Production of AAV vectors requires the introduction AAV rep and cap genes into cells. However, rep proteins can be toxic to some cell lines, thereby limiting cell growth and efficient creation of AAV vectors. The newly patented technology can limit toxicity of the rep proteins in those cell types by turning on only the rep gene during the manufacturing process.
This technology can be used in the company’s Ad-AAV hybrid manufacturing process, or can be integrated into the company’s existing stable cell-line manufacturing process.
“Our intellectual property in AAV vector manufacturing is significant, and the addition of this patent further solidifies our leadership position in cell-line based approaches to AAV production,” said H. Stewart Parker, President and Chief Executive Officer. “Targeted Genetics’ unmatched capabilities in AAV manufacturing cover both rapid production techniques as well as large scale manufacturing methods. This manufacturing competence has been an important factor in our ability to attract strategic partners enables us to undertake one of the largest and most advanced clinical development programs for AAV-based product candidates and continues to be an important asset for the Company.”
The combined attributes of persistent expression and a good safety profile make AAV vectors useful in developing gene-transfer treatments for a wide variety of chronic disease and the development of viral vaccines.