The private sector would doubtless find many of these

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Unformatted text preview: s. Generating molecular probes for exploring the basic biology of health and disease in academic laboratories would not supplant the major role of biopharmaceutical companies in drug development, but could contribute to the start of the drug development pipeline. The private sector would doubtless find many of these molecular probes of interest for further exploration through optimization by medicinal chemistry, target validation, lead compound identification, toxicological studies and, ultimately, clinical trials. Academic pursuit of this first step in drug development could be particularly valuable for the many rare mendelian diseases, in which often the gene defect is known but the small market size limits the private sector’s motivation to shoulder the expense of effective pharmaceutical development. Such translational research in academic laboratories, combined with incentives such as the US Orphan Drug Act, could profoundly increase the availability of effective treat842 ments for rare genetic diseases in the next decade. Further, the development of therapeutic approaches to single-gene disorders might provide valuable insights into applying geno...
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