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April 15, 2009 (Vol. 29, No. 8)

Firm Aims to Regulate RNA in Rare Diseases

PTC Therapeutics Focuses on Small Molecule Drugs for Genetic Disorders and Cancer

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    A PTC Therapeutics chemist conducting a compound synthesis experiment

    When Stuart Peltz, Ph.D., and Allan Jacobson, Ph.D., founded PTC Therapeutics in 1998, they realized that post-transcriptional control (PTC) mechanisms—all the regulatory events that take place after an RNA molecule is made—were an unexploited area of biology. At the time, Dr. Peltz was a researcher at Rutgers University Medical School, and Dr. Jacobson was chairman of the department of molecular genetics and microbiology at the University of Massachusetts Medical School. They both saw the potential opportunity to treat rare genetic disorders and other diseases.

  • Post-Transcriptional Control

    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.

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