INTRODUCTION
REASONS FOR THE STUDY
The use of antisense compounds as therapeutics has become increasing popular in the past few years. With the completion of the sequencing of the
human genome, antisense offers a tool for designing drugs to specifically treat any disease or disorder related to abnormal gene expression. In 1998,
the approval of the first antisense therapeutic for treating CMV retinitis, VitraveneÔ, ushered in a new wave of interest in antisense compounds
used as therapeutics.
OBJECTIVES OF THE STUDY
This report provides an overview of the gene expression process and how antisense compounds can be used to control gene expression. A summary is
provided of the technological advances made in antisense compounds and how each impacts antisense therapeutics. The applications of antisense
therapeutics and strategies used by each company to meet the needs of the application are examined.
AUDIENCE FOR THE STUDY
This report was written for those interested in the emerging field of antisense therapeutics. Individuals in the investment field (investor, market
analysts, fund managers, etc.) and biotechnology or pharmaceutical industries (CEOs, business development managers, licensing strategists, product
managers, research scientists, etc.) are the intended audience of this report.
SCOPE AND CONTENT OF THE STUDY
This report analyzes the potential markets for antisense therapeutics. Because of the early developmental stage of this industry, companies were
included that had at least one antisense therapeutic in preclinical development. This resulted in 18 companies and their products to be profiled in
this report. These companies have developed antisense therapeutics for used in a variety of applications: cancer, coronary heart disease, type 2
diabetes, immunological disorders, infectious diseases, polycystic kidney disease, and retinal disorders.
METHODOLOGY USED IN STUDY
This report forecasts the potential markets for antisense therapeutics. The data used in these forecasts were obtained from members of the
antisense therapeutic industry, research institutes, foundations, governmental and international organizations, scientific literature, as well as from
annual reports and other publications from antisense companies and other pharmaceutical companies.