's safety and tolerability in newborn males diagnosed with neonatal onset OTC deficiency. Secondary objectives include assessing the therapy's pharmacokinetics, efficacy, and its impact on disease-specific biomarkers, developmental milestones, and quality of life.
Gabriel M. Cohn, MD, Chief Medical Officer of iECURE, emphasized the significance of ECUR-506 as a potential alternative to liver transplants for OTC deficiency patients. He stated, "ECUR-506 offers hope to families affected by this life-threatening condition by enabling durable production of functional OTC enzyme through gene editing, thus eliminating the need for liver transplants and lifelong immunosuppressive treatments."
ECUR-506 utilizes a two-vector approach involving an ARCUS® nuclease vector and a donor vector, targeting gene editing at the PCSK9 gene locus to insert the functional OTC gene. The therapy's mechanism offers a potential path to permanent expression of a healthy gene. The OTC-HOPE study aims to enroll baby boys aged 24 hours to seven months with severe neonatal onset OTC deficiency to evaluate the safety, efficacy, and potential benefits of ECUR-506 administration.
Source: businesswire.com
