MGX-001, utilizing a highly specific and efficient MG29-1 nuclease, exhibits no identifiable off-target editing
MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes
Metagenomi Adenine Base Editor (ABE) demonstrates no detectable translocations and no significant genomic base composition differences in primary T-cells
EMERYVILLE, Calif., Dec. 11, 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 talk titled "Specific and efficient genome editing with metagenomics-derived tools for in vivo and ex vivo therapeutic applications" at the Nature Conference: RNA at the Bench and Bedside IV.
"We believe the value proposition for single-dose gene editing therapies requires exquisite specificity characterization to ensure safety and efficacy. Today's presentation highlights the precision of Metagenomi's next-generation nucleases and ABEs, discovered through the company's proprietary metagenomics platform and tailored for both in vivo and ex vivo therapeutic applications," said Alan Brooks, SVP and Head of Preclinical. "MGX-001, Metagenomi's development candidate for hemophilia A, which utilizes the novel nuclease MG29-1, exhibits no identifiable off-target editing using a series of orthogonal assays employed to evaluate potential off-target sites in the genome. The MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes. For Metagenomi's novel next-generation ABE for ex vivo cell therapy indications via multiplex editing, the data showed no detectable translocations and no significant genomic base composition differences in primary T-cells when compared to unedited cells. These examples demonstrate our strong capabilities in developing highly specific next-generation gene editing tools and support the company's ability to potentially progress these systems toward the clinic for the benefit of patients."
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 (CAST)). 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://metagenomi.co.
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Contact:
Simon Harnest - CIO, SVP Investor Relations
IR@metagenomi.co
