Precision BioSciences (DTIL) Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol Levels in Nonhuman Primates

February 19, 2021 7:04 AM EST

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Precision BioSciences, Inc. (Nasdaq: DTIL) a clinical stage biotechnology company, today announced the publication of a paper in Molecular Therapy describing three-year follow-up data showing long-term stable reduction of low-density lipoprotein (LDL) cholesterol levels in nonhuman primates (NHPs) following in vivo gene editing of the PCSK9 gene with its proprietary ARCUS® genome editing platform.

The study, “Long-term Stable Reduction of Low-density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9” was published online in Molecular Therapy and was led by James M. Wilson, M.D., Ph.D., a professor of Medicine and director of the Penn Gene Therapy Program and the Penn Orphan Disease Center, and Lili Wang, Ph.D., a research director in the Penn Gene Therapy Program and research associate professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania.

“Building on the work we previously published in Nature Biotechnology in 2018, which was the first demonstrated use of any gene editing technology to create a clinically relevant reduction of gene expression of the PCSK9 protein in nonhuman primates, these latest pre-clinical results showed that targeted in vivo gene disruption with ARCUS has had a lasting therapeutic effect after a single dose, and provide pivotal data for safety considerations that support advancement towards clinical translation,” said Dr. Wilson. “These results not only contribute to the growing evidence of gene editing for potential therapeutic use, but specifically showed that ARCUS nuclease gene editing could be a very promising new approach leading to treatments for heart disease patients that do not tolerate commonly used PCSK9 inhibitors.”

Researchers at the University of Pennsylvania delivered a gene encoding an ARCUS nuclease by adeno-associated virus (AAV) to inactivate the PCSK9 gene and inhibit protein expression, which would normally prevent receptors from removing excess LDL (or “bad” cholesterol) in the liver. NHPs have been monitored for more than three years and have continued to show a sustained reduction in LDL cholesterol levels while maintaining stable gene editing without any obvious adverse effects. After the one-time vector administration more than three years ago, NHPs treated with ARCUS have experienced stable reductions of up to 85% in PCSK9 protein levels and a 56% reduction of LDL cholesterol levels.

“To our knowledge, this is the longest duration gene editing data in a large animal model. The data demonstrates that a single administration of an ARCUS nuclease could represent a potential one-time, permanent treatment for familial hypercholesteremia,” commented Derek Jantz, Ph.D., co-author of the paper and Chief Scientific Officer at Precision BioSciences. “ARCUS has attributes that we believe significantly differentiate it from RNAi or conventional AAV gene therapy approaches, as well as CRISPR gene editing approaches. At more than three years out, we are seeing a stable gene edit that is being inherited by subsequent generations of hepatocytes, and evidence thus far supports that this is a permanent change. We look forward to continued monitoring of these animals and applying these learnings to our other in vivo gene editing programs.”



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