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Transposagen Biopharmaceuticals, Inc. is the recipient of a $1.4 million Phase II Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH).  The company will use this grant to further its research into the efficient production of genetically modified knockout animal models for drug discovery and development. This award follows the $100,000 SBIR Phase I grant the company received in 2005. Transposagen was also awarded an additional $1 million SBIR Phase II match from the Kentucky Department of Commercialization and Innovation in 2008.

The NIH performs extensive external peer review using experts from academia and industry to assess the scientific merit, technical merit, and commercial application of the proposed technology.  Receiving the NIH awards further validates the breakthrough technology being developed by Transposagen. The company controls the only commercially viable technology to create knockout rats, which are animals with single genes disrupted, causing the animals to closely mimic various human diseases such as diabetes, Alzheimer’s, and cancer.

Transposagen's products are called MutaRatTM Knockout Rat Models, and they are crucial tools for drug discovery and development.  The company’s ultimate goal is to produce a MutaRatTM Knockout Rat Model representing every gene in the rat genome within 5 years.

Transposagen is using its breakthrough technology to help the pharmaceutical industry solve two very pressing drug development problems. First, the company has developed the MutaRatTM Drug Target Discovery System and MutaRatTM Tissue Bank which will help pharmaceutical companies replenish their drug discovery pipelines. Today, the top 100 best-selling drugs in the United States address only 43 gene targets. Recent analyses suggest that there are many additional drug targets represented in the human genome and our MutaRatTM Drug Target Discovery System is a powerful tool to aid in their discovery.

Second, Transposagen is now producing MutaRatTM Knockout Rat Models that can be used for testing potential therapeutics. Most potential therapeutics, nearly 90%, fail in human clinical trials after an average investment of $400 million dollars.  This indicates that more reliable animal models are needed to predict the success or failure of compounds at an earlier pre-clinical stage of drug development.  Knockout rats can predict the efficacy and toxicity of a potential therapeutic better than other animal models. Therefore, the use of our MutaRatTM Knockout Rat Models makes testing of potential therapeutics more accurate in preclinical stages, thereby making the entire drug development process more efficient. This saves millions of dollars in the cost of drug development, which is a savings that is ultimately passed on to the patients who need the new therapeutics.