Metagenomi Presents Novel, Highly Specific and Efficient Adenine Base Editors for Broad Genome Editing at ESGCT

10.24.24

Metagenomi Adenine Base Editors (ABEs) demonstrated over 95% genome targetability

Simultaneous ABE triplex editing resulted in over 95% protein knockdown in primary T-cells

ABE demonstrated highly specific on-target deamination with no detectable translocations and no significant genomic composition differences

ABE triplex editing had no adverse effects on cell viability, expansion, or other measures of cell health

EMERYVILLE, Calif., Oct. 24, 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 (P0533 Poster Session III) titled “Efficient and specific genome editing with metagenomics-derived base editor for human therapeutic applications” at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress in Rome, Italy.

“Base editors are powerful gene editing tools that are able to modify genes through single nucleotide changes, including multiplexed gene editing applications without risk of translocations. Our ABEs display critical attributes necessary for successful multiplexed gene editing with remarkable targetability, efficiency, precision and tolerability,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We remain focused on advancing our base editors for in vivo genome editing programs, while pursuing partnerships to advance ex vivo cell therapy applications.”

Today’s poster presentation described the following key advantages of the Metagenomi ABE platform:

Targetability: Metagenomi’s ABE is targetable to over 95% of the human genome's base pairs, a significantly wider range of sites than first-generation SpCas9 base editors. This expanded scope provides unparalleled flexibility in addressing complex gene target locations.

Efficiency: The ABE platform achieved over 95% reproducible and durable triplex protein knockdown in primary T-cells, confirming its highly efficient application for multiplex gene editing. This platform is also compatible with various non-viral methods of site-specific template-integration in a simultaneous step, including CAR, engineered TCR, and/or regulatory genes leveraging a Metagenomi nuclease. This enables a potent all-in-one editing solution for the development of next-generation cell therapies.

Specificity: The ABE demonstrated highly specific on-target deamination with minimum-to-no indel formation. Genome-wide analyses confirmed no detectable translocations and no significant genomic composition differences when compared to unedited cells. This precision minimizes the risk of unintended genetic alterations, positioning the company’s ABE for long-term therapeutic applications.

Tolerability: Metagenomi’s ABE triplex protein knockdowns exhibited excellent tolerability in cells, with no adverse effects on cell viability, expansion, or changes in stress-related gene expression observed post-editing. Tolerability is especially critical in multiplex editing of primary T-cells, where maintaining cell health is vital for therapeutic efficacy.

About Metagenomi
Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://​metageno​mi​.co.

Cautionary Note Regarding Forward​Looking Statements
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Investor Contact:
Simon Harnest - CIO, SVP Investor Relations
simon@​metagenomi.​co

Media Contact:
Ashlye Hodge - Communications Manager
ashlye@​metagenomi.​co