Immusol proves the effectiveness of its Target Validation technology

Immusol, Inc, a privately-held biotechnology company, today announced scientific corroboration of the company's proprietary Target Validation technology. Immusol's work is described in Antisense and Nucleic Acid Drug Development, Volume 10, pages 409-414. This study proves the general utility of Immusol's Target Validation technology using ribozyme gene delivery for the rapid, efficient, high-throughput inactivation of specific gene sequences.

Now that most of the sequence of the human genome is known and published, the next important step for drug development is to determine which of these genes would make effective targets for pharmaceutical intervention. "Our Target Validation technology allows us to experimentally simulate the characteristics of a perfect drug, specifically inactivating any given gene sequence," said Jack Barber, PhD, Immusol's Vice President of Research and Development, "If inactivation of that gene results in a therapeutic response, without demonstrable toxicity, then we know that that gene would make a good target for drug screening." The company believes that Target Validation gives Immusol and its corporate partners the advantage of high-speed functional confirmation of drug target genes to speed up drug discovery.

This finding comes on the heels of several recently published discoveries from Immusol: The role of a transcriptional repressor gene for breast and ovarian cancer therapy (Proceedings of the National Academy of Sciences, USA volume 98, pages 130-135); The identification of a previously unknown "tumor suppressor" gene that specifically kills cancer cells but not normal cells (Genomics, volume 66, pages 274-283); The discovery of two new human cellular genes required for Hepatitis C Virus protein synthesis (Proceedings of the National Academy of Sciences, USA volume 97, pages 8566-8571); and the discovery that the gene telomerase can act as a "tumor suppressor" (Nucleic Acids Research, Volume 28, pages 2605-2612).

"Target Validation technology helps the company establish a cause-and-effect relationship between a specific gene and a specific biological function," said Tsvi Goldenberg, PhD, Immusol's Chairman and Chief Executive Officer. "According to the US Patent Trade Office's (US PTO) new guidelines, knowledge of biological function is required for gene patenting. We feel that puts us in a perfect position to establish an extensive intellectual property portfolio for drug discovery." The stated aim of the US PTO's new guidelines is to prevent companies from patenting genes before having a clearly defined use for them.

Immusol has also developed a unique approach to drug target gene discovery called Inverse Genomics. Inverse Genomics uses a library of over 10 million different ribozymes to sift through the entire genome and rapidly isolate those genes that are potentially useful as drug targets. This approach addresses the problem of drug target identification from the opposite direction of traditional discovery programs. Researchers using this means of discovery start with a biological feature with therapeutic potential and work backward, identifying only those genes that are potentially viable therapeutic targets. Conventional approaches start with the entire human genome and attempt to narrow down to those genes that control a biological feature with therapeutic potential. Immusol is currently using Inverse Genomics, in conjunction with its Target Validation technology, to identify and functionally validate therapeutic drug target genes in the areas of cancer, obesity/diabetes, viral infection, neurodegenerative disease and allergy/inflammation. Immusol's mission is to use functional genomics to develop therapeutics, both internally and in collaboration with pharmaceutical and biotechnology companies.

Source: Immusol, Inc.