Johnny Lam & Kyung Sung, FDA-CBER. "Meet the FDA: Regulatory Science Insights into Cell-Based Products"

"Meet the FDA: Regulatory Science Insights into Cell-Based Products"

Kyung Sung, Ph.D., Principal Investigator

Johnny Lam, Ph.D., Staff Fellow

Cellular and Tissue Therapies Branch, Division of Cellular and Gene Therapies, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research

This event is held in partnership with UCSF-Stanford CERSI.

Where & When

Room 2103, Mission Hall, UCSF Mission Bay (550 16th St., San Francisco)
2:00 to 3:00 PM, Friday, November 9

As described in the 21st Century Cures Act, products eligible for Regenerative Medicine Advanced Therapy (RMAT) designation include cellular therapies, therapeutic tissue engineered products, human cell and tissue products, or any combination products that use such therapies or products. Multipotent stromal cells (MSCs) and induced Pluripotent Stem Cells (iPSCs) have been popular sources for manufacturing RMAT products due to their ability to undergo lineage-specific differentiation. Despite great promise, successful clinical translation of such cell-based products is often hindered by manufacturing challenges and the lack of reliable markers that can predict the products’ in vivo performance. For instance, MSCs are very heterogeneous and responsive to their surrounding environment, resulting in distinct subpopulations of cells with potentially different amounts of qualities needed for product potency. Since there are numerous biochemical and biomechanical factors regulating the functions of MSCs, it is critical to develop reliable high-throughput assays that enable the efficient exploration of large and complex parameter spaces for evaluating cellular function. Microscale in vitro systems offer the practicality to fulfill this unmet need. Several simple microfluidic channel arrays have been successfully implemented in screening the influence of paracrine mediators and various tissue microenvironment components in the regulation of cellular functions. In addition, microphysiological three-dimensional organoids and tissue-like structures such as chondrogenic cell aggregates and blood vessels have been incorporated into high-throughput, cell-based screening platforms in efforts to provide functionally relevant in vivo-like conditions. This presentation will give an overview of practical microscale technologies that are simple to operate while enhancing throughput, relevance, and reliability. How such technologies could be employed in the assessment of cell-based products will be discussed.

About the Speakers

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Dr. Johnny Lam is a biomedical engineer with expertise in biomaterials and in developing practical microscale in vitro tools for medical and biological applications. Dr. Lam’s main research interests involve studying multipotent stromal cells (MSCs, otherwise known as mesenchymal stem cells) and how their quality attributes relate to their functional potential using physiologically relevant, higher-throughput platforms. Dr. Lam received his Ph.D. in Bioengineering in 2015 at Rice University, where he developed and evaluated injectable multi-layered hydrogel composites for cell and controlled growth factor delivery for in vivo cartilage tissue repair. Following his graduate studies, Dr. Lam joined the Center for Biologics Evaluation and Research at the FDA as a post-doctoral researcher, where he now works as a Staff Fellow. His research now focuses on the development and adaptation of wide-ranging microphysiological platforms to evaluate various functional outcomes of MSCs toward improving the quality and potency of manufactured cell-based products.

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Dr. Kyung Sung is a biomedical engineer with expertise in developing functional and practical microscale in vitro tools for medical and biological applications. Dr. Sung’s main research interests lie in studying cell-materials interactions and exploring cell behavior in various tissue microenvironmental conditions. Dr. Sung received her Ph.D. in Chemical Engineering in 2007 at the University of Michigan, and worked as a post-doctoral researcher in the Department of Biomedical Engineering at the University of Wisconsin-Madison, where she also worked as a Principal Investigator before she joined the FDA in 2015. She also worked as a patent examiner in Biotechnology at the US Patent and Trademark Office. During her previous research, she used principles from tissue and microsystems engineering to develop tissue-like structures such as blood vessels and mammary ducts in microfluidic channels to develop new practical tools to conduct cancer research in vitro. The microscale in vitro systems provide unique capabilities when studying complex interactions occurring in tissue microenvironment, by providing more precise controls of biochemical and biomechanical factors than traditional platforms. She has been able to create innovative opportunities and strategies for researchers to explore biology in different ways – particularly in understanding the role of the tissue microenvironment in regulating cellular functions.