iECURE Announces Presentation of Data on its Gene Editing Approach at the American Society of Cell and Gene Therapy Annual Meeting
May 3, 2022
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 that two oral presentations that highlight the company’s gene editing approach will be featured at the American Society of Cell and Gene Therapy (ASGCT) Annual Meeting, being held May 16-19, 2022 in Washington, D.C.
The first presentation, entitled “AAV-Meganuclease-Mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver,” will highlight long-term non-human primate data for iECURE’s gene editing approach for the treatment of ornithine transcarbamylase (OTC) deficiency. The second presentation, entitled “Generation of Efficient Lipid Nanoparticles for Liver-Directed Gene Therapy and Genome Editing,” focuses on the development of lipid nanoparticle (LNP) formulations targeted to the liver.
Title: AAV-Meganuclease-Mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver
Date and Time: Wednesday, May 18, 2022, 3:00 p.m. – 3:15 p.m. ET
Session Title: Presidential Symposium and Presentation of Top Abstracts
Location: Hall E
Abstract #: 811
Presenting Author: Lili Wang, Ph.D., Perelman School of Medicine at the University of Pennsylvania
Title: Generation of Efficient Lipid Nanoparticles for Liver-Directed Gene Therapy and Genome Editing
Date and Time: Tuesday, May 17, 2022, 5:15 p.m. – 5:30 p.m. ET
Session Title: Gene Editing in Cancer and Complex Diseases
Location: Hall E
Abstract #: 453
Presenting Author: Claude Warzecha, Ph.D., Perelman School of Medicine at the University of Pennsylvania
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.
Financial disclosure: The University of Pennsylvania (Penn) and Dr. Wilson hold equity interests in iECURE. Penn also receives significant sponsored research support from the company, and both Penn and Dr. Wilson benefit from licensing revenues received from iECURE based on successful technology development and commercialization of the technologies licensed from Penn. Dr. Wilson serves as Chief Scientific Advisor for iECURE.