MGX News & Events

Reports Q1 revenue $1.25M, consensus $5.92M. "We remain diligently focused on advancing our core genome-editing technologies, led by our MGX-001 program for hemophilia A, which remains on track for regulatory submission in the fourth quarter of this year and first-in-human studies in 2027," said Jian Irish, President and Chief Executive Officer of Metagenomi Therapeutics. "The promise of our novel technology, most recently highlighted by a Nature publication, in addition to the encouraging preclinical data and continued IND-enabling execution, gives us confidence in our goal to provide patients an option for one-time, curative treatments, beginning with hemophilia A."

Metagenomi Therapeutics announced a publication in Nature Structural & Molecular Biology highlighting the discovery and detailed characterization of MG119-28, a compact CRISPR nuclease with superior editing efficiency relative to previously identified compact nucleases. The publication, titled "Comparative characterization of Cas12f orthologs reveals mechanistic features underlying enhanced genome editing efficiency" highlights the potential of MG119-28 to overcome low editing efficiency in mammalian systems of previously identified compact nucleases from the Cas12f class. "The publication demonstrates that the enhanced editing performance is due to superior structural stability of the protein and inherently more stable guide RNA which enables faster DNA cleavage activity. The structural and mechanistic insights enabled engineering of MG119-28 to further improve activity across multiple target genes," the company said.

"I am excited by the direction we are headed as we enter 2026, led by MGX-001, our wholly owned hemophilia A program. MGX-001 recently demonstrated curative factor VIII activity in non-human primates with clear dose-dependent activity and no identifiable off-target editing, representing a therapy with best-in-class treatment potential," said Jian Irish, CEO. "The study informs our anticipated clinical dose regimen and based on recent regulatory engagement, we feel well positioned to file our IND/CTA submissions before year end 2026 and initiate a first-in-human study in 2027. The recent steps we took to strategically reprioritize our pipeline provide us a strengthened balance sheet with runway anticipated into the fourth quarter of 2027. Looking ahead, our new corporate name represents our focus and commitment to delivering curative genetic medicines to patients and accelerating development of these medicines by strategically deploying our most advanced, proprietary technologies built on our foundational science of metagenomics."