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
Key Highlights
- ➤MGX-001 preclinical data establish proof-of-concept in hemophilia A
- ➤Metagenomi plans global MGX-001 clinical trial in 2027, subject to regulatory clearance
- ➤MGX-001 targets site-specific Factor VIII gene insertion for one-time treatment
- ➤ASH oral presentation scheduled December 14, 2026, highlighting efficacy, safety and durability
Expert Statements
Jian Irish, President and CEO of Metagenomi Therapeutics
“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.”
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.”
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.
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