EMERYVILLE, Calif.--(BUSINESS WIRE)-- Metagenomi, a genetic medicines company with a versatile portfolio of wholly-owned, next-generation gene editing tools, today presented new preclinical data on proprietary CRISPR-associated systems that have the potential to be used in the development of cell therapy and a novel family of CRISPR-associated transposases (CASTs) at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting.

The first presentation demonstrates that Metagenomi’s gene editing systems achieve over 95% editing efficiency in human primary and stem cells at multiple target loci relevant to cell therapy development. As an important differentiation, these novel, proprietary gene editing systems lack pre-existing antibody and T cell immunity, potentially enhancing their translation to therapeutic application when compared to CRISPR-Cas9.

The second presentation demonstrates a novel, metagenomics-sourced CRISPR-associated transposase editing system, also known as CAST system, displaying a high level of integration at multiple, targeted sites in both endogenous and engineered loci in the E. coli genome.

“Our research presented at ASGCT illustrates key advantages of our gene editing systems. They are versatile and highly specific - properties essential to their therapeutic application,” said Brian C. Thomas, PhD, Founder and CEO of Metagenomi. “Our presentations at ASGCT reemphasize Metagenomi’s ability to rapidly identify and characterize novel gene editing systems from natural environments with potential advantages for in vivo as well as ex vivo applications.”

The first presentation, delivered by Gregory J. Cost, PhD, Vice President of Biology at Metagenomi, demonstrates the Company’s novel proprietary CRISPR-associated gene editing systems’ ability to edit primary human cells with high efficiency, including T cells, natural killer (NK) cells, B cells, hematopoietic stem cells (HSCs), and induced pluripotent stem cells, often reaching an editing efficiency of more than 95 percent. These novel nucleases were sourced from environmental samples using Metagenomi’s proprietary discovery engine, showing no detectable antibodies to these systems, overcoming the potential challenge of pre-existing immunity which other gene editing systems have.

The second presentation, given by Jason Liu, PhD, Scientist at Metagenomi, demonstrates a novel family of proprietary CRISPR-associated transposase (CAST) gene editing systems. These CAST systems are shown to be efficient, programmable, and allow for site-specific transgene integration - important capabilities for next-generation gene editing systems that need to go beyond knock-out and knock-in applications used by CRISPR/Cas-based nucleases. After introducing CASTs into E. coli, high rates of on-target integration at different endogenous and engineered loci were observed, a critical proof-of-concept for the next phase of development for these novel systems.

About 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/.

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