This patent covers adenovirus AAV (Ad-AAV) hybrid vectors encoding the receptor for very low density lipoprotein (VLDL). These vectors are potentially very efficient and useful in the production of AAV vectors to treat hyperlipidemia. The Ad-AAV hybrid vector covered by this patent could also be used as a standalone therapeutic. The patent was issued to University of Pennsylvania and is exclusively licensed to Targeted Genetics.

"Today's patent is an important addition to our preclinical hyperlipidemia program, which is designed to evaluate a number of therapeutic gene candidates delivered by AAV vectors, including the receptor for VLDL, an important receptor for regulating lipid levels including cholesterol in blood," said Barrie J. Carter, Ph.D., chief scientific officer of Targeted Genetics.

"AAV vectors have become one of the most promising systems for gene delivery, due to AAV's many characteristics that make it broadly applicable to in vivo prevention and treatment of a number of diseases," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "Our intellectual property covers a broad range of AAV assets, and places Targeted Genetics in a leadership position in the field. We believe that our AAV technology and manufacturing expertise sets us apart and will continue to be major assets in our future product development and partnering activities."

AAV vectors are derived from a naturally occurring virus that has never been associated with any disease. AAV vectors can efficiently deliver genetic information to numerous cell types and can be engineered to carry a variety of DNA sequences, including genes, RNAi, antisense, and nucleic acids to encode peptides and monoclonal antibodies. AAV vectors are highly stable and persist in cells for extended periods of time, which allows for long term gene expression.

Hyperlipidemia, which is thought to play a role in cardiovascular disease, is linked to the elevation of lipids, or fats, such as cholesterol, in the bloodstream. Approximately four million people in the United States have a genetic predisposition to some form of hyperlipidemia, such as familial hypercholesterolemia, familial combined hyperlipidemia and polygenic hypercholesterolemia. Approximately 10% of these patients have severe forms of the disease and do not respond to standard drug therapy, such as statins. If untreated, disease progression can lead to morbidity and death from heart attack or stroke.

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