UMDNJ "Gives Birth" to Biotech Company

PTC Therapeutics, Inc., a privately held biopharmaceutical company based in South Plainfield, has received $15 million in financing to advance drug discovery efforts based on its proprietary integrated ribonucleic acid (RNA) biology and RNA chemistry programs.

Stuart Peltz, PhD (right), professor of molecular genetics and microbiology at UMDNJ-Robert Wood Johnson Medical School (RWJMS), co-founded the company along with Tariq M. Rana, PhD, associate professor of pharmacology at RWJMS and Allan S. Jacobson, PhD, chairman of the Department of Molecular Genetics and Microbiology at the University of Massachusetts Medical School. Peltz is president and CEO of PTC, Jacobson is chairman, and Rana is chairman of the scientific advisory board. Others who have contributed to technical development are Jeffrey Willusz, PhD, of UMDNJ-New Jersey Medical School, Jonathan Dinman, PhD, Joseph Dougherty, PhD, and Terri Goss Kinzy, PhD, all of RWJMS. The University is a shareholder in the new venture.

"RNA is an exciting new area of drug discovery. RNA targets offer promise across a wide range of diseases, and PTC is well positioned to capitalize on these advances," says Peltz. "We believe that intervention in the translation of RNA to protein offers a way to regulate gene expression precisely and to improve our ability to control therapeutically useful proteins."

The central paradigm of molecular biology is that genes are composed of DNA, that DNA can be transcribed to RNA, and that RNA directs the synthesis of proteins. Genes may over- or under-express proteins or may produce faulty proteins, all events that may be linked to disease states. Hundreds of such connections between genes and disease have already been established, and more are being discovered.

PTC's scientific discoveries and platform technologies allow for the identification of small molecules that selectively bind to RNA molecules and modulate the synthesis of proteins. The exciting application of these technologies is that drugs can be identified for many diseases that are the results of mis-regulation of gene expression. RNAs, like proteins, have unique structures that allow small molecules to bind with great selectivity and specificity. PTC focuses on developing small molecule drugs that bind to a single RNA and regulate an aspect of the cell's biology.

The RNA technology base was assembled from multiple faculty inventions at UMDNJ and is under license to PTC. This technology is the culmination of over a decade of effort and is the result of a decision to focus new hiring and resources in RNA chemistry and biology made by RWJMS leadership in the early 1990s.

PTC's efforts have focused on a number of clinical areas including anti-infectious and anti-inflammatory processes, as well as cancer. In particular, PTC has begun to develop drugs to treat viral infections. There are relatively few treatments for these diseases and drug resistance is a significant issue. Because PTC's approaches target a host cellular process - the mechanism the virus uses to replicate - they may reduce the virus's ability to develop drug resistance.