In the central dogma of molecular biology - DNA to RNA to protein - much progress has been made in understanding (and modulating) the first step of gene expression - transcription of mRNA. But processes affecting protein translation are less well understood, even though control of RNA stability and translation may provide a number of targets for drug discovery to treat viral infections, genetic disorders, immune and inflammatory diseases and cancer. It is these processes that PTC Therapeutics Inc. is targeting.

"There are three ways we do this: we have a translation program; we have an RNA screening program to identify small molecules that bind selectively and specifically to RNAs; and we have a cell-free mRNA turnover assay," said Stuart Peltz, president and CEO. All three programs relate to situations where under- or over-expression of a particular protein leads to disease. "It is an important mechanism, but it's underdeveloped from a drug development approach," Peltz noted.