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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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This document was uploaded on 04/02/2014.
- Fall '14
- The Tempest