MGX-001 / Metagenomi - LARVOL DELTA

Metagenomi Therapeutics advances MGX-001 to IND submission with $120.7M cash runway through Q4 2027 (TradingView) - "Durability shown in NHPs: stable FVIII expression over ~19 months with dose-response and integration correlation; Off-target assessment in primary human hepatocytes found no off-target edits across 623 tested sites, including supratherapeutic doses; Pre-IND interaction with FDA completed Dec 2025; IND submission targeted Q4 2026 and first patient dosing planned in 2027."

IND • Preclinical • Hemophilia A

Second Quarter 2026 Updates: MGX-001 - Hemophilia A Program (GlobeNewswire) - "(i) IND-enabling studies for MGX-001 are expected to be complete by the end of the third quarter of 2026, including GLP toxicity. The Company remains on track to submit an IND application in the fourth quarter of 2026 for the global clinical program, and subject to regulatory clearance, initiate clinical trials in 2027; (ii) Two Metagenomi scientific posters received Top Poster Awards at the 2026 International Society on Thrombosis and Haemostasias (ISTH) Congress in Paris, France, including one highlighting extensive non-human primate preclinical data supporting MGX-001."

IND • New trial • Preclinical • Hemophilia A

MGX-001 - Hemophilia A Program (GlobeNewswire) - "Completed a pre-IND meeting for MGX-001 and remain on track for regulatory submission to advance global clinical program, including an IND in the fourth quarter of 2026, and subject to regulatory clearance, initiate clinical trials in 2027."

FDA event • IND • New trial • Hemophilia A

2026 Anticipated Milestones (The Manila Times) - "Submit IND/CTA in 4Q 2026 to advance MGX-001 into first-in-human studies in 2027."

IND • Hemophilia A

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