SMART AAV Analytical Method Toolbox - Live Online Training

Following a brief introduction to ATMPs and its regulations, this course will explore optimization strategies for the formulation, production, and analysis of adeno-associated viruses (AAVs). Participants will understand challenges and learn practical solutions for efficiency and compliance. The course highlights common obstacles and offers practical solutions demonstrated through case studies, providing insights into applying GMP principles in different scenarios.

In addition, expert speakers from manufacturing, research, and consultancy will share their experiences and lessons from GMP-compliant environments. Through their presentations, participants will gain a well-rounded understanding of the challenges and best practices in maintaining GMP compliance.

Gene therapies are rapidly gaining popularity as a cutting-edge approach to treating a wide range of diseases, offering enormous potential to address conditions from degenerative diseases to rare congenital neurological disorders. These therapies have the potential to revolutionize medicine by targeting the underlying genetic causes of illnesses, providing long-lasting or even permanent therapeutic effects. Central to many DNA-based gene therapies is the use of viral vectors to deliver genetic material directly into the patient’s cells, and among the various viral vectors available, the most commonly used and highly regarded is the adeno-associated virus (AAV).

The importance of AAV in the field of gene therapy cannot be overstated. Discovered in 1965, AAV has become the preferred vector due to its ability to efficiently transfer genetic material while exhibiting a relatively low immunogenic profile, meaning it is less likely to provoke an unwanted immune response. Additionally, AAV is known for its ability to transduce both dividing and non-dividing cells, making it a versatile tool for a variety of gene therapy applications. Its proven safety record, combined with its effectiveness in delivering therapeutic genes, has led to its widespread adoption in clinical trials and commercial gene therapies.

AAV-based gene therapies are already showing promise in treating conditions such as spinal muscular atrophy, hemophilia, and certain inherited forms of blindness, highlighting their growing role in modern medicine. However, as with any biopharmaceutical product, there are significant challenges associated with the development and production of AAV therapies, particularly in defining the appropriate Critical Quality Attributes (CQAs) that ensure the safety, efficacy, and consistency of the final product. Establishing these CQAs is a complex and highly regulated process, and failure to do so correctly can lead to inefficiencies, wasted resources, and even setbacks in the development process. Determining the best way to define and measure these CQAs—without wasting valuable time and financial resources—is a critical task for any organization working in AAV manufacturing.

 

This course will address these challenges head-on, providing valuable insights into how to effectively define CQAs for AAV products. It will explore strategies to optimize both the analytical methods and the manufacturing processes involved in the production of AAV-based gene therapies and their Quality Control. Participants will learn how to navigate these complexities efficiently and gain practical tools for improving the quality and scalability of AAV production, ultimately helping to bring these life-changing therapies to patients more quickly and reliably.