EMERYVILLE, Calif.--(BUSINESS WIRE)--Metagenomi, a genetic medicines company with a versatile portfolio of next-generation gene editing tools, today announced that it presented preclinical in vivo data on a novel, small, hypoimmune CRISPR-associated Type V gene editing system at the American Association for the Study of Liver Diseases (AASLD), The Liver Meeting, occurring virtually November 12–15.
“In vivo gene editing has the potential to treat many genetic diseases for which there are currently no cures, including those that impact the liver and its metabolic functions,” said Brian C. Thomas, PhD, CEO and Co-Founder of Metagenomi. “One challenge of developing effective genetic medicines is that pre-existing immunity to CRISPR/Cas-based systems may limit their efficacy as a therapeutic. To address this challenge, we search Earth’s natural environments for novel gene editing systems to which humans do not possess pre-existing immunity. Our Type V CRISPR-associated nuclease is a promising novel in vivo gene editing system that is small enough to fit in AAV, has shown highly specific and efficient editing in liver cells in a mouse model, and for which a panel of healthy human donors had no pre-existing immunity.”
In the findings presented, Metagenomi’s novel Type V CRISPR-associated nuclease was highly active in the liver of mice when systemically administered via lipid nanoparticles (LNP). The nuclease was derived from a unique natural environment and is phylogenetically distinct from previously identified Type V systems. Moreover, no antibodies to the nuclease were detected in serum from 50 healthy human donors, while between one third and half of the same serum samples contained antibodies that bind to spCas9, which is derived from human strep bacteria. In summary, this novel Type V CRISPR-associated nuclease is a promising new gene editing system for in vivo editing of the liver.
Details of the poster are below:
Title: A novel CRISPR Associated Type V Editing System Derived from Metagenomic Samples with Potent Activity in Liver Cells In Vitro and In Vivo
Presenting Author: Alan Brooks, PhD, Vice President of Pre-Clinical Research, Metagenomi
Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.