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Biotechnology and Applied Biochemistry (2002) 35, (141–148) (Printed in Great Britain)
Review
An adventure in biotechnology: the development of haemophilia A therapeutics – from whole-blood transfusion to recombinant DNA to gene therapy
Henry S. Kingdon* and Roger L. Lundblad†1
*Baxter Biosciences, Baxter Healthcare Corporation, Deerfield, IL 60615, U.S.A., and †Department of Pathology, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, U.S.A.
Key words: antibody affinity chromatography, cryoprecipitate, Factor VIII, plasma fractionation, viral pathogen. Abbreviations used: DDAVP, 1-amino-8-D-arginine or desmopressin; FEIBA, Factor VIII inhibitor-bypassing activity. 1To whom correspondence should be addressed, at P.O. Box 16695, Chapel Hill, NC 27516-6695, U.S.A. (e-mail r.lundblad@worldnet.att.net). 
The past decade has seen an explosion in the number of therapeutic proteins available for a wide spectrum of diseases. Some of these proteins are obtained from human plasma. Examples of these therapeutic proteins are albumin, intravenous immunoglobulins and prothrombin complex concentrates. The majority of new therapeutic proteins are, however, derived via recombinant DNA technology. There are other examples where the first therapeutic preparation was a crude preparation derived from plasma or tissue and where subsequent development has resulted in a recombinant form of the therapeutic protein. This article focuses on the development of therapeutics for the treatment of haemophilia A (deficiency of Factor VIII activity). The progression from crude plasma fractions to monoclonal-purified preparations to the more recent development of therapeutic concentrates via recombinant DNA technology is described in some detail. Finally, the current status of gene therapy for haemophilia A is evaluated. Both technical issues as well as market forces are described, as both have had significant impact on the product-development process.
Received 10 October 2001/20 December 2001; accepted 10 January 2002
Portland Press Ltd © 2002 |
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