Gene Delivery Vectors
Targeted Genetics discovers, develops, and delivers molecular medicine to treat inflammatory arthritis, HIV/AIDS, congestive heart failure, and Huntington’s disease. Its platform technology is based on a gene delivery technology in which DNA sequences are delivered to cells with the goal of preventing or treating disease.
Adeno associated virus (AAV) is a nano particle of 25 nm; it is not known to be associated with any human disease. Recombinant AAV (rAAV) vectors have a protein coat that encloses a piece of DNA which, when introduced into a cell, will result in expression of the gene of interest encoded by the DNA fragment. The AAV capsid forms the protein coat, which has affinity with certain cell-surface receptors.
“When the particle is injected, it is recognized by the receptor and is transported into the cell, whereupon the gene is expressed,” explains Pervin Anklesaria, Ph.D., vp of therapeutic development.
Targeted Genetics intra-articularly delivers a gene that expresses a TNF-a antagonist for local treatment of rheumatoid arthritis. In another application, AAV is used to deliver DNA-encoding HIV antigens that can be used as a prophylactic vaccine to induce immune response. “The AAV vector genomes are more stable and persist in cells for a longer time than traditional plasmids or adenovirus vectors, so they require less frequent dosing. The AAV is also safe because it does not integrate into chromosomal DNA,” adds Dr. Anklesaria.
Targeted Genetics has developed a manufacturing process for AAV based on the same technology used to produce mAbs and therapeutic proteins. The company’s strengths reside in an understanding of the different attributes of the AAV products and the manufacturing and purification process, according to Dr. Anklesaria. It has a scalable manufacturing process for its AAV vector products and is able to quantify and evaluate the products with appropriate analytical methods, thus resulting in a well-characterized process and products.
“Our product is a biological product. Even though it is different from traditional protein products, it is important that it is tested, analyzed, and characterized in the same way as the other biologics. We make sure we have the right analytical tools and that the process that we use to produce them is state-of-the-art,” concludes Dr. Anklesaria.
Progress in the development of new, more powerful analytical instrumentation for biologics-based materials is ongoing. As scientists continue to look at their molecules in greater detail and resolution they will be able to make quicker research decisions. The efficiency of new drug development will increase and push science forward.