PTC Therapeutics has been awarded a Phase II Small Business Technology Transfer (STTR) Grant from the National Institute of Allergy and Infectious Disease of the National Institutes of Health (NIAID/NIH) to develop a virus-cell-based assay using HIV-1 vector systems for the discovery of novel drugs with anti-HIV activity.

Human immunodeficiency virus (HIV) is the causative agent of acquired immunodeficiency syndrome (AIDS). Despite efforts to prevent the spread of HIV, it is clear that the worldwide population of patients infected with this virus and developing AIDS is still increasing significantly. Although drugs that inhibit the activity of certain HIV enzymes, and thus inhibit HIV replication, have been identified and utilized successfully to treat AIDS (e.g., inhibitors of the viral reverse transcriptase and protease), these drugs do not completely eliminate the virus. In addition, strains of HIV that are resistant to available antiviral drugs are rapidly emerging, posing a continued health threat worldwide.

"The emergence of human immunodeficiency virus type 1 (HIV-1) strains resistant to active antiretroviral therapy necessitates continued drug discovery and development efforts for treatment of HIV-1 infection. Most current drug discovery strategies are directed against a single point in the HIV-1 replication cycle. A virus-cell-based assay, which can be adapted to high-throughput screening (HTS), would allow screening of multiple targets simultaneously," commented Joseph Dougherty, Professor of Molecular Genetics, Microbiology, and Immunology at University of Medicine and Dentistry (UMDNJ), principal investigator and collaborator on this grant with PTC Therapeutics. "This assay is based upon HIV-1 vector systems that mimic the HIV-1 life cycle without yielding replication-competent virus, making this assay safe, rapid and cost effective. Preliminary studies with five known anti-HIV-1 drugs indicate that this system provides the necessary sensitivity for drug screening."