Metagenomi, a gene editing company, today presented data on their novel CRISPR-associated transposases (CAST) system that allows for precise integration of large DNA fragments into genomes at the 24th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), which is being held virtually.
"Gene editing holds enormous promise for the development of therapies that can treat a wide range of diseases, but there is a need to expand our gene editing tool kit to allow for edits that are not possible with current techniques," said Brian C. Thomas, Ph.D., Co-Founder and CEO of Metagenomi. "Our research presented at ASGCT describes how our first-in-class programmable CAST gene editing system can be used to precisely integrate large fragments of DNA into target genomes and the potential of these systems in the development of both ex vivo and in vivo gene therapies."
CRISPR-associated transposases can be reprogrammed to integrate at specific genomic sites using guide RNAs. In the presentation given at ASGCT by Daniela S. A. Goltsman, Ph.D., Senior Scientist at Metagenomi, targeted integration by Metagenomi’s CAST system was studied in vitro and integration of the DNA fragment into the target genome was confirmed via PCR amplification and sequencing. CAST natively uses a dual guide RNA, however Metagenomi has developed a smaller, single guide RNA that reduces the size of the system while maintaining targeted integration. The novel CAST used in this study was identified by Metagenomi’s proprietary discovery engine that leverages insights from the field of metagenomics and can scan hundreds of thousands of microbial genomes to identify promising systems for gene editing.
In addition to this presentation, Metagenomi presented posters on three additional topics relating to novel CRISPR systems on May 11th at ASGCT. These posters included: A novel CRISPR associated Type V editing system derived from metagenomic samples with potent activity in liver cells; Expanding PAM recognition of CRISPR-associated endonucleases by domain engineering; and Novel families of CRISPR systems enriched in small effectors with genome editing capability. Metagenomi also gave a short talk titled: "Novel CRISPR-associated Gene-editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering for Cell Therapy Development."
At Metagenomi, we are accelerating innovation in cell and gene therapy with a wave of proprietary CRISPR-based 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 therapeutic tools that can be leveraged by partners and fuel our own pipeline of potentially curative medicines. Our goal is to revolutionize gene editing and unlock its power for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.
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