Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Investor Presentation (May 2026)
24 slides from Metagenomi Therapeutics's investor presentation, May 11, 2026
Slide 1: Precision gene editing designed to deliver durable, curative medicines
Precision gene editing designed to deliver durable, curative medicines Hemophilia A lead program | IND 2026 Corporate Presentation May 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 An in vivo CRISPR gene editing company capitalizing on its proprietary technologies to create curative genetic medicines Focusing on lead program MGX-001 in hemophilia A advancing to the clinic Expanding indications l
Slide 4: Differentiated gene editing beyond CRISPR/Cas9
20,000 + Differentiated gene editing beyond CRISPR/Cas9 Signature editing systems from Metagenomi's database High specificity Precise editing Multiplexed editing Multi-gene capability Durable integration Large gene insertion Broad targeting Expanded genome access Proprietary CRISPR genome editing We
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 Large Gene Integration for Protein Replacement Gene editing in the presence of DNA templates to allow for site-specific gene int
Slide 6: Focused pipeline anchored by differentiated lead program
Focused pipeline anchored by differentiated lead program Protein replacement via gene insertion Protein reduction via gene knockout ALB MGX-001 Hemophilia A Editing platform: Editing target: Discovery Lead optimization IND-enabling Clinical Undisclosed Undisclosed TTR AGT APOC3 Undisclosed Cardiomet
Slide 7: MGX-001 development roadmap to clinic
NOW 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 I
Slide 8: MGX-001 - designed to deliver a durable, one-time treatment for Hemophilia A
MGX-001 - designed to deliver a durable, one-time treatment for Hemophilia A
Slide 9: Hemophilia A: large, validated market still lacking a durable cure
Hemophilia A: large, validated market still lacking a durable cure Hemophilia A is the most common X-linked inherited and de novo bleeding disorder, largely affecting males. Caused by variety of mutations in the Factor VIII (FVIII) gene leading to loss of functional FVIII protein. Current Standard o
Slide 10: Hemophilia A is an ideal indication for genome editing approach
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 ideal curative approach for Hemophili
Slide 11: Compelling preclinical profile across efficacy, durability, and safety
Compelling preclinical profile achieved across efficacy, durability, and safety Extensive and supportive preclinical data set FVIII activity achieved in curative range with clear dose response in NHPs Durable FVIII activity over approximately 19-month study in NHPs Encouraging safety profile with si
Slide 12: Curative FVIII range achieved
Curative FVIII range achieved Dose range finding study in NHP identified minimum and optimal efficacious doses Normal FVIII range 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
Slide 13: Durable FVIII expression demonstrated ~19 months
Durable FVIII expression demonstrated ~19 months NHP durability study: Stable expression over ~19 months Plasma FVIII activity levels unchanged between 3-6 months and 12-19 months and correlate to integration: FVIII activity values are the mean and standard deviation of at least 3 independent assay
Slide 14: No genotoxicity observed
No genotoxicity observed Discovery of potential off-target sites No validated off-target sites observed 2. Biochemical off-target discovery: No potential off-targets were discovered in cell-based assays. Development candidate Genome integrity maintained as observed via off-target editing and AAV int
Slide 15: MGX-001: leveraging natural promoter through FVIII gene integration
MGX-001: leveraging natural promoter through FVIII gene integration 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 AAV delivers FVIII gene (donor DNA): Promoterless FVIII g
Slide 16: Our goal: To enable a new standard of care for hemophilia A
Our goal: To enable a new standard of care for hemophilia A Potential to deliver a durable cure for both adult and pediatric patients with hemophilia A Designed to enable endogenous FVIII expression for hemostatic regulation Compelling pre-clinical data Curative FVIII activity Durable FVIII expressi
Slide 17: Expanding applications of site-specific large gene integration system
Expanding applications of site-specific large gene integration system
Slide 18: Leverage site-specific large gene integration across additional indications
Leverage site-specific large gene integration across additional indications Expanding MGX-001 site-specific gene integration system into additional therapeutic targets Normal circulating levels of target protein achieved in secreted protein disorder mice with multiple construct designs LNP delivers
Slide 19: Proof-of-concept demonstrated in additional disease models
Animal 1001 Animal 1002 Animal 1003 Proof-of-concept demonstrated in additional disease models N=3 AAV LNP Baseline Step 1 Step 2 In-life, study Ongoing at 46 days AAV dose LNP dose (vg/kg) (mg/kg) 1.0 x 1013 1.0 1. Lane DA, Bayston T, Olds RJ et al. Antithrombin mutation database: 2nd (1997) update
Slide 20: Broaden therapeutic potential with transformative gene editing
Broaden therapeutic potential with transformative gene editing
Slide 21: 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 delivery2,3 Four-plex knockdown and dua
Slide 22: Strategic partnership expands reach into large cardiometabolic markets
Strategic partnership expands reach into large cardiometabolic markets TTR AGT APOC3 Undisclosed Current indications: MGX's in vivo genome editing complements Ionis leadership in cardiometabolic space 4 targets: two co-development and co-commercialization options Multibillion dollar TAM
Slide 23: Building a leading gene editing company focused on cures
Building a leading gene editing company focused on cures Broad and differentiated library of proprietary gene editing technologies representing a significant long-term value driver Advancing MGX-001 for hemophilia A with clear development path and well-defined clinical and regulatory endpoints Exten
Slide 24: Thank you
Thank you apls Metagenomi THERAPEUTICS
Metagenomi Therapeutics — Investor Presentation (May 2026) on 6ix