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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 68th 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/.