Metagenomi

Metagenomi introduces MG119-28 in a study in Nature Structural & Molecular Biology — a compact Cas12f nuclease that the researchers report shows faster DNA cleavage and improved editing efficiency due to greater protein stability and a more stable guide RNA. The study, conducted with the University of Texas at Austin, builds on proof-of-concept data that demonstrated a 64% reduction of the Atxn2 protein in mice after AAV administration. The company is evaluating applications beyond hepatic indications, while the clinically active biotech, with a market value of $54 million, continues to burn cash and carries the typical development risks. » Altro su de.investing.com

Metagenomi has completed its rebranding and is prioritizing preclinical in‑vivo therapeutics, foremost the hemophilia A program MGX-001, which the company says showed curative factor VIII activity in non‑human primates without identifiable off‑target effects. After a pre‑IND meeting, Metagenomi plans to file IND/CTA applications in Q4 2026. The company expects financial runway into Q4 2027, reports more cash than debt but continues to burn cash; analysts see price targets of $7–12. » Altro su de.investing.com

Metagenomi reports that its experimental gene therapy MGX-001 demonstrated therapeutically relevant factor VIII activity in a dose‑finding study in non‑human primates; five of six dose cohorts achieved significant results and the dose intended for clinical use produced an average of 49% FVIII activity (range 29.3–59.5%). The treatment was generally well tolerated, with transient liver enzyme elevations only at the highest LNP dose. Metagenomi plans a pre‑IND meeting in Q4 2025 and aims to file IND/CTA applications by the end of 2026, while undergoing a restructuring that will cut 25% of its workforce. The company is trading at a market capitalization of USD 80.7 million; an analysis by InvestingPro rates Metagenomi as undervalued despite a lack of profitability. » Altro su de.investing.com