8:30 am Morning Refreshments & 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
10:00 am Structured Networking & Refreshment Break
Synopsis
As the analytical community is reunited, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. Running concurrently as an in-person and digital session, all attendees will have the opportunity to meet and network with their academic and industry colleagues.
Drug Substance Characterization
Confidently Measure Safety in Gene Engineered Next Generation Products
11:30 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
12:00 pm 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)
Meeting GMP in Analytical Process Development
Manufacturing Hurdles for New Devices and Process Changes
11:30 am 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
12:00 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
12:30 pm Lunchtime Refreshments & Networking
2:00 pm Round Table Discussion Sessions;
Synopsis
Join this discussion session to share ideas and collaborate with your colleagues on critical issues affecting the field.
3:00 pm 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
3:30 pm Vector Copy Number in Safety and Beyond: ddPCR as a Tool for Next-Gen Product Characterization
Improving Turnaround Times with Faster Analytics
3:00 pm Discussion Session: Achieving Faster Analytics Through Advanced PCR Technology
Synopsis
• Describe the benefits of ddPCR vs qPCR, including reduced variation, as well as a reduction in the number of samples needed
• Assess this as a faster and cheaper analytical tool
• Multiplexing assays to ensure faster release