Metagenomi Therapeutics (MGX) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Scientific and technology platform updates

• Leveraging metagenomics and AI to discover novel, highly efficient, and specific gene-editing systems from uncultivated microbes worldwide.

• Developed a proprietary database and high-throughput screening to identify programmable CRISPR nucleases, base editors, and prime editors with unique properties.

• Created ultra-small gene-editing enzymes, enabling new delivery methods such as AAV for therapeutic applications.

• Demonstrated multiplex editing and robust gene-editing enzyme development using AI and ancestral state reconstruction.

• Published findings on ultra-small nucleases and their mechanisms in Nature Communications.

Pipeline and program progress

• Lead program in hemophilia A (HemA) advanced with successful long-term durability studies in non-human primates and positive regulatory engagement with the FDA.

• Achieved in vivo proof of concept for secreted protein deficiency disorders using the MGX-001 platform, with >200% normal activity in preclinical models.

• Partnership with Ionis advanced four cardiometabolic targets into lead optimization, all with in vivo rodent proof of concept.

• Ongoing optimization of gene-editing components to increase efficacy and reduce AAV dose requirements.

• Demonstrated high specificity and minimal off-target activity in gene-editing assays for HemA platform.

Strategic priorities and future milestones

• 2024 milestones include completion of NHP durability study, pre-IND and ex-U.S. regulatory meetings for HemA, and disclosure of lead indications for secreted protein disorder platform.

• Plan to file IND/CTA for HemA in 2026, with parallel U.S. and ex-U.S. clinical site activation to accelerate proof of concept.

• Ionis partnership to nominate 1-2 development candidates in 2024 and expand into additional indications in 2026.

• Continued innovation in gene-editing technology and expansion of the platform to address a broad range of genetic diseases.

• Announced addition of Eric Bjerkholt, an experienced biotech executive, to the board.