Metagenomi (MGX) investor relations material

Strategic focus and technology platform

• Leveraging proprietary metagenomics and AI-driven discovery to develop novel in vivo genome editing tools for curative genetic medicines.

• Generated over 20,000 gene editing systems, expanding beyond CRISPR-Cas9 for improved precision and broader genome targeting.

• Compact editing systems compatible with AAV delivery broaden therapeutic reach.

• RNA and DNA mediated integration systems enable programmable gene correction and exon insertion.

• Capital allocation prioritized for programs with high probability of success and near-term value creation.

Lead program MGX-001 and clinical development

• MGX-001 targets hemophilia A via site-specific gene integration in albumin, aiming for a pre-IND FDA meeting in 2025 and IND/CTA filing in Q4 2026.

• Dose range finding study in non-human primates showed curative, dose-dependent factor VIII activity with robust safety margin.

• Durability study demonstrated stable factor VIII expression for up to 19 months with no off-target editing or genotoxicity.

• MGX-001 mechanism enables endogenous factor VIII production, potentially offering a one-time, lifelong therapy for both children and adults.

• Clinical trial design will use fixed AAV dose and LMP dose escalation to simplify development.

Market opportunity and competitive landscape

• Hemophilia A affects 26,500 in the US and 500,000 globally, with current treatments costing $565,000-$750,000 annually.

• Existing therapies include frequent infusions and bispecific mimetics, both with significant patient burden and breakthrough bleeding risk.

• Gene therapy adoption has been limited by efficacy variability, steroid use, and lack of redosing options.

• MGX-001 aims to address these gaps with durable, predictable efficacy and reduced treatment burden.