Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Investor Presentation (January 2026)
20 slides from Metagenomi Therapeutics's investor presentation, Jan 12, 2026
Slide 1: Corporate Presentation January 2026
Corporate Presentation January 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: From novel genome editing systems to curative therapies
From novel genome editing systems to curative therapies An in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines Focusing on wholly owned programs in hemophilia A and secreted protein disorders, and partnered assets targeting cardiometabolic
Slide 4: Overcoming limitations of CRISPR/Cas9 Nucleases
Overcoming limitations of CRISPR/Cas9 Nucleases Enabling gene knockdown, large deletions and large gene integration RIGS Enabling gene correction CAST Enabling large gene insertion Higher specificity No off-target cuts or translocations seen in genome-wide screens Multiplexed editing Using multiple
Slide 5: Progressing wholly-owned and partnered programs toward the clinic
Progressing wholly-owned and partnered programs toward the clinic 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 secreted protein diseases
Slide 6: Focused on generating clinical data in 2027
Focused on generating clinical data in 2027 2026 2027 2025 MGX-001 Hemophilia A Secreted protein deficiencies Cardiometabolic programs Other therapeutic programs Complete NHP durability and dose range finding (DRF) studies Disclose lead indication for secreted protein deficiency platform File IND an
Slide 7: MGX-001 in hemophilia A advancing toward clinic
MGX-001 in hemophilia A advancing toward clinic Demonstrated curative Factor VIII activity with best-in-class treatment potential Dose-dependent efficacy of both AAV and LNP Therapeutically relevant FVIII activity in each animal treated in all but the lowest dose Data informs clinical dose regimen s
Slide 8: Hemophilia A: a validated target waiting for a durable cure
Hemophilia A: a validated target waiting for 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 SOC: ~26,500 pati
Slide 9: MGX-001 mechanism leverages natural promoter
DNA FVIII protein DNA DNA sgRNA promoter MG29-1 RNP complex LNP DNA sgRNA MG29-1 AAV cell nucleus DNA MGX-001 mechanism leverages natural promoter AAV delivers promoterless FVIII gene. AAV enters hepatocytes and the donor FVIII cassette localizes to the nucleus. LNP delivers MG29-1 nuclease mRNA + g
Slide 10: NHP study designs
Animal 1001 Animal 1002 Animal 1003* NHP study designs N=3 Durability study AAV LNP Baseline Step 1 Step 2 Dose range finding study Human FVIII Pre-development candidate Development candidate Study duration: 19 months NHP FVIII N=24 Study duration 3 weeks FVIII evaluation: days 5, 8, 11 Group 1: N=4
Slide 11: Dose dependent FVIII activity in NHP
Dose dependent FVIII activity in NHP: minimally efficacious and clinically relevant doses identified bleeding risk normal FVIII range thrombosis risk FVIII activity for each animals is the mean of values on d5, d8, d11 post LNP measured with a capture-chromogenic assay. hFVIII activity was stable fr
Slide 12: Durable, therapeutic levels of FVIII achieved in NHP
Durable, therapeutic levels of FVIII achieved in NHP NHP durability study: 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 runs with each sample run in a
Slide 13: No genotoxicity observed with MGX-001
No genotoxicity observed with MGX-001 Discovery of potential off-target sites No validated off-target sites observed 2. Biochemical off-target discovery: 3. In cell off-target discovery: No potential off-targets were found. No potential off-targets were discovered in cell-based assays. No validated
Slide 14: 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 De-risked and clear opportunity for genome editing in hemophilia A propri
Slide 15: Potential cure for adults and children living with hemophilia A
Potential cure for adults and children living with hemophilia A Designed to enable the body's own ability to produce FVIII Expected durable FVIII activity via gene integration Established regulatory and clinical pathways for advancement to pivotal study Our goal: to provide patients with a hemophili
Slide 16: Beyond Hem A: pursuing secreted protein diseases
Beyond Hem A: pursuing secreted protein diseases with site-specific large gene insertion Expanding MGX-001 site-specific gene integration system into additional secreted protein deficiency therapeutics Normal circulating levels of target protein achieved in mice with multiple construct designs LNP d
Slide 17: In vivo PoC in AT-III achieved with MGX-001
Animal 1001 Animal 1002 Animal 1003 In vivo PoC in AT-III achieved with MGX-001 site-specific large gene integration system 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 mutatio
Slide 18: Pursuing cardiometabolic indications in partnership
Pursuing cardiometabolic indications in partnership 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 19: Team
Team Jian Irish, PhD, MBA President and CEO Pamela Wapnick, MBA CFO Katalin Kauser, PhD, MD, Sc.D SVP, Translational Biology Board of Directors: Executive Leadership: Willard H. Dere, MD Professor Emeritus, Internal Medicine, University of Utah Eric Bjerkholt, MBA CFO, Mirum Pharmaceuticals, Inc. Jü
Slide 20: Thank you
Thank you apls Metagenomi T HIE RA) EE UNT ICes