MGX-001 / Metagenomi - LARVOL DELTA

Metagenomi Presents New Preclinical Data from MGX-001 Hemophilia A Program Supporting Advancement into Clinical Development (GlobeNewswire) - "The data demonstrated curative factor VIII (FVIII) activity in non-human primates (NHPs) and informs a clinical dose regimen strategy for a therapy with best-in-class treatment potential. Metagenomi intends to advance MGX-001 into clinical development....At a proposed clinical dose of AAV at 5e12 vg/kg and LNP at 0.6 mg/kg, MGX-001 achieved average FVIII activity of 49% within a range of 29.3% - 59.5%....Pre-IND regulatory meeting expected in Q4 2025 with investigational new drug (IND) and clinical trial application (CTA) submissions expected in 4Q 2026."

FDA event • IND • Preclinical • Hemophilia A

Metagenomi Highlights Progress Across Therapeutic Portfolio and Outlines Anticipated Milestones (GlobeNewswire) - "Recent Pipeline Advancement and Corporate Updates - MGX-001 - Hemophilia A Program:...2025 and 2026 Anticipated Milestones: (i) 2025: Finalize ongoing NHP durability study, continue Investigational New Drug (IND) enabling efforts, and Pre-IND/ ex-US regulatory meetings to support IND/ Clinical Trial Application (CTA) submissions; (ii) 2026: Submit IND/CTA to advance MGX-001 into first-in-human studies."

IND • Preclinical • Regulatory • Hemophilia A

MGX-001 update (GlobeNewswire) - "A separate NHP study designed to support the company’s lead hemophilia A development candidate, MGX-001...demonstrated significantly higher FVIII activity compared to wild type FVIII, despite similar integration frequency between the bioengineered construct and wild type gene. This result suggests the MGX-001 bioengineered construct may enable therapeutic FVIII activity at lower AAV doses with the potential for associated safety benefits. In addition, MGX-001 showed no identifiable off-target editing to-date in a series of orthogonal assays employed to discover and validate potential off-target sites."

Preclinical • Hemophilia A

Metagenomi Reports Business Updates and Third Quarter 2024 Financial Results (GlobeNewswire) - "Metagenomi, Inc...today provided a business update and reported third quarter 2024 financial results....The company initiated manufacturing activities to support IND enabling studies, and MGX-001 remains on track for an IND filing in 2026. Building on the hemophilia A program, the company is advancing additional wholly-owned therapeutic candidates targeting secreted protein disorders, leveraging the MGX-001 editing system with the goal of achieving targeted and durable gene expression."

IND • Preclinical • Genetic Disorders • Hemophilia • Hemophilia A