Will gene therapy trump factor treatment in hemophilia?

Abstract

Hemophilia treatment is entering a new phase, with the exciting possibility of gene therapy promising a cure. Novel gene transfer strategies are being considered for patients with inhibitors.Improvement of factor-replacement therapy is being aggressively pursued with long-acting factor concentrates, many of which are in clinical trials. Whether gene therapy will be safe and cost effective to eventually supersede factor-replacement therapy is yet to be determined. It is hoped that with the profusion of clinical trial programs in hemophilia care, it will eventually provide affordable treatment to many patients who currently cannot access adequate treatment in the developing countries.

Keywords::

• ex vivo gene therapy
• gene therapy
• hemophilia
• inhibitors
• long-acting factor VIII/IX

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.