PTC's ability to develop drugs that target post-transcriptional control of gene expression has substantial advantages:

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.