Core Insights - Metagenomi's MGX-001 has shown curative factor VIII (FVIII) activity in non-human primates, indicating its potential as a best-in-class treatment for hemophilia A [1][2] - The company plans to submit investigational new drug (IND) and clinical trial application (CTA) submissions by the end of 2026, following a pre-IND regulatory meeting expected in Q4 2025 [1][2] Study Design and Results - The preclinical study involved administering a single dose of AAV containing a B-domain deleted human FVIII gene to 24 non-human primates across six dose cohorts, with doses ranging from 5.0e11 vg/kg to 4.0e13 vg/kg [2][3] - Therapeutically relevant FVIII activity was achieved in the five highest AAV doses, with average FVIII activity ranging from 49% to 81% at a fixed LNP dose of 0.6 mg/kg [4] - The treatment was well tolerated, with no significant elevation of liver enzymes observed, except for transient elevations in the highest LNP dose [4] Implications for Hemophilia A Treatment - MGX-001 represents a potential paradigm shift in hemophilia A treatment, enabling endogenous production of FVIII and potentially providing a lifelong cure [4][5] - The prevalence of hemophilia A is estimated at up to 26,500 patients in the US and over 500,000 globally, highlighting the significant market potential for effective treatments [5] Company Overview - Metagenomi is focused on in vivo genome editing to create curative genetic medicines, leveraging proprietary technologies to address genetic mutations [6][7] - The company aims to expand its pipeline by developing treatments for other secreted protein deficiencies and cardiometabolic diseases [7]