New results support continued development of AAV-based human factor VIII gene therapy

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, August 20, 2018–Gene therapy using an optimized adeno-associated virus (AAV) to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals. These promising data combined with evidence that the optimized vector AAVhu37 is suitable for manufacturing and purification at scale, suggests that AAVhu37-based gene therapy has the potential to advance to the clinic to treat hemophil-ia A, according to an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the full-text article free on the Human Gene Therapy website through September 20, 2018.

James M. Wilson, MD, PhD, University of Pennsylvania, Philadelphia and colleagues from University of Pennsylvania and Ultragenyz Gene Therapy, Cambridge, MA coau-thored the article entitled "Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques." The researchers compared two AAV cap-sids – AAVrh10 and AAVhu37 – combined with two liver-specific promoters and en-hancers – E03.TTR and E12.A1AT. The researchers concluded that development of AAV-based gene therapeutics for hemophilia A should continue using the AAVhu37 capsid.

"The field now appreciates that there is a finite dose window in which systemic rAAV will be safe and effective," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chan-cellor, University of Massachusetts Medical School, Worcester, MA. "This means that optimization of the potency of vector constructs may be critical to enable a long-term clin-ical success in the treatment of hemophilia A."


About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. 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, Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its companion journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. 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 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers ( website.

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

Media Contact

Kathryn Ryan
[email protected]