Metagenomi Therapeutics, Inc.

We are an in vivo genome editing company that uses our own technologies to create genetic medicines. Our company was founded on the science of metagenomics, which is the study of genetic materials from the natural environment. This science helps us discover and develop gene editing tools that may be able to correct any type of genetic mutation in the human genome. We use machine learning and artificial intelligence to improve our gene editing systems. In late 2025, we decided to focus our resources on our lead programs that use our most advanced gene-editing capabilities, aiming for sustainable growth and long-term value. We are concentrating on high-value programs for diseases with well-understood biology and clear clinical development and regulatory paths. MGX-001 is our primary, wholly-owned development program for hemophilia A. In non-human primates, this program has shown targeted genome editing and lasting gene expression from a single treatment, with a profile that could compete with other top treatments. MGX-001 is designed to offer curative, lifelong protection against bleeding and joint damage in adults and children with hemophilia A from one administration. We are also developing treatments for other secreted protein deficiencies using the MGX-001 site-specific genome integration system, and we have partnered assets targeting cardiometabolic diseases. We plan to expand our pipeline using our genetic editing capabilities for site-specific deletion, integration, and correction, with the goal of creating curative therapeutics. We have generated over 20,000 signature gene editing systems from our novel, proprietary metagenomics database.