ABSTRACT
Since 1980, with more than 90 recombinant drugs approved for use in the United States, recombinant protein products have become an integral part of pharmaceutical development. Listed in Table 1 are therapeutic agents derived from these biotechnologies that have been approved for human use in the treatment of diseases. In recent years, as a result of advances made in the mapping of the human genome, there has been explosive growth of the targets being identified. A survey by the Pharmaceutical Research Manufacturers of America (PhRMA) found 369 medicines in the pipeline that meet the definition of “biotechnology medicines.” These medicines, with some exceptions, are recombinant versions of proteins found in vivo. For some, these protein agonists activate specific receptors located in defined target tissues. They are used either as replacement therapy, e.g., insulin and growth hormone, or as a supplement to increase the activity of endogenous proteins. They are also used as therapeutic antibodies and site-specific carriers of toxic drugs for therapy or as imaging agents. The investigational medicines target more than 200 diseases that include cancer, arthritis, asthma, multiple sclerosis, and heart disease. Numerous recent articles further record and reflect the influence of biotechnology-based medicines on modern pharmaceuticals [1-5]. This chapter will discuss the basic concepts behind these technologies and will introduce the reader to the significant tasks and challenges encountered during product development to provide commercial medicines.
