Metagenomi Therapeutics Presents at Jefferies Healthcare Conference — MGX-001 Hemophilia A IND Targeted Q4 2026 Using Compact AAV-Compatible CRISPR Nuclease

Metagenomi Therapeutics (MGX) CEO Jian Irish, Ph.D., M.B.A., presented a fireside chat at the 2026 Jefferies Global Healthcare Conference in New York on June 4, 2026 at 7:35 a.m. ET. The company highlighted its lead wholly owned program MGX-001 for hemophilia A (factor VIII deficiency, affecting ~400,000 people globally), which uses the company's proprietary compact CRISPR nuclease Al3Cas12f (designated MG119-28) to make a one-time correction to the F8 gene via AAV vector delivery. Key preclinical results: MGX-001 achieved curative factor VIII (FVIII) activity in non-human primates — well above the 50 IU/dL threshold considered curative — with no identifiable off-target editing detected across multiple orthogonal assays. Metagenomi plans to submit IND/CTA applications for MGX-001 in Q4 2026, with first-in-human trials targeted for 2027. The compact nuclease Al3Cas12f was characterized in a publication in Nature Structural & Molecular Biology (April 16, 2026; DOI: 10.1038/s41594-026-01503-5), demonstrating editing efficiency rising from less than 10% to over 80% when delivered via AAV8 vectors — a breakthrough enabling in vivo delivery of large CRISPR machinery in a single AAV capsid. Metagenomi also discussed its cardiometabolic and neuromuscular pipeline programs sharing the same AAV-compatible compact nuclease platform. The company is backed by Bayer AG (strategic investor) and has raised over $200M in venture funding. The Al3Cas12f platform represents a potential step-change in AAV-delivered in vivo CRISPR delivery, addressing the long-standing challenge of fitting Cas9 and its guide RNA into a single AAV capsid (Cas9 exceeds AAV packaging limits; compact nucleases like Cas12f avoid split-intein engineering).