Targeted Genetics is the leading innovator of technologies for manufacturing and developing AAV-based products. Our portfolio of AAV-related intellectual property reflects this leadership and positions us as the partner of choice for companies and organizations seeking to apply the broad utility of AAV to their own product development programs.

AAV vectors have a number of
potential therapeutic applications, including:

•  Localized delivery of therapeutic proteins.
•  Sustained delivery of therapeutic proteins.
•  Vaccines.
•  Enabling gene-based approaches to targeting disease-related pathways that are not amenable to modulation with small molecule drugs or protein therapies.
•  Delivery of short interfering RNA (siRNA) constructs to inhibit the activity of disease-related genes.


We added several new patents to our portfolio of AAV-related intellectual property in 2006.
Some of the most notable U.S patents that were issued include:

"Methods for generating high titer helper-free
preparation of released recombinant AAV vectors."
  The patent describes a double chemistry chromatography process for AAV purification. This approach leads to highly purified AAV vector stocks and is a more cost-effective and scalable purification approach than can be achieved by centrifugation or single chemistry chromatography.

"Methods for generating high titer helper-free
preparation of released recombinant AAV vectors."
  The patent describes an AAV manufacturing process in which numerous growth conditions used to culture the production cells are controlled in a manner that leads to the release of AAV vector particles into the cell culture medium without the need for breaking open the host cell membrane. This release of vector into the media leads to improved production quality and allows for a more cost-effective approach to large-scale manufacturing of AAV vectors.

"Adeno-Associated Virus Serotype 1 Nucleic Acid
Sequences, Vectors And Host Cells Containing Same."
  The patent covers compositions of AAV vectors that encode capsid proteins derived from AAV serotype 1 (AAV1) and host cells into which such vectors are introduced and is applicable to both AAV1 serotype and AAV1 pseudotyped vectors. Vectors of one serotype that are engineered to express capsid proteins of a different serotype are said to be "pseudotyped." Vectors expressing AAV1 capsid proteins have been shown to have higher transduction efficiencies than vectors based on other serotypes.

"Compounds and methods to enhance rAAV  transduction."
  The patent describes a novel approach for improving the efficiency with which AAV vectors deliver genes to target cells. This approach covers small molecule compounds and methods that enhance the trafficking of AAV vectors to the nucleus of a host cell. Transport to the nucleus is necessary for expression of delivered genes.

"Metabolically Activated Recombinant Viral
Vectors and Methods for Their Preparation and Use."
  The patent covers the use of AAV vectors to deliver genes or small therapeutic genetic constructs or small therapeutic genetic constructs including therapeutic RNA molecules such as RNAi. It describes the use of AAV vectors that are more efficient for expression of therapeutic genetic constructs. RNAi has promising therapeutic potential, and AAV vectors may provide a safe and effective method for delivering therapeutic RNA molecules to target cells.


While our AAV patent estate is important to our proprietary and partnered programs, the portfolio also represents an asset from which we can create value and derive revenue. In December 2006, we entered into a non-exclusive licensing agreement for two AAV1-related patents. Under this agreement, we received a significant licensing fee and will receive potential milestone payments and royalties on the sale of any products commercialized using the licensed technology.

Strengthening and expanding our AAV patent estate is a pillar of our business strategy. Already in 2007 we have announced another patent titled "Adeno-Associated Virus (AAV) Serotype I Nucleic Acid Sequences, Vectors and Host Cells Containing Same" describes the use of AAV vectors that contain the inverted terminal repeat (ITR) and capsid sequences derived from AAV1 and host cells transduced with such vectors. This patent is important because it expands our position in the use of AAV1 and AAV1 pseudotyped vectors.