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| GEMS |
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GEMS (Gene Expression Modulation by Small-Molecules) is a novel and proprietary screening technology for the identification of small molecules
that modulate post-transcriptional control mechanisms. Compounds identified through the GEMS technology modulate gene expression by targeting the post-transcriptional control processes that act through the UTRs of mRNA molecules. GEMS was utilized in the discovery of PTC299, in multiple ongoing drug discovery programs and has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Gilead, Merck, Pfizer, Roche and Parent Project Muscular Dystrophy. |
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The untranslated regions (UTRs) of a messenger or mRNA (shown above in red and indigo) sit on both sides (ie, 5’ and 3’) of the mRNA’s reading frame and contribute to the regulation of protein production. The GEMS technology constitutes a potent approach to drug discovery by facilitating the identification of small molecule drugs that specifically modulate the activity of these UTR regions to alter the level of protein production.
PTC's GEMS technology has many advantages to traditional drug discovery efforts, including:
- The ability to address a wide variety of genes as potential targets for drug discovery. These include new targets and previously intractable targets, as well as validated targets that can be addressed by an alternative mechanism (which can provide the opportunity for synergy with existing drugs).
- The ability to avoid the drug delivery challenges associated with biologics, antisense, gene therapy, or RNAi. PTC's approach offers the opportunity to replace such drugs with orally delivered small-molecule drugs.
- The ability to use orally bioavailable small-molecule drugs to either up- or down-regulate protein levels.
- The ability to address targets even when their function is unknown, bypassing the need to understand the complete disease pathway.
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