Metagenomi reports preclinical proof of concept for MGX-001 in hemophilia A
MGX•Metagenomi Therapeutics reported preclinical proof of concept for MGX-001, a potential one-time treatment for hemophilia A, and plans a global clinical trial in 2027, subject to regulatory clearance.
1. Preclinical results
Metagenomi Therapeutics reported preclinical proof of concept for MGX-001, designed to insert a Factor VIII gene into the albumin locus for sustained Factor VIII production. The company plans to present preclinical efficacy, safety and durability data on December 14, 2026, at the American Society of Hematology meeting.
