Lexicon Pharmaceuticals - Technology

Technology

Lexicon uses patented gene trapping and gene targeting technologies to generate and study knockout mice to discover the physiological and behavioral effects that result from the disruption of a single gene knockout. Because there is a close similarity in gene function and physiology between mice and humans, with a large majority of human genes having a counterpart in the mouse genome, knockout mouse technology has become a powerful tool in the discovery of new medicines.

The value of Lexicon’s technology in drug discovery has been described in a retrospective analysis by the scientific journal Nature. The conclusion of this analysis was that, in most cases, there was a direct correlation when comparing the physiological characteristics, or phenotypes, of knockout mice to the therapeutic effect of the 100 best-selling drugs of 2001. The tremendous utility of knockout mouse technology was recognized in 2007 with the Nobel Prize in Physiology or Medicine to Drs. Mario Capecchi, Martin Evans, and Oliver Smithies.

In developing small molecule drugs for its validated targets, Lexicon uses sophisticated medicinal chemistry known as "click chemistry." Dr. K. Barry Sharpless, who was awarded the 2001 Nobel Prize in Chemistry, pioneered this set of powerful and reliable tools for the rapid synthesis of novel compounds. Lexicon uses solution-phase chemistry to generate diverse libraries of optically pure compounds that are built using highly robust and scalable organic reactions that allow the company to generate compound collections of great diversity and to specially tailor the compound collections to address various therapeutic target families. Lexicon’s medicinal chemists design these libraries by analyzing the chemical structures of drugs that have been proven safe and effective against human disease and using that knowledge in the design of scaffolds and chemical building blocks for the generation of large numbers of new drug-like compounds.

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