Metagenomi Therapeutics (MGX) TD Cowen 45th Annual Healthcare Conference summary

Platform and technology strategy

• Leveraging metagenomics and AI to discover and engineer novel genome editing tools from diverse microbial sources.

• Proprietary database enables identification of rare, highly specific nucleases and other editing modalities.

• Toolbox includes nucleases, base editors, RNA-mediated integration systems (RIGS), and CAST for a range of genome modifications.

• Small enzyme size allows for efficient delivery, including packaging into AAVs for targeting organs beyond the liver.

• AI-driven engineering enhances enzyme activity and specificity in human cells.

Pipeline and lead program updates

• Lead program targets hemophilia A using a two-vector gene editing system for factor VIII replacement.

• Preclinical NHP studies show durable factor VIII expression for over 16 months, with activity in the 10%-150% range considered a functional cure.

• Bioengineered factor VIII variant increases activity, enabling lower AAV doses and improved safety.

• IND and CTA filings for the hemophilia A program are targeted for 2026, with GMP manufacturing and GLP studies underway.

• Platform allows rapid adaptation to other indications by swapping payloads and guides.

Partnerships and business development

• Ongoing partnership with Ionis in cardiometabolic diseases, with four programs in early stages and one to two development candidates expected this year.

• Decision to opt in on Ionis programs will depend on progress and strategic fit.

• Active business development strategy includes seeking additional partnerships, especially for large indications or those requiring advanced delivery.

• Preference to retain smaller, manageable indications in-house, while partnering for larger or more complex disease areas.