The Science Behind Our Pipeline.

At PTC, we are focused on modulating protein expression within the cell. We have developed and assembled an integrated set of proprietary technologies focused on our understanding of RNA biology for the discovery of novel small molecule therapeutics. Our technologies allow us to screen our compound library against targets across multiple therapeutic areas.

 

Nonsense Readthrough

A nonsense mutation results in a premature stop signal in the messenger RNA (mRNA), which stops the ribosome before it completes translating the protein. Our nonsense readthrough technology identifies small molecules that increase readthrough at these premature stop signals to enable the production of full-length proteins. Using this technology, we identified ataluren for the treatment of nonsense mutation genetic disorders. In addition to increasing readthrough, we are also focused on identifying small molecules that stabilize nonsense-containing mRNAs that can enhance the effect of a compound like ataluren that acts through the nonsense readthrough mechanism.

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