Conference Day One
7:45 am Chair’s Opening Remarks
Supercharging Upstream Process Development to Minimize Cost of Goods and Maximize Product Consistency
8:00 am Case Study: Identifying Critical Quality Attributes to Ensure Clinical Safety and Efficacy
Synopsis
• Defining critical quality attributes for your cell therapy such as polyfunctionality, cytokine secretion, persistence in line with IHC guidelines
• Designing assays to measure critical quality attributes and ‘global picture’ of your cells, and finding the best markers
• Considering how critical quality attributes and the assays used to measure them translate to a clinical setting
• Feeding back from the clinic to inform assay design
8:30 am Case Study: Expanding Cells to Retain Desired Product Characteristics
Synopsis
• Minimizing or removing the expansion phase and examining the impact on cell count and product implications
• Managing heterogeneous cell starting materials and developing reactive expansion and manufacturing protocols to funnel heterogeneous cells to homogenous products
• Scaling expansion and identifying more ‘gentle’ cell processing techniques to maintain critical quality attributes
9:00 am Driving the Wave of Non-Viral Engineering to Minimize Time, Complexity and Cost of Processes
Synopsis
• Discussing how non-viral engineering changes the overall process vs. viral-engineered cell therapies
• Examining safety profiles and the consequences of unintended editing, and setting up processes to mitigate risk
• Starting with the end in mind to design scalable, cost-effective, reliable processes for high-quality cell production using next-generation technologies from early-stage development
9:30 am Case Study: Creating and Closing Processes for Next-Generation Cas9-Engineered T Cell Therapies with Large Payloads
Synopsis
• Integrating large modules and circuits into T cells with DNA plasmid vectors and Cas9 engineering
• Closing processes to increase sterility, process control, and safety
• Discussing the challenges of leveraging novel engineering technologies in process development, and sharing lessons learned so far
10:00 am Morning Refreshments & Speed Networking
Streamlining Processes to Minimize Time and Cost of Production
11:00 am Case Study: Convening on Regulations to Establish Frameworks and Best Practices for the Industry
Synopsis
This deep-dive will be a series of presentations, roundtables, and problem-solving group activities to provide a framework of experience with regulatory filings in Europe and South America, and to discuss key questions for the field with the session leaders to help guide your own regulatory journey.
• Communicating processes and aligning with regulatory requirements and achieving compliance with SOPs and controls
• Discussing the published guidance on process development for cell therapies
• Highlighting the gaps in how generalized regulations apply to process development
• Generating supporting documentation in process development to justify changes from PD to QC
• Highlighting the different experiences in Europe and the US with materials, quality and process requirements
• Managing the evolving regulatory environment alongside product heterogeneity and developing assays and processes to enable flexibility
TECHx Talks
Synopsis
Each speaker will have 15 minutes to present their ‘use case’ for a novel technology, how they implemented it, what challenges they faced, and what they learned through this process development with novel tech
12:30 pm Discussing How Product Characteristics Can Streamline Process to Improve Process Simplicity and Reduce COGS
Synopsis
• Characterizing tumor infiltrating lymphocyte product expressing membrane bound IL15 and impact of process on product quality
• Improving process design to leverage product characteristics and optimizing cytokine supplementation
• Discussing challenges and lessons learned in this process development, and benefits from cytokine regimen optimization
12:30 pm Case Study: Moving Tech In-House to Reduce Reliance on Third Parties
Synopsis
• Developing tech in-house, to automate and build out digital systems and reduce third party expenditure
• Outlining the journey through development from building a business case, building teams, and developing tech
1:00 pm Lunch & Networking
Advancing Allogeneic Process Development for Scalable Off-the-Shelf Cell Therapy
2:00 pm Bringing Allogeneic CAR-T Cell Therapies to Fruition by Developing Strategies for Multiplexed Engineering using chRDNA Genome-Editing Platform
Synopsis
• Creating processes to produce highly efficacious allogeneic cell therapies with potential to expand to new markets
• Sharing engineering strategies to enhance persistence of allogeneic cells that may translate to improved clinical outcomes
• Establishing dosage requirements to inform batch size and manufacturing scale for off-the-shelf therapies
2:30 pm Case Study: Establishing Processes for iPSC-Derived Cell Therapies and Managing the Drug Product Workflow
Synopsis
• Achieving and maintaining the most pluripotent state, and reflecting ‘natural’ cell phenotypes upon lineage differentiation
• Scaling up iPSC-derived therapy platforms, outlining the number of batches produced and how to define a batch
• Transferring manufacturing processes from auto to allo, identifying areas of synergy to streamline needs for training, GMP space
• Demonstrating safety and purity of iPSC-derived products for clinical application
3:00 pm Panel Discussion: Reflecting on the Trials, Tribulations, Victories and Remaining Hurdles in Process Development Across Contrasting Allogeneic Sources
Synopsis
• Contrasting the process development steps, and relative advantages and pitfalls between different allogeneic sources from iPSC-derived, healthy donor, and umbilical cord-derived cells
• Highlighting synergies and learnings that can be applied from the autologous field, and areas where innovation and a ‘clean slate’ is required to optimize platforms
• Sharing experience of process development for allogeneic cell therapies: the trials, tribulations and opportunities
3:45 pm Case Study: Scaling Up Allogeneic Therapies to Move Towards Industrial-Scale Production and Defining a ‘Batch’ for Off-the-Shelf Products
Synopsis
• Establishing the fundamental characteristics of an allogeneic platform
• Developing flexibility in processes to enable scalability through phases in line with market need
• Formulating and freezing large batches, defining a ‘batch’ for allo therapies, and communicating this with regulators
• Managing scalability with a limited donor pool of minority cell populations, and demonstrating product safety and efficacy at scale