Metagenomi slashes workforce to focus on hemophilia A gene-editing therapy

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California-based Metagenomi will reduce its workforce by 25% in order to focus on its gene-editing hemophilia A program — MGX-001 — and later-stage preclinical pipeline.

In addition, the company’s president and former COO, Jian Irish, Ph.D., M.B.A, will take over as CEO, while the company’s founder and former CEO, Brian Thomas, Ph.D., will remain on the board of directors. As a result of these actions, Metagenomi expects to extend its cash runway into the fourth quarter of 2027.

Separately, Metagenomi reported new dose range finding data from the MGX-001 program, demonstrating curative factor VIII (FVIII) activity in non-human primates and intends to advance MGX-001 into clinical development.

“In light of the encouraging preclinical MGX-001 hemophilia A results we reported today, we made the decision to strategically reprioritize our pipeline and discovery efforts to focus resources on driving forward our lead program for hemophilia A and pursuing programs that leverage our most advanced, signature gene-editing capabilities. These include programs addressing additional secreted protein deficiencies that deploy the site-specific genome integration system used in MGX-001, and cardiometabolic indications in collaboration with Ionis,” said Irish.

Hemophilia A is the most common X-linked inherited bleeding disorder, caused by a large variety of mutations in the FVIII gene leading to a loss of functional FVIII protein. Intracranial bleeding is of greatest concern as this can lead to major morbidity and mortality. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children with hemophilia A.

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