CHALLENGES AND SOLUTIONS TO QUALITY GMP SUPPLY OF AAV VECTORS

摘要

Adeno-associated virus (AAV) has emerged as the vector of choice for safe, efficacious and potent gene therapy. With increasing clinical successes, demands for high-quality, robust, cost-effective cGMP supplies of viral vector are soaring. Bioprocess engineers and manufacturers are tasked to meet these growing needs and are challenged to find solutions that can evolve at the same pace as evolving therapies. Lonza is developing and implementing solutions to meet the clinical supply demand in its flexible, multi-product facility, and can accommodate a variety of production systems (transfection, producer cell line, recombinant baculovirus or herpesvirus-mediated) from raw materials to the final filled drug product. The advantages and disadvantages of the production systems will be discussed.