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On July 20, 2012, a committee of the European Medicines Agency recommended for regulatory approval the first gene therapy using an adeno-associated virus vector to treat lipoprotein lipase deficiency (LPLD), a very rare genetic disease. With a normal diet, patients lacking sufficient levels of lipoprotein lipase have abnormally high serum triglycerides and high levels of very low-density lipoprotein (VLDL), resulting in acute severe pancreatitis and chronic conditions associated with high levels of VLDL, such as cardiovascular diseases. 

In 1995, Dr. Warren J. Leonard of the National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), reported the creation of an interleukin-2 receptor gamma chain (IL-2Rg) deficient mouse. His lab also demonstrated earlier that mutations in the gene encoding IL-2Rg result in X-linked severe combined immunodeficiency (XSCID) and that IL-2Rg is a subunit common to multiple cytokine receptors. The IL-2Rgdeficient mice created by Dr. Leonard provide a unique animal model, the first of its kind, to study XSCID and other immune deficiencies.

Medical research to combat disease is now a global effort and one that now requires an international technology transfer effort to see that new discoveries from research are effectively developed and commercialized to reach patients. While the United States in general, and the National Institutes of Health (NIH) in particular, have long led the way in effective biomedical technology transfer, such is not the case with new or younger transfer programs at research institutions outside the US, especially those in emerging and middle-income countries.