Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Investor Presentation (September 2026)
22 slides from Metagenomi Therapeutics's investor presentation, Sep 22, 2026
Slide 1: Precision gene editing designed to deliver durable, curative medicines
Precision gene editing designed to deliver durable, curative medicines Corporate Presentation September 2026
Slide 2: Forward-looking statements
This presentation includes forward-looking statements, including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this presentation are forward looking statements, including st
Slide 3: A differentiated in vivo gene editing company advancing curative genetic medicines
A differentiated in vivo gene editing company advancing curative genetic medicines 1 Cash includes cash, cash equivalents and available-for-sale marketable securities An in vivo CRISPR gene editing company capitalizing on proprietary technologies to create curative genetic medicines Focusing on lead
Slide 4: Differentiated and proprietary gene editing technologies
20,000 + Differentiated and proprietary gene editing technologies Suite of novel editing tools from Metagenomi’s unique database High specificity Precise editing Multiplexed editing Multi-target capability Durability Long term effectiveness Broad targeting Expanded genome access We improve editing p
Slide 5: A versatile platform designed to address a wide range of genetic diseases
A versatile platform designed to address a wide range of genetic diseases Potentially capable of correcting any type of genetic mutation found anywhere in the human genome Metagenomi Technology Large Gene Integration Protein replacement Gene editing in the presence of DNA templates to allow for site
Slide 6: Focused pipeline anchored by differentiated gene editing technologies
Protein replacement via gene insertion Protein reduction via gene knockout ALB MGX-001 Hemophilia A Undisclosed Undisclosed TTR AGT APOC3 Undisclosed Cardiometabolic Diseases Editing platform: Editing target: Discovery Lead optimization IND-enabling Clinical Indication: Focused pipeline anchored by
Slide 7: MGX-001 - designed to deliver a durable, one-time curative therapeutic for hemophilia A
MGX-001 - designed to deliver a durable, one-time curative therapeutic for hemophilia A
Slide 8: MGX-001: leveraging natural promoter to drive durable Factor VIII production
AAV delivers FVIII gene LNP delivers CRISPR cargo CRISPR cargo forms complex Cut at albumin locus and FVIII gene integrates Albumin promoter drives sustained FVIII production MGX-001: leveraging natural promoter to drive durable Factor VIII (FVIII) production AAV delivers donor DNA: Promoterless FVI
Slide 9: Hemophilia A: potentially life-threatening, blood-clotting disease with high treatment burden
~25,000 patients in the U.S.2 Hemophilia A: potentially life-threatening, blood-clotting disease with high treatment burden - Stonebraker, J. S., et al, 2010. Haemophilia. Vol. 16, pp. 20–32 - CDC Surveillance 2024 - World Federation of Hemophilia Bleeding Disorders Registry, 2023 Hemophilia A is ca
Slide 10: Hemophilia A unmet needs: high treatment burden and lack of durable cure
Hemophilia A unmet needs: high treatment burden and lack of durable cure - ICER. Gene Therapy for Hemophilia B and A: Final Evidence Report. Dec 22, 2022. - Curtis R., et al. Poster presented at: 65th ASH Annual Meeting & Exposition; December 11, 2023; San Diego, CA. Current Standard of Care: Factor
Slide 11: Genome editing offers a potentially curative approach for hemophilia A
Strong advocacy and infrastructure Robust preclinical models and regulatory familiarity Clearly defined target threshold of curative FVIII level & wide safety range Monogenic and well-characterized biology with clear biomarker Genome editing offers a potentially curative approach for hemophilia A Si
Slide 12: Curative FVIII range achieved in preclinical model
Curative FVIII range achieved in preclinical model FVIII activity for each animals is the mean of values on d5, d8, d11 post LNP dosing measured with a capture-chromogenic assay. hFVIII activity was stable from d5 to d11 post LNP. Xyntha was used for the standard curve. Normal FVIII range Developmen
Slide 13: Durable FVIII expression demonstrated ~19 months
Durable FVIII expression demonstrated ~19 months cFVIII NHP durability study: Stable expression over ~19 months Plasma cFVIII activity levels unchanged between 3-6 months and 12-19 months and correlate to integration Cynomolgus FVIII (cFVIII) activity values are the mean and standard deviation of at
Slide 14: Off-target evaluation confirmed gene editing safety profile
Off-target evaluation confirmed gene editing safety profile Discovery of potential off-target sites Identified 623 potential off-target editing sites using industry standard orthogonal methods Development candidate On-target site Evaluation of potential off-target sites In silico prediction In cell
Slide 15: Novel mechanism of action integrates gene directly into chromosome
Novel mechanism of action integrates gene directly into chromosome FVIII integration leveraging albumin promoter to achieve rapid onset of normal FVIII activity level Lower dose of promoterless AAV designed to deliver safety advantage over traditional gene therapy Precise FVIII integration facilitat
Slide 16: MGX-001 development roadmap to clinic
Pre-IND Interaction Complete Dec 2025 FDA meeting — collaborative, favorable outcome Q4 2026 IND Submission Submit IND for first-in-human Phase 1/2 study 2027 First Patient Dosed Global single-arm study; observe initial safety and efficacy within weeks of dosing 2027 and beyond Dose Escalation Ident
Slide 17: Broaden therapeutic potential with transformative gene editing
Broaden therapeutic potential with transformative gene editing
Slide 18: Leverage site-specific large gene integration across additional indications
Leverage site-specific large gene integration across additional indications Potential to substitute therapeutic transgene delivered by MGX-001 system to address additional diseases Achieved normal circulating levels of target protein in an additional disease Minimal target level Increase in Target P
Slide 19: Multiplex editing and compact editors enable next-generation therapies
Multiplex editing and compact editors enable next-generation therapies Combined protein knockdown and CAR knock-in in a single-step for cell therapy applications Compact nucleases and base editors demonstrated high editing efficiency and compatibility with AAV for extrahepatic delivery2,3 Four-plex
Slide 20: Strategic partnership for protein knockdown programs expands reach into cardiometabolic markets
All four targets in Wave 1 are in the lead optimization phase of development and are designed to achieve protein reduction via gene knockout. Strategic partnership for protein knockdown programs expands reach into large cardiometabolic markets TTR AGT APOC3 Undisclosed Current indications: MGX's in
Slide 21: Metagenomi pipeline and expected updates
Metagenomi pipeline and expected updates Protein replacement via gene insertion Protein reduction via gene knockout ALB MGX-001 Hemophilia A Undisclosed Undisclosed TTR AGT APOC3 Undisclosed Cardiometabolic Diseases Editing platform: Editing target: Discovery Lead optimization IND-enabling Clinical
Slide 22: Thank you
Thank you THERAPEUTICS | ple Metagenomi’
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