Metagenomi develops genome editing tools and genetic medicines by tapping natural environmental genomes. It sources genetic material from diverse ecosystems (metagenomics), sequences it, and engineers promising proteins into genome editing tools. The tools are designed to be efficient, specific, and targetable, and are advanced using artificial intelligence and high-throughput screening to sift billions of proteins quickly. The main revenue pathway is likely the sale of approved genetic medicines to healthcare providers and patients. The key differentiator is leveraging naturally evolved proteins discovered from unexplored environments, combined with AI-assisted discovery and rapid screening to identify strong genome editors. The company’s goal is to create potentially curative therapeutics for a range of diseases through gene editing.
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Industries
Company Size
51-200
Company Stage
IPO
Headquarters
Emeryville, CA
Founded
2016
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Metagenomi Therapeutics to present preclinical data supporting MGX-001, a potential one-time therapy for hemophilia A, in oral presentation at 68th American Society of Hematology Annual Meeting. MGX-001 data establish preclinical proof-of-concept in hemophilia A On track to initiate global clinical trial in 2027 EMERYVILLE, Calif., Nov. 04, 2026 (GLOBE NEWSWIRE) - Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the "Company"), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today announced that Dr. Alan Brooks, Senior Vice President of Research at the Company, will deliver an oral presentation on preclinical data supporting MGX-001 at the 68th American Society of Hematology (ASH) Annual Meeting in New Orleans, Louisiana. The presentation will highlight preclinical data on MGX-001, including the Company's most recent efficacy and safety results. MGX-001 is the Company's lead program in hemophilia A, and, subject to regulatory clearance, the Company plans to initiate a global clinical trial in 2027. Presentation details are as follows: Title: In vivo targeted FVIII gene integration demonstrating pre-clinical safety, efficacy and durability supporting development of a one-time therapy for hemophilia A Abstract: 952 Session: Gene Therapies: Beyond Transplant: The Expanding Role of Gene Therapy in Hematology Presentation Time: Monday, December 14, 2026, 2:45pm CT "Presenting MGX-001 in an oral session at ASH puts our preclinical data in front of the hematology community that understands the potential of our significantly differentiated profile for a one-time treatment in hemophilia A. The totality of the MGX-001 preclinical data supports our intent to initiate a global clinical trial in 2027 subject to regulatory approval," said Jian Irish, President and CEO of Metagenomi Therapeutics. "MGX-001 is designed to insert a Factor VIII gene site-specifically in the albumin locus to drive sustained production of Factor VIII from a single treatment, a novel approach to treating hemophilia A that is mechanistically distinct from prior gene therapy for this disease. The data we will present on efficacy, durability and safety reinforces our conviction that MGX-001 has the potential to free people with hemophilia A, including both adults and children, from a lifetime of treatment burden and the risk of bleeding events and joint damage that remain with currently available therapies." About Metagenomi Therapeutics Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel CRISPR gene-editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, insertion and correction. MGX-001, the Company's lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile with best-in-class treatment potential, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children, potentially enabling a new standard of care for the treatment of hemophilia A. The Company is also currently pursuing indications leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.co/. Media Contact: Michael Fitzhugh LifeSci Communications [email protected]
Metagenomi Therapeutics, Inc. to present at the Wells Fargo 21st Annual Healthcare Conference. MWN-AI** Summary. Metagenomi Therapeutics, Inc. (Nasdaq: MGX) is a pioneering in vivo genome editing firm that specializes in developing curative genetic medicines by leveraging its proprietary technologies. Founded on the principles of metagenomics - an exploration of genetic materials from natural environments - the company is advancing a range of innovative CRISPR gene-editing tools aimed at rectifying a variety of genetic mutations in the human genome. Under the leadership of President and CEO Jian Irish, Ph.D., M.B.A., Metagenomi focuses on developing high-value programs targeting diseases with well-established biology and clear regulatory pathways. The company's flagship development program, MGX-001, aims to treat hemophilia A and has shown promising preclinical results, offering the potential for best-in-class treatment. This one-time therapy is designed for targeted genome editing and sustained gene expression, promising lifelong protection from bleeding events and joint damage in patients. Metagenomi's strategic focus also includes expanding its pipeline through its proprietary genetic editing technologies, which encompass site-specific deletion, insertion, and correction. In addition to hemophilia A, the company is exploring other indications using the MGX-001 genome integration system and is developing partnerships to address cardiometabolic diseases. Metagenomi is set to participate in a fireside chat at the Wells Fargo 21st Annual Healthcare Conference in Boston, Massachusetts, on September 10, 2026. The presentation will be available for viewing on the company's website. As Metagenomi continues to innovate within the genetic medicine landscape, it aims to establish a new standard of care while providing transformative solutions for patients affected by genetic disorders. For more information, please visit their official website at https://metagenomi.co/. MWN-AI** Analysis. Metagenomi Therapeutics, Inc. (Nasdaq: MGX) is poised for significant attention as it presents at the Wells Fargo 21st Annual Healthcare Conference on September 10, 2026. As a pioneering company in in vivo genome editing, Metagenomi is leveraging its proprietary CRISPR technologies to develop curative genetic therapies, positioning itself as a strong player in the rapidly expanding biopharmaceutical sector. The company's upcoming presentation will likely highlight its lead program, MGX-001, which targets hemophilia A. With promising preclinical results suggesting best-in-class potential for durable gene expression through a single treatment, MGX-001 could redefine treatment standards in hemophilia management. The focus on clear regulatory pathways reinforces investor confidence, along with its strategy to expand into other high-value disease areas, including cardiometabolic conditions. Investors should be particularly attentive to the outcomes of ongoing and future trials, as well as any strategic partnerships that could enhance Metagenomi's research capabilities or market reach. The Company's foundation on metagenomics not only differentiates it from traditional genetic medicine firms but also suggests a robust pipeline leveraging innovative gene-editing technologies, which could attract collaborations and partnerships with larger pharmaceutical entities. As with any investment in biotech, risks remain prevalent, particularly around regulatory approvals and the unpredictable nature of clinical trial outcomes. However, if Metagenomi can successfully navigate these challenges, it may provide significant upside potential. In summary, investors looking for exposure to cutting-edge therapeutic modalities may find Metagenomi Therapeutics a compelling prospect, especially post-conference as more clarity on MGX-001's developmental path and the company's strategic initiatives emerge. Watching management's insights during the fireside chat will provide valuable context for future market actions. **MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release. September 01, 2026 08:00:00 am EMERYVILLE, Calif., Sept. 01, 2026 (GLOBE NEWSWIRE) - Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the "Company"), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today announced that Jian Irish, Ph.D., M.B.A., President and Chief Executive Officer of the Company, will participate in a fireside chat at the Wells Fargo 21st Annual Healthcare Conference, being held in Boston, MA, on Thursday, September 10, 2026, at 12:45 p.m. ET. The fireside chat presentation will be made available on the "News & Events" page in the Investors section of the Company's website at https://ir.metagenomi.co/news-events/events. About Metagenomi Therapeutics Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel CRISPR gene-editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, insertion and correction. MGX-001, the Company's lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile with best-in-class treatment potential, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children, potentially enabling a new standard of care for the treatment of hemophilia A. The Company is also currently pursuing indications leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.co/. FAQ**. How does Metagenomi Therapeutics Inc Com MGX plan to leverage its CRISPR gene-editing tools to enhance the efficacy of its pipeline programs beyond hemophilia A? Metagenomi Therapeutics Inc (MGX) plans to leverage its CRISPR gene-editing tools to enhance the efficacy of its pipeline programs by focusing on expanding applications in rare genetic diseases and other therapeutic areas, emphasizing safe and precise gene editing technologies. What specific disease indications is Metagenomi Therapeutics Inc Com MGX targeting next, and how will these align with its clinical development strategy? Metagenomi Therapeutics Inc. is currently focusing on advancing its gene-editing therapies for rare genetic diseases and neurodegenerative disorders, aligning this strategy with its goal of addressing unmet medical needs and driving progression through clinical trials. Can you elaborate on the expected timeline for clinical trials related to the MGX-001 program in hemophilia A and any potential partnership opportunities for Metagenomi Therapeutics Inc Com MGX? As of now, Metagenomi Therapeutics anticipates initiating clinical trials for the MGX-001 program in hemophilia A in late 2024, with potential partnership opportunities likely emerging as they progress through trial phases and validate therapeutic efficacy. How does Metagenomi Therapeutics Inc Com MGX intend to differentiate its gene-editing technologies from competitors in the rapidly evolving landscape of genetic medicine? Metagenomi Therapeutics Inc Com MGX aims to differentiate its gene-editing technologies by focusing on the development of novel, highly specific gene-editing tools that offer greater precision, efficiency, and safety compared to existing solutions in the genetic medicine landscape. **MWN-AI FAQ is based on asking OpenAI questions about Metagenomi Therapeutics Inc Com (NASDAQ: MGX).
Metagenomi Therapeutics has published research in Nature Structural & Molecular Biology detailing MG119-28, a compact CRISPR nuclease with enhanced genome editing efficiency. The study demonstrates that MG119-28 overcomes the low editing efficiency that previously limited compact nucleases from the Cas12f class. The improved performance stems from superior structural stability and more stable guide RNA, enabling faster DNA cleavage. The company previously demonstrated 64% knockdown of Atxn2 protein in mice using a single AAV delivery of MG119-28. The research, conducted with the University of Texas at Austin, supports developing clinically relevant genome editing systems deliverable via AAV to tissues beyond the liver. Metagenomi plans to explore applications in neuromuscular diseases whilst advancing its lead programme, MGX-001, for haemophilia A treatment.
Metagenomi Therapeutics, Inc. to present at the 46th Annual TD Cowen Healthcare Conference. EMERYVILLE, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) - Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the "Company"), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today announced that Jian Irish, Ph.D., M.B.A., President and Chief Executive Officer of the Company, will participate in a fireside chat at the 46th Annual TD Cowen Healthcare Conference, being held in Boston, MA, on Monday, March 2, 2026 at 11:10 a.m. ET. The fireside chat presentation will be made available on the "News & Events" page in the Investors section of the Company's website at https://ir.metagenomi.co/news-events/events. About Metagenomi Therapeutics, Inc. Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, integration and correction. MGX-001, the Company's lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile potentially competitive with best-in-class treatment options, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children with hemophilia A. The Company is also currently pursuing other secreted protein deficiencies leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.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 product development programs, including the timing of and our ability to conduct IND-enabling studies and make regulatory filings such as INDs, expectations regarding MGX-001, including the preclinical profile being potentially competitive with best-in-class treatment options and timing to submit the IND/CTA package, statements regarding the Company's plans to prioritize its preclinical pipeline and potential for value creation and sustainable growth, statements regarding upcoming milestones including the nomination of development candidates, statements concerning the potential of therapies and product candidates, statements concerning the impact of the organizational restructuring, statements concerning our anticipated cash runway, 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 IND submissions and 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 and the current regulatory environment; patent and intellectual property matters; competition; the volatility of capital markets and other adverse macroeconomic factors; as well as other risks described in "Risk Factors," in our most recent Form 10-K and other risk factors set forth from time to time in our filings with the Securities and Exchange Commission made pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. 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. Investor Contacts:
EMERYVILLE, Calif., May 14, 2025 (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 announced the presentation of three abstracts at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place from May 13 - 17, 2025, in New Orleans, Louisiana.
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Industries
Company Size
51-200
Company Stage
IPO
Headquarters
Emeryville, CA
Founded
2016
Find jobs on Simplify and start your career today
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