Metagenomi Highlights Progress Across Therapeutic Portfolio and Outlines Anticipated Milestones
MGX-001 in Hemophilia A Advances Towards US and Ex-US Regulatory Interactions in 2025
NHP Proof-of-Concept Anticipated for Secreted Protein Deficiency Platform in 2025 Leveraging MGX-001 Editing Approach
On Track for One to Two Development Candidate Nominations in 2025 from Wave 1 Ionis Collaboration Programs Focusing on Cardiometabolic Indications
Cash Runway Anticipated to Support Operating Plans into 2027
EMERYVILLE, Calif., Jan. 16, 2025 (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 provided updates on the successful achievement of critical milestones across its therapeutic development programs and technology platforms in 2024 and anticipated milestones for 2025 and 2026.
“Our vision at Metagenomi is to create curative genetic medicines for patients by harnessing the power of our metagenomics platform,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We made tremendous progress toward this goal in 2024, with accomplishments including substantially advancing MGX-001, our potentially curative development candidate for hemophilia A, as well as leveraging the MGX-001 gene editing system to drive our wholly-owned programs in secreted protein deficiencies. Our partnership with Ionis in large cardiometabolic indications remains on track. We also announced advancements across our gene editing toolbox, including next-generation base editors, ultra-small nucleases and large gene integration systems. Building on this momentum, we look forward to significant additional milestones for Metagenomi in 2025 as we progress our wholly-owned and partnered programs toward the clinic.”
Recent Pipeline Advancement and Corporate Updates
MGX-001 - Hemophilia A Program:
2024 Achievements
- Declared wholly-owned Development Candidate MGX-001, engaged with FDA in initial regulatory discussions and initiated GxP manufacturing activities
- Presented data in an oral presentation at the American Society of Hematology (ASH) 66th Annual Meeting and Exposition in December 2024
- Achieved sustained Factor VIII (FVIII) activity in an ongoing nonhuman primate (NHP) study over more than 16 months of follow-up
- Achieved higher FVIII activity at similar integration rates with bioengineered MGX-001 FVIII construct compared to wild type FVIII construct in rodent studies
2025 and 2026 Anticipated Milestones