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Metagenomi outlines anticipated 2025 milestones
The Fly

Metagenomi outlines anticipated 2025 milestones

“Our vision at Metagenomi (MGX) is to create curative genetic medicines for patients by harnessing the power of our metagenomics platform,” said Brian Thomas, CEO. “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.” MGX-001 – Hemophilia A Program: 2025 and 2026 Anticipated Milestones: 2025: Finalize ongoing NHP durability study, continue Investigational New Drug enabling efforts, and Pre-IND/ ex-US regulatory meetings to support IND/ Clinical Trial Application submissions; 2026: Submit IND/CTA to advance MGX-001 into first-in-human studies. Secreted Protein Deficiencies: 2025 and 2026 Anticipated Milestones: 2025: Demonstrate NHP proof-of-concept for lead secreted protein deficiency target; 2026: Nominate Development Candidate for lead secreted protein deficiency target. Cardiometabolic Programs: 2025 and 2026 Anticipated Milestones: 2025: Nominate one to two Development Candidates from the Wave 1 Ionis collaboration development programs and disclose remaining therapeutic indications in large cardiometabolic indications for the remaining development programs 2026: Initiate IND-enabling activities for the Development Candidates nominated in 2025 and nominate additional Development Candidates from the remaining Wave 1 targets. Technology Development: 2025 and 2026 Anticipated Milestones: Continue to advance early-stage pipeline for multiple future IND filings

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