PHILADELPHIA--(BUSINESS WIRE)-- iECURE, a gene editing company focused on mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, today announced the appointment of George A. Diaz, M.D. Ph.D., as VP, Therapeutic Area Lead for Urea Cycle Disorders (UCDs). Dr. Diaz will lead iECURE’s clinical development and program strategy for its UCD programs which include ornithine transcarbamylase (OTC) deficiency and citrullinemia type 1 (CTLN1).

“George is a key hire as we solidify the clinical strategies for our gene editing programs. Importantly, he’s a highly regarded scientific leader with deep expertise in urea cycle disorders and the clinical development of novel therapeutics for rare genetic diseases, and that experience will be invaluable for the advancement of our programs,” said Joseph Truitt, Chief Executive Officer of iECURE. “Both CTLN1 and OTC deficiency specifically can be devastating and often result in severe neurological damage, coma and, unfortunately, can be fatal. Patient communities in UCDs need durable treatment options and George’s guidance will be integral as iECURE embarks on clinical development and beyond.”

Dr. Diaz has more than 25 years of experience in genetics and pediatrics, particularly in metabolic diseases and monogenic disorders. Most recently, he served as Chief of the Division of Medical Genetics and Professor in the Departments of Genetics & Genomic Sciences and Pediatrics at Icahn School of Medicine at Mount Sinai in New York. Over his remarkable career at Mount Sinai, Dr. Diaz has collaborated extensively with industry partners to bring novel therapeutics to patients with UCDs and other rare genetic disorders. In addition to his roles at Mount Sinai, he has served as Director of the Program for Inherited Metabolic Diseases, a nationally recognized specialty center for the evaluation and care of patients with inborn errors of metabolism. His work in this arena has been recognized at the national level with service to the Coriell Institute Scientific Advisory Board for the National Institute of General Medical Sciences (NIGMS) Genetic Cell Repository and as a faculty member of the North American Metabolic Academy, the SIMD-sponsored national workshop course in inborn errors of metabolism. His clinical research in innovative therapeutics for rare diseases has resulted in the initiation of multiple clinical trials using advanced gene, RNA and enzyme substitution therapies across a number of rare genetic disorders. Dr. Diaz earned his B.A. in biology from Cornell University and his M.D. and Ph.D. from State University of New York Health Science Center at Brooklyn.

“Having the opportunity to join the iECURE team when the company is on the cusp of entering clinical trials with in vivo gene insertion therapies is exciting,” said Dr. Diaz. “Throughout my career I have been committed to driving innovation for young patients and their families impacted by devastating monogenic disorders. With strong foundational science and a mutation-agnostic approach, iECURE has the potential to develop transformational medicines for these devastating liver diseases, and I look forward to being a part of it.”

About iECURE

iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of monogenic liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program, or GTP, led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit www.iecure.com and follow on LinkedIn.

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