7:55 am Coffee & Networking

8:55 am Chair’s Opening Remarks

  • Nathan Majewski Associate Director, Cell Therapy Analytical Development, BMS

9:00 am Panel Discussion: Assess Novel Technologies for Analytical Development

  • Anthony Leyme Senior Scientist, Analytical Development, Elevate Bio
  • Michael Sadick Senior Director, CMC Analytical Development, Precision Biosciences
  • Sean Bradley Scientist, Analytical Development & Cell Therapy, CRISPR Therapeutics
  • Radhika Raheja Senior Scientist, Analytical Development, 2seventy bio

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

  • Ben Hall Scientist, Analytical Development, 2SeventyBio

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

  • Liam Chung Scientist, Analytical Development & Quality Control, Century Therapeutics

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;

  • Radhika Raheja Senior Scientist, Analytical Development, 2seventy bio
  • Sean Bradley Scientist, Analytical Development & Cell Therapy, CRISPR Therapeutics

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

  • Chantal Martin Director, Analytical Development & Quality Control, Turnstone Biologics

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”

  • Michael Sadick Senior Director, CMC Analytical Development, Precision Biosciences

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

3:00 pm Chair’s Closing Remarks

  • Nathan Majewski Associate Director, Cell Therapy Analytical Development, BMS

3:05 pm Close of Conference