14 –Year Quest For An Approved Orphan Disease Drug

By Cliff Mintz, Ph.D., Contributing Editor

Stuart Peltz, Ph.D., was very satisfied with his life as a tenured professor at the University of Medicine and
Dentistry of New Jersey (UMDNJ). He had spent most of his professional career studying the effects of posttranscriptional RNA (ribonucleic acid) control (all the regulatory events that take place after an RNA
molecule is made) on the regulation of gene expression and protein synthesis. However, by the late 1990s, after studying RNA biology for 20 years, Peltz was convinced he could effectively use post-transcriptional regulatory
control targets to identify new treatments that might provide therapeutic benefits to a wide variety of patients — especially those with rare genetically inherited diseases like cystic fibrosis (CF) and Duchenne Muscular
Dystrophy (DMD). This prompted him to leave his life as a tenured UMDNJ professor and start Post-Transcriptional Control Therapeutics (PTC) in 1998.