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.
- Drug:
- MGX-001
- Announced Date:
- November 10, 2026
- Indication:
- Hemophilia A gene editing investigational therapy
Announcement
Metagenomi Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for MGX-001, its lead, wholly owned program for the treatment of hemophilia A.
Read Announcement- Drug:
- MGX-001
- Announced Date:
- November 11, 2025
- Indication:
- Hemophilia A gene editing investigational therapy
Announcement
Metagenomi, Inc. reported new dose range finding data from the Company's MGX-001 hemophilia A program.
AI Summary
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.
Read Announcement- Drug:
- MGX-001
- Announced Date:
- December 9, 2024
- Indication:
- Hemophilia A gene editing investigational therapy
Announcement
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.
AI Summary
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.
Read Announcement
Metagenomi FDA Events - Frequently Asked Questions
As of now, Metagenomi (MGX) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Metagenomi (MGX) has reported FDA regulatory activity for MGX-001.
The most recent FDA-related event for Metagenomi occurred on November 10, 2026, involving MGX-001. The update was categorized as "FDA Clearance," with the company reporting: "Metagenomi Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for MGX-001, its lead, wholly owned program for the treatment of hemophilia A."
Currently, Metagenomi has one therapy (MGX-001) targeting the following condition: Hemophilia A gene editing investigational therapy.
More FDA Event Resources from MarketBeat
Companies With Recent FDA Events
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:MGX last updated on 11/10/2026 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
