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Metagenomi Presents Updated Preclinical Data in Hemophilia A at American Society of Hematology (ASH) 66th Annual Meeting
Therapeutically relevant levels of Factor VIII (FVIII) activity sustained in ongoing nonhuman primate (NHP) study through more than sixteen months of follow
About this update from Metagenomi Therapeutics, Inc.
[{"type":"text","content":"Therapeutically relevant levels of Factor VIII (FVIII) activity sustained in ongoing nonhuman primate (NHP) study through more than sixteen months of follow up MGX-001 bioengineered FVIII construct exhibited higher FVIII activity at similar integration rates compared to wild type FVIII construct in preclinical studies; program on track for IND filing in 2026 EMERYVILLE, Calif., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today presented updated preclinical NHP data for its hemophilia A program in an oral presentation (link here) at the American Society of Hematology (ASH) 66th Annual Meeting and Exposition in San Diego. “The distinguishing feature of our gene editing approach to hemophilia A, compared to conventional gene therapies, lies in our ability to achieve durable Factor VIII activity levels through precise in vivo integration of the FVIII gene. Today, we shared updated preclinical data demonstrating durable FVIII activity over 16 months in an ongoing NHP study. Additionally, we are pleased to highlight our lead development candidate, MGX-001, which uses a B domain deleted bioengineered FVIII construct, achieved higher levels of FVIII activity versus wild type FVIII, with preclinical evidence of durable FVIII activity levels. Together, these studies help support proof-of-concept as we progress MGX-001 toward the clinic. MGX-001 represents a potentially one-time curative treatment for both adults and children, with the goal to change the treatment paradigm for patients living with hemophilia A,” said Brian C. Thomas, PhD, CEO and Founder of Metagenomi. NHP durability study update: In a preclinical study, a cynomolgus version of the FVIII gene (cFVIII), used to avoid the confounding effects of anti-human FVIII antibodies, was administered to three NHPs via AAV at a dose of 2.0E13 vg/kg. Five weeks later, each NHP was administered an LNP at a dose of 1.0 mg/kg, delivering the MG29-1 nuclease mRNA and associated guide RNA. Each animal received only a single dose of dexamethasone prior to the AAV and LNP doses. Plasma was collected and assayed for safety parameters and FVIII activity. Data was generated over 16 months and the study remains ongoing. Results of the stu...