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Metagenomi Presents AI-Enabled Advancements of SMART Editing Platform at the Cold Spring Harbor Laboratory CRISPR Frontiers Conference
AI-generated, compact SMART nucleases demonstrated robust genome editing activity in vitro in mammalian cells at multiple therapeutically relevant loci
About this update from Metagenomi Therapeutics, Inc.
[{"type":"text","content":"AI-generated, compact SMART nucleases demonstrated robust genome editing activity in vitro in mammalian cells at multiple therapeutically relevant loci Compact SMART genome editing tools that fit within delivery technologies constrained by cargo size may enable broad disease targeting EMERYVILLE, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today presented a poster titled “Unlocking the Therapeutic Potential of Compact SMART Nucleases through Ancestral Reconstruction, Structure-Guided Engineering, and Generative AI” at the tenth meeting on Genome Engineering: CRISPR Frontiers at Cold Spring Harbor Laboratory, hosted in Cold Spring Harbor, NY from August 27-31, 2024. The work leverages the company’s metagenomics-derived gene editing tools and a variety of AI methods to rapidly develop novel programmable CRISPR nuclease and base editing systems. “Gene editing is a revolutionary technology and novel gene editing tools are needed to address limitations with current systems, including the need for small systems that are compatible with currently available delivery technologies,” said Brian C. Thomas, CEO and founder of Metagenomi. “While there is no shortage of naturally occurring proteins that we can discover using our metagenomics platform, AI is one of several approaches we leverage to generate novel systems capable of highly efficient in vivo gene editing. We believe our metagenomics-based, AI-enhanced approach will allow us to achieve our goal of having the most effective gene editing tools to address any genetic disease anywhere in the human genome.” In today’s poster presentation, Metagenomi demonstrated that metagenomics-informed synthetic sequence generation, including ancestral sequence reconstruction and generative AI techniques, can produce novel nucleases and base editors. These AI-based gene editing system variants show high editing efficiency in mammalian cells and include compact base editors that are less than 1,000 amino acids in length. Other key points from the presentation include: Both the SMART nucleases and base editors fit well within the carrying capacity of viral vector-based delivery systems.Compact SMART nucleases are promising for genome...