Conference Day Two
Thursday, December 5, 2024
8:00 am Check In & Morning Coffee
8:45 am Chair’s Opening Remarks
Overcoming Ongoing Analytical Development Hurdles in Cell Therapy to Accelerate Therapeutic Advancements
9:00 am Optimizing Analytical Validation for Commercialization Success in Product Development
Synopsis
- Exploring methods to strategically validate assays, minimizing the number required for commercialization while ensuring regulatory compliance.
- Discussing the transition towards PAT to accelerate testing timelines, with a focus on stability assessments and early data acquisition.
- Investigating innovative approaches to obtain stability data earlier in the manufacturing process, enabling expedited release of the final drug product.
9:30 am Agilent’s Cell-based Analytical Solutions for Process Development and Manufacturing of Immune Cell Therapies
Synopsis
- Overview and performance analysis of Agilent’s real-time, label-free cell analysis platforms for assessment of critical quality attributes of cell therapy products
- Discuss the advantages of metabolic fitness and immune cell killing functional assays during the evaluation of critical process parameters for manufacturing of engineered immune cells
- Explore using in-process monitoring of cell identity, potency and metabolic fitness to shorten cell therapy manufacturing process.
10:00 am Round Table: Top Five Regulatory & CMC Highlights to Advance the Field in 2024
Synopsis
- Decentralized/distributed manufacturing.
- Potency assays and assurance.
- New designations – AMTD and PTD.
- Acceleration of CMC in advent of expedited clinical programs.
- iPSC-based therapies.
10:30 am Morning Break & Speed Networking
Track A – Cell Based Assay
Track B - Immunophenotype Assay
Developing Standardized Protocols for Characterization Across the Cell Therapy Modalities
11:30 am Standardizing Cell-Based Assays for Diverse Cell Therapy Modalities to Ensure Consistency & Reproducibility
Synopsis
- Investigating techniques to ensure methodological consistency across cell-based assays.
- Exploring quality control measures such as assay validation, inter-laboratory proficiency testing, and data normalization techniques.
- Discussing the integration of advanced technologies such as automation, microfluidics, and high throughput screening platforms.
12:00 pm Single-Cell Multi-omic Assessment of Gene Editing Outcomes, Toxicity, & VCN for High Resolution Characterization of Cell Therapies
Synopsis
- Despite advancements in cell therapy engineering, conventional methods for measuring gene transfer and gene editing techniques lack the resolution and representation to truly reflect sample composition and either report a population average (bulk) or involve laborious and time-consuming clonal outgrowth.
- Mission Bio has developed an end-to-end solution from panel design to data analysis for single-cell targeted DNA sequencing to interrogate transgenes to confirm the distribution of vector copy number (VCN).
- Additionally, the solution simultaneously measures the co-occurrence and zygosity of on-target edits, off-target edits, translocations between predicted edit sites, as well as the genomic CNV landscape (including focal CNV) in over thousands of cells in parallel to reveal potential genotoxicity events in a single-cell context.
12:10 pm Panel Session: Contrasting Characterization Strategies Across Cell Therapy Modalities to Understand the Diversity of Cell Therapy Platforms
Synopsis
- Explore the unique challenges and considerations in characterizing immunotherapy products, including antigen specificity, T-cell functionality, and cytokine secretion profiles.
- Discuss characterization strategies for stem cell-based therapies, covering pluripotency assessment, differentiation potential, and genomic stability profiling.
- Compare and contrast characterization approaches between autologous and allogeneic cell therapies, examining donor variability, immune compatibility testing, and scalability considerations.
Optimizing Sample Management & Handling for Enhanced Immunophenotype Assays
11:30 am Process Analytical Utility of Raman Microscopy for Allogeneic Cell Therapy Manufacturing
Synopsis
- Continued advances in laser technology and data analytics are increasing the process analytical utility of spectroscopy.
- Cell composition changes measured by Raman micro-spectroscopy distinguish many cell types, even the accumulation of insulin in cells to treat diabetes.
- Information-rich, non-invasive spectroscopic technologies should complement conventional quality assurance methods.
12:00 pm Innovation Partner Presentation
Synopsis
12:10 pm Navigating Miniaturization Challenges by Balancing Precision with Sample Constraints
Synopsis
- Analyzing the current landscape of miniaturization efforts in analytical assays and the driving factors behind the push for smaller sample volumes.
- Discussing the technical challenges associated with miniaturizing assays, including maintaining sensitivity, reproducibility, and accuracy while reducing sample volume.
- Evaluating the need for standardized approaches to miniaturization in analytical assays, exploring potential solutions, and addressing the lack of uniformity across the industry.
12:40 pm Lunch Break & Networking
Strategies to Address Variability in Cell-Based Assays for Enhanced Reliability
2:00 pm Approaches to Activate Human Gamma-Delta T Cells for Immunotherapy Treatment
Synopsis
- Human gamma-delta T cell activation and suppression.
- Tumor cell killing and immune evasion.
- Gamma-delta T cell-based immunotherapy.
2:30 pm Functional Characterization of Cell Therapies with Real-Time Live Cell Analysis for Accelerated Efficacy Assessment
Synopsis
- Discover cutting-edge tools that simplify and enhance CAR T-cell research and manufacturing workflows, providing efficient and scalable options for functional characterization and therapeutic assessment.
- Learn how the Maestro Z platform enables robust, non-destructive evaluation of CAR T cells by supporting real-time, label-free monitoring of cytotoxicity.
- Explore new data on a streamlined protocol that enables rapid bioactivity assessment using an impedance-based assay, supporting accelerated decision-making in cell therapy development.
2:40 pm Roundtable Discussion: Navigating Inherent Limitations in Cell-Based Assays: Strategies for Improved Reliability & Reproducibility
Synopsis
- How can we better understand and mitigate the sources of variability in cell-based assays to improve reliability and reproducibility of results?
- What alternative assay formats or technologies can be employed to complement or replace cell-based assays in situations where variability poses significant challenges?
- What quality control measures and best practices can be implemented to minimize test-retest variability and enhance the robustness of cell-based assays for analytical testing?
Advancing Immunophenotype Assays with Technologies & Methodologies for Cell Therapy Excellence
2:00 pm Strategies for Designing Quality Control-Friendly Flow Cytometry Panels in Cell Therapy Development
Synopsis
- Explore strategies for designing flow cytometry panels that balance the need to cover product characteristics and impurities while adhering to quality control-friendly limitations, such as color restrictions.
- Examine the implications of the recent FDA potency assay draft guidelines on cell therapy development programs, including the importance of studying mechanism of action from early phases and ensuring compliance with evolving regulatory standards.
2:30 pm Fit for Purpose Immunophenotype Characterization in Cell Therapy Development, From Draw-To-Thaw™
Synopsis
- Characterization of donor variability to assure optimal cell sourcing
- Characterization of starting material to assure identity and quality
- Monitoring the identity and cell health during manufacturing to improve success rate
- Release testing of critical quality attributes to assure the quality of final product
2:40 pm Overcoming Challenges in Qualifying Flow Cytometry Assays for Cell-Based Therapies
Synopsis
- Ensuring reproducibility is challenging due to the complexity and heterogeneity of cell populations and the variability in sample preparation and instrument performance.
- Developing robust, standardized protocols and maintaining regular calibration and maintenance schedules for flow cytometers are essential to achieving consistent and reliable results.
- Utilizing advanced data analysis tools, consistent gating strategies, and early engagement with regulatory bodies can help manage the complexity of data and ensure compliance with regulatory standards.
3:10 pm Afternoon Break & Networking
Scaling Up Success with Strategies for Efficient Cell Therapy Production & Regulatory Compliance
3:45 pm Seamlessly Integrating Analytical Technologies in Cell Therapy Development While Ensuring Compliance & Efficacy
Synopsis
- Discuss strategies for seamlessly integrating new analytical technologies into existing laboratory workflows and data management systems.
- Explore the importance of rigorous validation and qualification processes to meet regulatory standards for accuracy, precision, and reliability.
- Highlight the necessity of collaboration between analytical scientists, regulatory affairs professionals, and clinical researchers to ensure the technology meets the specific requirements of marketing authorization and clinical studies.
4:15 pm Use of Cell Mimics as In Process and Potency Controls in Cell Therapy Development
Synopsis
- Explore the limitations of biological controls examining variability and resource demands that compromise reproducibility in validation assays.
- Discuss the application of cell mimics as scalable, reproducible solutions that address the limitations of biological materials
- Demonstrate how cell mimics help standardize key aspects of the cell therapy process and support regulatory compliance
5:00 pm Overcoming Challenges in Characterization of Scaled-Up Cell Therapy Products
Synopsis
- Explore strategies for ensuring consistency in product quality and characteristics as cell therapy production scales up, addressing factors such as variability in raw materials, manufacturing processes, and environmental conditions.
- Discuss innovative approaches to sampling and analytical methods to obtain representative samples from larger volumes and adapt traditional techniques for use at scale, facilitating accurate characterization of scaled-up products.
- Examine the complexities introduced by large-scale cell culture systems, including nutrient and oxygen gradients, shear forces, and cell-cell interactions, and discuss strategies for understanding and mitigating these factors to ensure predictable product behavior and consistent characterization across scales.
5:30 pm Chair’s Closing Remarks
5:35 pm Close of Conference
