October 2024 Featured Report

Guided by standardized production and purification platforms, developers of monoclonal antibodies (mAbs) and other recombinant-protein therapeutics can take a relatively straightforward path when designing and establishing facilities for commercial-scale operations. By contrast, designing gene-therapy (GT) facilities involves a more complex calculus. Multiple approaches are available for producing transgene-bearing viral vectors at commercial scales. GT developers must choose carefully, knowing that each manufacturing strategy raises distinctive advantages and limitations for process scalability, equipment implementation, storage, material and personnel flow, and ultimately, facility output. The plurality of manufacturing strategies — and the possibility of further platform advancements — requires GT companies and their facility-design teams to be forward-thinking in their planning. All the while, GT facilities must be engineered for good manufacturing practice (GMP) operations and work with viral vectors...

The field of bioprocess development has witnessed remarkable advancements in recent years driven by increasing demand for biopharmaceuticals, including gene-therapy products such as recombinant adenoassociated virus (rAAV) vectors. To date, global regulatory bodies have approved several rAAV-based gene therapies, and multiple clinical trials are ongoing as forecasts project significant growth over the next five to 10 years (1) . The field of rAAV production continues to be dominated by platforms using human embryonic kidney (HEK) and insect cells. For instance, uniQure’s proprietary platform is based on insect-cell production and a baculovirus expression vector system (BEVS) (2–5) . As the gene-therapy clinical pipeline expands, ensuring efficient, cost-effective, and scalable processes for viral-vector production becomes critical (1) . Central to that endeavor are scale-down models and fast-paced scale-up. Scale-down models are small-scale representations that are designed to mimic key process paramet...