Metagenomi announces data from hemophilia study supporting MGX-001

Metagenomi announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001. “We are thrilled to achieve this preclinical milestone supporting our recent decision to declare MGX-001 as our development candidate for hemophilia A,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We conducted this NHP study in response to a competitive landscape where gene therapies have been unable to achieve long term persistence of FVIII activity levels in patients. Establishing proof-of-concept of site specific gene integration and durable activity levels of FVIII in NHPs over twelve months in hemophilia A represents an important validation of our platform. Our goal for MGX-001 is to provide a one-time, curative treatment for adults and children with hemophilia A. Furthermore, we intend to leverage the MGX-001 editing platform to pursue additional therapies for secreted protein disorders.”…”The treatment of hemophilia, which has undergone many transformative changes over the past 60 years, is poised for yet another disruptive change: the use of genome editing, with site specific integration of FVIII, to produce functional cures in patients with hemophilia A. I am encouraged by the preclinical progress in the genome editing space to potentially provide a new path to a one-time, curative treatment option for both adults and children in hemophilia A in the future,” said Dr. Glenn Pierce, member of the Metagenomi Scientific Advisory Board.