Overcoming AAV Manufacturing Challenges

By Sybil Danby, Senior Vice President of Business Development and Strategy, Center for Breakthrough Medicine

Gene therapies represent a very different paradigm than conventional therapeutics. Not only are most single-dose, curative treatments, many candidates in the clinic today and nearing commercialization target rare diseases with limited patient populations and no existing treatment options. Product development companies and patient advocacy groups are pushing strongly to get these products rapidly moved through the development cycle. In addition, it is essential for innovators to be first to receive approval in a particular indication in order to address the initial and greatest demand.

Time is of the essence, and consequently developers often compromise on their manufacturing processes—not from a safety perspective, but with respect to scalability and cost of goods—in ways that would not be acceptable for engineered proteins and monoclonal antibodies (mAbs).