 Dr. Zuben Sauna
Director, Division of Hemostasis, Office of Plasma Protein Therapeutics, Office of Therapeutic Products, Center for Biologics Evaluation and Research
Food and Drug Administration
United States of America |
Keynote Speech:
New Approach Methodologies (NAMs) in the assessment of immunogenicity during drug development
Abstract:
The FDA’s roadmap for advancing New Approach Methodologies (NAMs) identifies unwanted immunogenicity as a priority area, emphasizing scientifically validated alternatives to animal testing. Progress in the last two decades has allowed the development of an integrated framework combining in silico, in vitro, and ex vivo NAMs for comprehensive immunogenicity assessment. The tools include: (i) In silico approaches such as T-cell epitope prediction algorithms, AI/ML structural modeling, and population HLA diversity analysis. (ii) In vitro methodologies that encompass dendritic cell-T cell activation assays, cytokine profiling panels, and mass spectrometry-based tools to identify T-cell epitopes. (iii) Ex vivo platforms that utilize HLA-stratified PBMC and whole blood assays from genetically diverse donor cohorts to predict human immune responses. In addition, emerging tools such as small ankyrin repeat proteins called Ankyrons that bind with high affinity to diverse protein targets can be used to interrogate immune mechanisms.
Here I will provide specific examples of the use of NAMs in addressing immunogenicity issues during drug development. I will also discuss the complexities of evaluating the immunogenicity risk of diverse novel modalities and how platform-specific immunogenicity assessment strategies can be developed. Key unmet needs will also be identified and the need for collaborative efforts between industry, academia, and regulatory agencies to standardize tools and develop reference reagents will be emphasized.
Biography:
Dr. Zuben E. Sauna serves as Director of the Division of Hemostasis at the FDA’s Office of Plasma Protein Therapeutics, leading a team of over 34 reviewers and research scientists. An internationally recognized thought leader in regulatory science, Dr. Sauna’s laboratory has published groundbreaking research in high-impact journals including Nature Biotechnology, Nature Medicine, and Science Translational Medicine. His work pioneered immunogenicity risk assessment strategies now routinely used by pharmaceutical companies and advanced the study of immune responses to gene editing technologies.
With over 106 publications garnering more than 13,000 citations, Dr. Sauna has received numerous honors including the NIH Merit Award and multiple FDA scientific achievement awards. He holds a Ph.D. in Biotechnology from Poona University and has delivered over 60 invited presentations at national and international conferences, establishing himself as a sought-after expert in biotherapeutic development and immunogenicity assessment.
|
