Metagenomi Gets Investigational Approval for Hemophilia Treatment

By Katherine Hamilton

 

Metagenomi Therapeutics received approval from the Food and Drug Administration for its investigational new drug application for a hemophilia A treatment.

Preclinical data of the drug, known as MGX-001, demonstrated a normal range of FVIII activity, which is a blood protein that helps create blood clots. The results were in non-human primates.

Metagenomi plans to initiate a global clinical trial of the drug in the first half of 2027. It aims to provide initial clinical data in the second half of the year.

"In a landscape with no cure for hemophilia A, we believe MGX-001 has the potential to provide long-term benefit to both adult and pediatric patients," Chief Executive Jian Irish said.

 

Write to Katherine Hamilton at katherine.hamilton@wsj.com

 

(END) Dow Jones Newswires

November 10, 2026 17:42 ET (22:42 GMT)

Copyright (c) 2026 Dow Jones & Company, Inc.

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