Metagenomi Therapeutics to Present Preclinical Data Supporting MGX-001, a Potential One-Time Therapy for Hemophilia A, in Oral Presentation at 68th American Society of Hematology Annual Meeting

The planned move into a global clinical trial in 2027 remains subject to regulatory clearance.

Rhea-AI Summary

Metagenomi Therapeutics (Nasdaq: MGX) will present preclinical data supporting its hemophilia A program, MGX-001, at the American Society of Hematology meeting.

The oral presentation is scheduled for December 14, 2026, at 2:45pm CT in New Orleans. It will cover efficacy, safety and durability findings. The company describes the data as establishing preclinical proof-of-concept and plans to initiate a global clinical trial in 2027, subject to regulatory clearance.

MGX-001 is designed to insert a Factor VIII gene at a specific site in the albumin gene region, with the aim of sustaining Factor VIII production after a single treatment. The program is being developed as a potential one-time therapy; the announcement provides no numerical efficacy or safety results.

2 points · 0 major

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Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate point. Forward-looking: it has not happened yet and may not happen.Global clinical trial initiation is planned for 2027.
• Minor pointMGX-001 preclinical data establish proof-of-concept in hemophilia A, in the company's assessment.

Negative

• Minor pointRegulatory clearance remains a condition for initiating the planned MGX-001 global clinical trial.

Key Figures

Planned global clinical trial initiation: 2027

Planned global clinical trial initiation

2027

MGX-001 for hemophilia A; subject to regulatory clearance

Key Terms

fviii, albumin locus, gene integration

3 terms

fviii medical

"In vivo targeted FVIII gene integration demonstrating pre-clinical safety"

Factor VIII (often written FVIII) is a protein in the blood that helps form clots and stop bleeding; people with low or absent FVIII have a bleeding disorder called hemophilia A. It matters to investors because therapies, drugs, or diagnostics that replace, mimic, or modify FVIII are central to large clinical programs and markets; success or failure in developing FVIII-related treatments can affect regulatory outcomes, revenue forecasts, and a company’s valuation, much like a key ingredient determines a recipe’s outcome.

albumin locus technical

"insert a Factor VIII gene site-specifically in the albumin locus"

The albumin locus is the specific place on a genome that contains the albumin gene and the nearby DNA elements that control its activity; it includes the gene’s coding sequence, promoters, enhancers and often adjacent related genes from the same gene family. In practical use this term identifies the genomic region targeted in laboratory assays, genetic studies or therapeutic approaches, and its exact DNA sequence and regulatory structure can differ between species and individuals.

gene integration technical

"targeted FVIII gene integration demonstrating pre-clinical safety"

Insertion of new genetic material into a cell's chromosomes so the added DNA becomes part of the host genome; this can be done by viral vectors, transposons, or genome‑editing methods that either insert DNA at random locations or at a specific target site using homology‑directed repair or site‑specific nucleases. Gene integration produces durable, heritable expression in dividing cells but can disrupt native genes or regulatory regions (insertional mutagenesis), so it is distinct from transient, non‑integrated delivery of genetic material and is subject to regulatory and safety assessment.

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MGX-001 data establish preclinical proof-of-concept in hemophilia A
On track to initiate global clinical trial in 2027

EMERYVILLE, Calif., Nov. 04, 2026 (GLOBE NEWSWIRE) -- Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the “Company”), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today announced that Dr. Alan Brooks, Senior Vice President of Research at the Company, will deliver an oral presentation on preclinical data supporting MGX-001 at the 68^th American Society of Hematology (ASH) Annual Meeting in New Orleans, Louisiana.

The presentation will highlight preclinical data on MGX-001, including the Company’s most recent efficacy and safety results. MGX-001 is the Company’s lead program in hemophilia A, and, subject to regulatory clearance, the Company plans to initiate a global clinical trial in 2027. Presentation details are as follows:

Title: In vivo targeted FVIII gene integration demonstrating pre-clinical safety, efficacy and durability supporting development of a one-time therapy for hemophilia A
Abstract: 952
Session: Gene Therapies: Beyond Transplant: The Expanding Role of Gene Therapy in Hematology
Presentation Time: Monday, December 14, 2026, 2:45pm CT

“Presenting MGX-001 in an oral session at ASH puts our preclinical data in front of the hematology community that understands the potential of our significantly differentiated profile for a one-time treatment in hemophilia A. The totality of the MGX-001 preclinical data supports our intent to initiate a global clinical trial in 2027 subject to regulatory approval,” said Jian Irish, President and CEO of Metagenomi Therapeutics. “MGX-001 is designed to insert a Factor VIII gene site-specifically in the albumin locus to drive sustained production of Factor VIII from a single treatment, a novel approach to treating hemophilia A that is mechanistically distinct from prior gene therapy for this disease. The data we will present on efficacy, durability and safety reinforces our conviction that MGX-001 has the potential to free people with hemophilia A, including both adults and children, from a lifetime of treatment burden and the risk of bleeding events and joint damage that remain with currently available therapies.”

About Metagenomi Therapeutics

Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel CRISPR gene-editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, insertion and correction.

MGX-001, the Company’s lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile with best-in-class treatment potential, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children, potentially enabling a new standard of care for the treatment of hemophilia A. The Company is also currently pursuing indications leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. For more information, please visit https://metagenomi.co/.

Investor Contact:
Brian Ritchie
LifeSci Advisors
britchie@lifesciadvisors.com

Media Contact:
Michael Fitzhugh
LifeSci Communications
mfitzhugh@lifescicomms.com

FAQ

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What MGX-001 data will Metagenomi present at ASH 2026?

Metagenomi will present preclinical efficacy, safety and durability data supporting MGX-001 for hemophilia A. The oral presentation is scheduled for December 14, 2026, at 2:45pm CT. The company describes the data as establishing preclinical proof-of-concept.

When does Metagenomi plan to start the MGX-001 clinical trial?

Metagenomi plans to initiate a global clinical trial in 2027, subject to regulatory clearance. MGX-001 is its lead program in hemophilia A.