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- W2097758889 abstract "PTC Therapeutics, Inc. (PTC) has established a new program to identify compounds that modulate the levels of the survival of motor neuron (SMN) protein at the post-transcriptional level. The program is driven by three innovative approaches. The first approach utilizes PTC’s proprietary drug discovery platform technology called GEMS (Gene Expression Modulation by Small-molecules) to identify small molecules that up- or down-regulate the production of proteins, such as SMN, via the untranslated regions of mRNAs. The GEMS technology has proven to be very robust and has been applied successfully by PTC to address challenging drug targets across multiple therapeutic areas. The second approach utilizes PTC’s nonsense suppression platform to identify compounds that read through the aberrant stop codon present in the SMNdeltaEx7 mRNA, which it is believed could improve the stability of the abnormal SMNdeltaEx7 protein that is present in all SMA patients. In the third approach, we are screening for small molecules that alter the splicing pattern of the SMN2 RNA and increase inclusion of exon 7 into SMN2 transcripts and therefore, increase the level of the SMN protein. We have implemented and run high-throughput screens based on these three approaches and have identified compounds that increase the levels of functional SMN. The molecules identified in each of the three screens are being characterized for biological, pharmacological and pharmaceutical properties. The next stage of this drug discovery effort will include medicinal chemical optimization of the most promising lead molecules with the ultimate goal of identifying a drug candidate for preclinical and clinical development." @default.
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- W2097758889 date "2007-10-01" @default.
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- W2097758889 title "G.P.2.17 An innovative and integrated approach to identify compounds that modulate SMN protein expression at the post-transcriptional level" @default.
- W2097758889 doi "https://doi.org/10.1016/j.nmd.2007.06.073" @default.
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