7:55 am Coffee & Networking
8:55 am Chair’s Opening Remarks
9:00 am Panel Discussion: Assess Novel Technologies for Analytical Development
Synopsis
• Stress the need for higher throughput technologies, with less variability and greater user ease
• Regulatory agency guidelines on the use of new technologies
• Compare thoughts on ddPCR Vs qPCR … Elisa Vs Ella testing
Confidently Measure Safety in Gene Engineered Next Generation Products
9:45 am Addressing Safety Assessment in Next Generation Products
Synopsis
• Autologous cellular therapies that incorporate gene editing require safety assays
to ensure absence of aberrant transformation in final drug product
• These assays include evaluating chromosomal abnormalities, IL-2 independent
growth as a surrogate for malignant transformation, and changes resulting from
on- or off-target editing
• Challenges with these assays such as determining acceptance criteria, qualifying
QC sites for assay implementation, and long turn-around-time can ultimately
impact needle-to-needle time
• Long-term goals are to build robust analytical tools, generate sufficient
characterization data, and continually engage with regulatory agencies to
mitigate challenges and ensure safety of final drug product
10:15 am Analytical Tools for Characterization and Safety Testing of Gene Edited Allogeneic CAR-T Cell Therapies
Synopsis
• Introduction to production and safety considerations associated with allogeneic CAR-T products
• Review assays and techniques to characterize efficiency of gene editing
• Examine analytical methods to assess product safety profiles specific to gene editing processes (flow cytometry, qPCR/ddPCR, ELISA, and NGS)
10:45 am Refreshment Break
Synopsis
Share your work with the experts of the analytical community and gain feedback
from those onsite!
11:45 am A Risk-Based Approach for Establishing Genomic Integrity and Stability for Genetically-Engineered, Allogenic Cell Therapy Products
Synopsis
• Discuss challenges of evaluating off target integration in genetically engineered products
• Discuss different approaches on off target assessment
• How to evaluate change in variants in cell therapy product throughout the manufacturing process
12:15 pm Round Table Discussion Sessions;
Synopsis
Join this discussion session to share ideas and collaborate with your colleagues on critical issues affecting the field.
1:00 pm Lunchtime Refreshments
Manufacturing Hurdles for New Devices and Process Changes
2:00 pm When the Process is the Product, Analytical Testing to Support Tech Transfer and Manufacturing Changes
Synopsis
• Address analytical challenges that are encountered as a result of process changes
• Discuss how your analytical toolbox can support process changes and comparability
• Consider how a robust analytical control strategy can support a seamless process tech transfer
2:30 pm Resetting Expectations for CAR-T Potency Assessment… “Baby, You Can Drive My CAR”
Synopsis
• ‘Full serial dilution curve’ bioassays have been difficult to establish for cell therapy modalities, but they provide critical assessment of potency/relative potency
• Target cell cytotoxicity assays may well be more MOA reflective than cytokine release bioassays
• This presentation will describe successful utilization of a luciferase-complementation-based cytotoxic assay (Promega HiBiT assay)
• Application of this assay format to support of cellular therapies will be discussed