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Targeted Genetics Receives Patent for Enhanced Core Technology Including Expressed RNA


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Targeted Genetics Corporation has announced the issuance of an additional patent related to its adeno-associated virus (AAV) vector technology that describes delivery of genes or delivery of small therapeutic genetic constructs or small therapeutic genetic constructs including therapeutic RNA molecules such as RNAi.

U.S. patent #7,125,717 entitled "Metabolically Activated Recombinant Viral Vectors and Methods for Their Preparation and Use" describes the use of AAV vectors that are efficient for expression of therapeutic genetic constructs.

Single stranded Deoxyribonucleic acid (DNA) vectors like AAV require conversion to a double-stranded DNA form in order for the therapeutic gene of interest to be expressed. The AAV virus DNA is about 4.7 Kb in size.

However, a significant number of either genes used in gene therapy as well as genetic constructs such as expressed RNA or RNAi are approximately half this size or less.

This patent demonstrates that AAV vectors containing genetic sequences that are roughly half the size of an AAV payload can form intrastrand based pairing, converting the therapeutic construct to an expressible form.

This results in an efficient expression of the therapeutic genetic construct, and has the potential for increased benefit in treating or preventing disease.

"The issuance of Targeted Genetics' latest patent adds to a portfolio of intellectual property that enhances the utility of AAV vectors in two important ways: first, for expressing therapeutic genes in order to add back necessary proteins to prevent or correct a disease state, and second, for expressing therapeutic RNA molecules, such as RNAi, which can silence or block the expression of abnormal proteins associated with other disease states," said Barrie J. Carter, Ph.D., executive vice president and chief scientific officer of Targeted Genetics Corporation and inventor on the patent.

"Abnormal production of proteins is the cause of many human diseases; and RNAi naturally occurs within cells to regulate gene expression, thus blocking or silencing the production of specific disease causing proteins."

"The use of AAV Vectors described in this patent to express these specific double stranded therapeutic RNAs, such as RNAi, may specifically block the expression of these abnormal proteins associated with disease."

"Targeted Genetics has over ten years of expertise in the development of AAV vectors to treat a broad array of diseases and using AAV vectors to treat diseases where delivery and efficient expression of a therapeutic gene of interest has long been a focus of the company."

"Given the recent advances and excitement in the field of developing a new class of RNA therapeutics, such as RNAi, that block the expression of abnormal proteins, development of the use of vectors described in this issued patent is a natural extension of our work as a company and broadens our class of potential therapeutic targets," said H. Stewart Parker, president and chief executive officer of Targeted Genetics.

"The key is that utilizing these vectors may allow us to treat disease by essentially turning on or turning off gene expression associated with disease states."

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