10 September 2021 - - US-based clinical-stage biotechnology company Precision BioSciences, Inc. (NASDAQ: DTIL) and US-based mutation-agnostic in vivo gene editing company iECURE have inked a license and collaboration agreement under which iECURE plans to advance Precision's PBGENE-PCSK9 candidate into Phase 1 studies and gain access to Precision's PCSK9-directed ARCUS nuclease to develop additional gene editing therapies for genetic diseases, initially targeting liver diseases, the companies said.

Under the terms of the agreement, iECURE plans to file a clinical trial application as early as 2022 to advance the PBGENE-PCSK9 clinical candidate through Phase 1 clinical studies for the treatment of familial hypercholesterolemia.

Precision will retain rights to PBGENE-PCSK9, including all products developed for genetic indications with increased risk of severe cardiovascular events such as FH.

In return, Precision has granted iECURE a license to use its PCSK9-directed ARCUS nuclease to insert genes into the well-characterized PCSK9 locus to develop treatments for four other pre-specified rare genetic diseases.

Precision will receive an equity stake in iECURE and is eligible to receive milestone and royalty payments on sales of iECURE products developed with ARCUS.

James M. Wilson, M.D., Ph.D., chief scientific Advisor of iECURE and Professor in the Departments of Medicine and Pediatrics, Perelman School of Medicine, University of Pennsylvania, director, Gene Therapy Program, will present new non-human primate data demonstrating ARCUS-mediated gene addition TODAY, September 9, 2021 during the Precision BioSciences gene editing R and D event.

Dr. Wilson and his team have demonstrated in non-human primates that it is possible to use ARCUS to insert new genes stably into the PCSK9 locus, which could be used as a potential approach for treating multiple genetic diseases with a single therapeutic strategy.

ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock in), remove (knockout), or repair DNA of living cells and organisms.

ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA.

Precision's platform and products are protected by a comprehensive portfolio including more than 80 patents to date.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life with its novel and proprietary ARCUS genome editing platform.

ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind.

Using ARCUS, the company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist.

iECURE is an in vivo gene editing company striving to cure liver disorders with high unmet need. It is advancing a pipeline in close partnership with the translational engine at the University of Pennsylvania's Gene Therapy Program.