GlobeNewswire·Nov 10, 2026, 4:05 PM ET

Metagenomi Therapeutics Announces FDA Clearance of Investigational New Drug Application for MGX-001 and Reports Third Quarter 2026 Financial Results

FDA cleared Metagenomi Therapeutics' Investigational New Drug application for MGX-001, a one-time genome editing treatment for hemophilia A.
The company plans to initiate the global Phase 1/2 INTEGR8 trial in the first half of 2027 with initial clinical data expected in the second half of 2027.
MGX-001 utilizes site-specific integration of the Factor VIII gene into the albumin locus to enable durable, endogenous production, differing from prior episomal gene therapies.
Preclinical data in non-human primates showed FVIII activity within the normal physiologic range, durable expression over 19 months, and no detectable off-target editing.
Metagenomi reported $101.7 million in cash, cash equivalents, and marketable securities as of September 30, 2026, with operations funded into 2028.
Research and development expenses for the quarter ended September 30, 2026, were $17.5 million, down from $25.3 million in the same period in 2025.
General and administrative expenses totaled $6.9 million for the quarter, including a $1.2 million one-time net litigation settlement expense.
Additional preclinical efficacy and safety data for MGX-001 will be presented at the 68th American Society of Hematology Annual Meeting on December 14, 2026.
The company is evaluating other protein deficiency diseases using the same site-specific large gene integration technology at the albumin locus.