Gene Therapies for Liver Diseases the Focus of New Collaboration


Precision BioSciences is collaborating with iECURE, a new company focused on mutation-agnostic, liver disease gene therapies.

Precision BioSciences and iECURE are collaborating to develop gene editing therapies for liver diseases.1

The collaboration allows iECURE access to Precision’s PBGENE-PCSK9 candidate, developed with their proprietary ARCUS technology, which they plan to advance into phase 1 studies for familial hypercholesterolemia. iECURE also gains access to the ARCUS nuclease which they plan to use to develop gene editing therapies, initially for liver diseases.

“We are excited to continue working with Jim Wilson under this new in vivo gene editing license and collaboration agreement with iECURE, as iECURE looks to rapidly advance our PBGENE-PCSK9 candidate, file for a clinical trial application in 2022, and use our PCSK9-directed ARCUS nuclease, and its knock-in capabilities, to pursue new treatments for rare genetic diseases,” said Derek Jantz, PhD, chief scientific officer and co-founder, Precision BioSciences, in a statement.1 “Through this collaboration we expect to gain important clinical validation for in vivo gene editing with ARCUS, while retaining rights to this PCSK9-directed nuclease, which we believe offers a safe harbor locus for DNA gene editing knock-in without deleterious effects when the PCSK9 gene is disrupted.”

iECURE plans to file a clinical trial application in 2022 for the PBGENE-PCSK9 phase 1 studies in familial hypercholesterolemia. ARCUS, which uses nucleases to edit genomes by insertion or deletion, will be used to develop therapies for 4 rare liver diseases.

READ MORE: Gene Therapies for Neurological Diseases Will Be Focus of New Shape Therapeutics, Roche Collaboration

James M. Wilson, MD, PhD, chief scientific advisor, iECURE and professor, Departments of Medicine and Pediatrics, Perelman School of Medicine, and director, gene therapy program, University of Pennsylvania, presented data on ARCUS-mediated gene addition in non-human primates during the Precision BioSciences gene editing R&D event on September 9, 2021. The data demonstrated the successful insertion of genes into the PCSK9 locus.

“We founded iECURE with the aim of focusing on genetic diseases with significant unmet need that we could target in a mutation-agnostic manner. After evaluating different gene editing technologies and platforms, we believe gene editing with ARCUS, including use of the uniquely designed ARCUS nuclease as a gene insertion tool targeting the PCSK9 gene will help us rapidly advance several candidates to the clinic with the potential to deliver on the promise of highly efficient, specific, and safe gene insertion,” said Joe Truitt, chief executive officer, iECURE.1 “We are excited to partner with Precision on this key pillar of our gene editing strategy, to advance this work for rare genetic diseases.”

iECURE launched in September 2021 based off of research in in-vivo gene editing for liver diseases from the University of Pennsylvania gene therapy program as part of their foundational collaboration with iECURE, which allows them access to 3 liver disorder programs being researched at the UPenn gene therapy program.2 They are focused on developing mutation-agnostic gene therapies.

“Through our foundational collaboration with Penn, we are gaining access to a comprehensive, liver-focused, in vivo gene editing program that Dr. Wilson and his team have been developing in his labs for several years,” Truitt said in a previous statement.2 “We are excited to partner with Penn to develop potentially groundbreaking treatments for patients suffering from debilitating disorders of the liver.”

1. Precision BioSciences and iECURE announce license and collaboration agreement to develop ARCUS-based gene editing therapies. News release. Precision BioSciences. September 9, 2021.
2. iECURE launches with $50 million series A financing to develop in vivo gene insertion approaches for devastating diseases. News release. IECURE. Seotember 9, 2021.
Related Videos
Paula Cannon, PhD, the president elect of ASGCT and a distinguished professor of microbiology at Keck School of Medicine of USC
George Tachas, PhD
Alexandra Gomez-Arteaga, MD
Pietro Genovese, PhD, the principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
© 2024 MJH Life Sciences

All rights reserved.