According to Dr. Peltz, post-transcriptional control mechanisms include the decoding of the RNA molecule leading to protein synthesis. The efficiency of RNA in making protein or the length of time RNA lives in a cell can have a direct impact on how much protein is produced. Post-transcriptional control processes are used to control the amount of proteins made in all tissues and cell types. Multiple diseases are caused by the altered control of these regulatory events.
PTC’s approach is to discover and develop small molecule drugs that inhibit or enhance protein production by targeting post-transcriptional control mechanisms. By focusing on the modulation of gene expression at the post-transcriptional level, PTC connects the enhanced therapeutic opportunity of targeting a new area of biology with the effectiveness of small molecule drugs.
PTC “started doing the classic venture capital route,” by raising $132 million from offerings in the U.S., Europe, and Asia in the years 2000 through 2005. The second tier of financing ($180 million in cash) came from strategic investors and collaborators representing major pharmaceutical companies. Finally, there was grant and foundation support, totaling $85 million.