FDA Events for Metagenomi (MGX)
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:
- 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- Drug:
- MGX-001
- Announced Date:
- September 3, 2024
- Indication:
- Hemophilia A gene editing investigational therapy
Announcement
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.
AI Summary
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.
Read Announcement- Drug:
- MGX-001
- Announced Date:
- September 3, 2024
- Indication:
- Hemophilia A gene editing investigational therapy
Announcement
Metagenomi, Inc. announced that Program on track for IND filing in 2026
AI Summary
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.
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 December 9, 2024, involving MGX-001. The update was categorized as "Data Presentation," with the company reporting: "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."
Currently, Metagenomi has one therapy (MGX-001) targeting the following condition: Hemophilia A gene editing investigational therapy.
More FDA Event Resources from MarketBeat
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
This page (NASDAQ:MGX) was last updated on 7/12/2025 by MarketBeat.com Staff
