Metagenomi (MGX) FDA Approvals

Metagenomi's Drug in the FDA Approval Process

This section highlights FDA-related milestones and regulatory updates for drugs developed by Metagenomi (MGX). Over the past two years, Metagenomi has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as MGX-001. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

MGX-001 FDA Regulatory Timeline and Events

MGX-001 is a drug developed by Metagenomi for the following indication: Hemophilia A gene editing investigational therapy. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Data - November 11,2025

• Drug: MGX-001
• Announced Date: November 11, 2025
• Indication: Hemophilia A gene editing investigational therapy

Metagenomi, Inc. reported new dose range finding data from the Company's MGX-001 hemophilia A program.

Metagenomi reported dose range finding results for MGX-001, a one-time genome editing therapy for hemophilia A, showing curative factor VIII (FVIII) activity in non-human primates. In a study of 24 animals across six AAV dose cohorts (5.0e11–4.0e13 vg/kg), each followed by a single LNP dose (0.2, 0.6 or 2.0 mg/kg) delivering the MG29-1 nuclease mRNA and guide RNA, therapeutically relevant FVIII levels were achieved in the five highest AAV dose groups. With LNP fixed at 0.6 mg/kg and AAV from 1.6e12–4e13 vg/kg, cohort averages ranged from 49% to 81% of normal FVIII.

FVIII activity showed clear dependence on both AAV and LNP doses, and no animal exceeded 150% of normal. At a proposed clinical dose (AAV 5e12 vg/kg; LNP 0.6 mg/kg) average FVIII was 49% (range 29.3%–59.5%). The treatment was generally well tolerated, with only transient liver enzyme rises at the highest LNP dose. Orthogonal assays detected no identifiable off‑target editing. Metagenomi says these data support advancing MGX-001 toward clinical development as a potential one‑and‑done curative approach.

Data Presentation - December 9,2024

• Drug: MGX-001
• Announced Date: December 9, 2024
• Indication: Hemophilia A gene editing investigational therapy

Metagenomi, Inc. 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.

Metagenomi, Inc. recently presented updated preclinical data on its hemophilia A program at the American Society of Hematology’s 66th Annual Meeting in San Diego. The company highlighted results from an ongoing nonhuman primate (NHP) study where therapeutically relevant levels of Factor VIII (FVIII) activity were maintained for over 16 months. This study demonstrated that their bioengineered FVIII construct, used in MGX-001, achieved higher FVIII activity compared to the wild type, even with similar gene integration rates. The findings support the proof-of-concept behind their unique gene editing approach, which integrates the FVIII gene in vivo, potentially offering a one-time curative treatment for hemophilia A patients. These encouraging results are a crucial milestone as the company moves forward with MGX-001 toward clinical development, with an IND filing targeted for 2026.

Preclinical Data - September 3,2024

• Drug: MGX-001
• Announced Date: September 3, 2024
• Indication: Hemophilia A gene editing investigational therapy

Metagenomi, Inc. announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company's hemophilia A gene editing investigational therapy, MGX-001.

Metagenomi, Inc. recently announced encouraging data from an ongoing preclinical study focused on its hemophilia A gene editing investigational therapy, MGX-001. The study is designed to provide evidence supporting the potential durability and safety of the therapy as it moves closer to clinical development. Early findings indicate that MGX-001 may offer long-lasting benefits while maintaining a strong safety profile, a critical factor for advancing gene editing treatments in patients with hemophilia A.

This preclinical data is an important milestone for Metagenomi, as it helps build the foundation necessary for future regulatory discussions and clinical trials. The company’s commitment to advancing innovative gene therapies reflects its goal of addressing severe genetic disorders with groundbreaking technology, potentially offering new hope to patients affected by hemophilia A.

Provided Update - September 3,2024

• Drug: MGX-001
• Announced Date: September 3, 2024
• Indication: Hemophilia A gene editing investigational therapy

Metagenomi, Inc. announced that Program on track for IND filing in 2026

Metagenomi, Inc. recently announced that its program is on track for an Investigational New Drug (IND) filing in 2026. This milestone is an important step in the company’s strategy to advance its innovative treatment into clinical trials and ultimately towards market approval. The planned filing with the FDA is a key indicator of Metagenomi’s commitment to developing new therapies and enhancing its product pipeline. It underscores the company’s focus on meeting regulatory requirements and maintaining high standards in drug development. With the IND filing on the horizon, Metagenomi aims to demonstrate the safety and potential efficacy of its treatment, which could provide new options for patients in need. Stakeholders and the scientific community are watching closely as the company prepares to take this critical next step in its journey toward delivering promising medical solutions.