AAV

Targeted Genetics Corporation (NASDAQ: TGEN) today announced the sale to Genzyme Corporation of certain assets, including manufacturing technologies and other adeno-associated viral (AAV) vector technology, for up to $7 million in cash. The purchased assets consist primarily of patents, know-how and manufacturing-related equipment. Genzyme also received a license to use certain technology and materials necessary for manufacturing AAV vectors.

San Diego, CA - November 26, 2008 – Ceregene, Inc., a biopharmaceutical company, today reported clinical data from a double-blind, controlled Phase 2 trial of CERE-120 in 58 patients with advanced Parkinson’s disease. The trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Both groups showed an approximate 7 point improvement in the protocol-defined primary endpoint (Unified Parkinson’s Disease Rating Scale- motor off score at 12 months), relative to a mean at baseline of approximately 39 points.

Introduction:

Adenoviruses were first developed as expression vectors by Dr. Frank Graham and his colleagues at McMaster University. Microbix collaborates extensively with Dr. Graham, who provides access to the leading edge of Ad vector production technology and vector design. They have developed recombinant adenoviruses for gene delivery and vaccine purposes for many years, and benefit from access to technologies such as Dr. Graham's popular AdMax™ Cre-lox/FLP-frt based system for rapid, high-efficiency Ad vector construction

Molecular Biology of the Adeno-Associated Virus

Adeno-associated viruses are non-enveloped and non-pathogenic parvoviruses comprising a double-stranded 5.5 kb genome packaged into a capsid assembly of proteins. At less than 50 nm, this virus is one of the smallest used in gene therapy applications. Consequently, its genome is comprised of only two genes, rep and cap encoding for non-structural and structural genes respectively, which are flanked by two 145 bp ITRs (see figure one).