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Metagenomi Presents Highly Specific and Efficient Genome Editing Tools at Nature Conference “RNA at the Bench and Bedside IV”

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Metagenomi (Nasdaq: MGX) presented data demonstrating the precision and safety of its gene editing tools at the Nature Conference: RNA at the Bench and Bedside IV. The company highlighted two key developments: MGX-001, their hemophilia A candidate using the MG29-1 nuclease, showed no identifiable off-target editing and no evidence of translocations in primary human hepatocytes. Additionally, their Adenine Base Editor (ABE) for ex vivo cell therapy demonstrated no detectable translocations and no significant genomic base composition differences in primary T-cells compared to unedited cells.

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Positive

  • MGX-001 demonstrated zero off-target editing in genomic assays
  • MG29-1 nuclease showed no evidence of translocations in human hepatocytes
  • Adenine Base Editor exhibited no detectable translocations in T-cells

Negative

  • None.

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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://​metageno​mi​.co.

Cautionary Note Regarding Forward‐​Looking Statements

This press release contains ​“forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as ​“anticipate,” ​“believe,” ​“could,” ​“estimate,” ​“expect,” ​“goal,” ​“intend,” ​“look forward to,” ​“may,” ​“plan,” ​“potential,” ​“predict,” ​“project,” ​“should,” ​“will,” ​“would” and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in ​“Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Contact:
Simon Harnest - CIO, SVP Investor Relations
IR@​metagenomi.​co


FAQ

What are the key safety findings for Metagenomi's MGX-001 hemophilia A treatment?

MGX-001, using the MG29-1 nuclease, showed no identifiable off-target editing and no evidence of translocations in primary human hepatocytes, demonstrating strong safety characteristics.

How effective is Metagenomi's (MGX) Adenine Base Editor in T-cell editing?

Metagenomi's ABE demonstrated high precision with no detectable translocations and no significant genomic base composition differences in primary T-cells compared to unedited cells.

What therapeutic applications is Metagenomi (MGX) developing with its gene editing tools?

Metagenomi is developing tools for both in vivo applications, such as MGX-001 for hemophilia A, and ex vivo cell therapy applications using their Adenine Base Editor technology.

What technology platform does Metagenomi (MGX) use for discovering gene editing tools?

Metagenomi discovers its next-generation nucleases and ABEs through their proprietary metagenomics platform, which is used to develop tools for therapeutic applications.
Metagenomi Therapeutics, Inc

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Biotechnology
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