Determining the minimally effective dose for gene therapy in hemophilia A

Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A. This pharmacology study, performed in a mouse model and designed to support the initiation of a Phase 1 clinical trial in patients with hemophilia A, is described in the peer-reviewed journal Human Gene Therapy. Click here to read the article now.

“While there are been a number of other gene therapy programs focused on hemophilia A, none has resulted in an FDA-approved therapy”, says Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD.  “The key to success is to develop a vector product that is potent enough that it will be effective within a safe dose range.”

James M. Wilson, MD, PhD, from the Perelman School of Medicine, University of Pennsylvania, and colleagues, used the adeno-associated virus (AAV) vector AAVhu37 to deliver the gene for human Factor VIII (hFVIII) to a hemophilia A mouse model. Since they detected hFVIII activity among all of the mouse groups administered different doses of the vector, the researchers concluded that the minimally effective dose was the lowest dose evaluated in the study. The activity levels seen suggested that the AAVhu37-based gene therapy approach would be sufficient to modify severe hemophilia A in humans.

“Our results strongly suggest that our gene therapy product would demonstrate clinical efficacy in hemophilia A patients,” state the investigators.

About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s more than 100 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.