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

(NASDAQ:MGX) Metagenomi Therapeutics, Inc. announced that Dr. Alan Brooks, Senior Vice President of Research, will deliver an oral presentation on preclinical data supporting MGX-001 at the 68th American Society of Hematology (ASH) Annual Meeting. The presentation, titled 'In vivo targeted FVIII gene integration demonstrating pre-clinical safety, efficacy and durability supporting development of a one-time therapy for hemophilia A', will take place on Monday, December 14, 2026, at 2:45pm CT. The abstract number for the presentation is 952 and it is part of the session 'Gene Therapies: Beyond Transplant: The Expanding Role of Gene Therapy in Hematology'. MGX-001 is Metagenomi Therapeutics' lead, wholly-owned development program in hemophilia A. The company states that 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. The company highlights that MGX-001 has demonstrated a preclinical profile with best-in-class treatment potential, including targeted genome editing and durable gene expression in a one-time treatment. The company plans to initiate a global clinical trial of MGX-001 in 2027, subject to regulatory clearance. Jian Irish, President and CEO, stated that the preclinical data supports the intent to initiate a global clinical trial in 2027, subject to regulatory approval. The company describes MGX-001 as a novel approach to treating hemophilia A that is mechanistically distinct from prior gene therapy for this disease. The company claims that MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children. The company is also pursuing indications leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. Metagenomi Therapeutics focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. The company was founded on the science of metagenomics 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.