Metagenomi is a clinical-stage biotechnology company developing gene editing therapies for genetic diseases. The company has no approved products and no product revenue — its business is entirely built around advancing its proprietary gene editing platform toward curative, one-time treatments. Metagenomi's lead wholly-owned program, MGX-001, targets hemophilia A, an inherited bleeding disorder affecting an estimated 26,500 patients in the U.S. MGX-001 aims to permanently insert a functional FVIII gene into liver cells in a single dose, with the goal of providing stable, lifelong clotting factor expression — an improvement over existing treatments, which require repeated administration, and over the only approved gene therapy, which has shown declining efficacy over time. Metagenomi targets an IND filing for MGX-001 in Q4 2026 and first-in-human trials in 2027. The company also has a collaboration with Ionis Pharmaceuticals focused on up to four cardiometabolic gene knockout programs, including transthyretin amyloidosis and hypertriglyceridemia. Under that deal, Ionis leads development and commercialization while Metagenomi provides its editing platform and manufactures key components. The collaboration generated an $80M upfront payment, with additional milestones and royalties tied to future progress. Metagenomi's core technology is a library of over 20,000 gene editing systems discovered through metagenomics, which the company argues offers broader genomic reach and greater specificity than conventional CRISPR-based tools.