NEWS
Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months
Metagenomi (Nasdaq: MGX) has announced promising 12-month durability data for its lead hemophilia A program, MGX-001.
The preclinical study in nonhuman primates (NHPs) demonstrated durable Factor VIII (FVIII) activity levels through twelve months, with two NHPs showing normal/near-normal levels (82% and 41%) and one in the mild hemophilia range (9%).
The treatment, involving a single dose of AAV followed by an LNP containing Metagenomi's novel nuclease, was generally well-tolerated.
Key findings include:
Consistent FVIII activity levels from 3-6 months to 9-12 months
Gene integration in liver biopsies correlating with FVIII activity
Program on track for IND filing in 2026
Metagenomi aims to provide a one-time, curative treatment for adults and children with hemophilia A and plans to leverage this platform for other secreted protein disorders.
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