Kick start day two with clinical case study reviews, group discussion forums, and new updates on the full end-to-end drug development landscape for allogeneic cell therapies.
Leaders from; AgenTus, Bone Therapeutics, Bristol Myers Squibb and TCR2 will be showcasing:
- Novel strategies to overcome safety and tolerability risks
- Processes to achieve reproducibility and comparability in manufacture
- Experience navigating global regulatory challenges for off-the-shelf production
… and much more!
Key speakers include:
9:30 - 5:05 EDT | 6:30 - 2:05 PDT - All times are shown in EDT
9:30 am Virtual Coffee Networking Rooms
10:00 am Chair’s Opening Remarks
Keynote Clinical Case Studies
10:05 am Realizing the Clinical Potential of CRISPR/Cas9 for Best-in-Class Allogeneic Therapies
Synopsis
• Showcase clinical data from CTX110, CTX120 and CTX130 to review how allogeneic cells function in vivo
• Analayze depth of response, persistence and durability of the allogeneic cells to achieve complete responses
• How do allogeneic cells perform compared to autologous cell therapies?
10:35 am Learnings from Allogeneic CAR-T Clinical Trials
Synopsis
• Discussing patient conditioning without biologic immuno-depletion
• Outlining dosing considerations
• Evaluating cell expansion, persistence, and durability of response
11:05 am How an Effective Source Material Supply Chain Can Enhance Scalability of Cell and Gene Therapies
Synopsis
- Analyzing the impact of source material variability and characterization on downstream efficacy of cell therapy applications
- Utilizing reliable/recallable and dedicated donor pool strategies to ensure consistency and reproducibility
- Identifying how cell supply partners can help mitigate risk for clinical translation of allogeneic therapies
11:35 am Differentiated Non-Gene Edited Technologies to Generate Off-The-Shelf CAR-T Cells for the Treatment of Hematopoietic and Solid Malignancies
Synopsis
• Leveraging non-gene edited technologies to disrupt TCR signaling to generate allogeneic CAR T cells
• Exploiting natural receptor/ligand interactions for CAR T cell therapy of metastatic colorectal cancer
• Discussing the first-in-class shRNA-based BCMA-targeting allogeneic CAR T candidate for the treatment of multiple myeloma
12:05 pm Tech Slam – Benchmark Gold-Star Donor Material With Leading Blood Bank Providers
Synopsis
From Clinic to Commercial Reality: Leveraging Transplant Experience to Overcome Cell Therapy Supply Chain Hurdles
Joy Aho, Senior Product Manager, Be The Match BioTherapies
12:15 pm Refreshment Break
Synopsis
Take this time to follow up with your new connections with a 1-1 or group video call or take a break and make a coffee!
1:00 pm Panel Discussion: Best Practices to Prevent Rejection and Achieve Long Lasting Responses
Synopsis
• Review methods to prevent rejection and improve long term durability of allogeneic cells
• Compare the different safety profiles of alternative cell types and the qualities that prevent rejection
• Understand the best in class approaches for achieving increased depth of response and efficacy
1:45 pm Increase Safety by Managing GVHD
Synopsis
• Review cell types like NK and NKT that have advantages in safety with no GVHD, and increased tolerability
• Explore innovative gene editing to overcome GVHD
• Review the toxicity risk of incomplete purification of TCR positive cells from the product, which can lead to random pairing and GVHD
• Discuss if HLA typing matter? Review different approaches of mismatching vs. knock out
1:00 pm Panel Discussion: Does a Super Donor Exist?
Synopsis
• Benchmark strategies to select final donors, list the key requirements that are explored beyond HLA matching
• Can an ultimate donor be consistent across multiple samples when the body’s immunology changes constantly
• Generally what attributes have been identified in workflow which correlates with better efficacy,
persistence, and engraftment
• Understand what the attributes are to achieve a super donor, how does this differ for stem cells Vs T cells
1:45 pm Comparability Testing for US Required Raw Material Sourcing Vs. European Sourcing
Synopsis
• Outline comparability protocol
• Describe extended characterisation
• Reference data
• Specific considerations for off-the-shelf cell products including batch size for multiple patients per batch
2:15 pm Workflow Solutions for Scalable Pluripotent Stem Cell Manufacturing
Synopsis
• Begin with the end in mind: identify suitable ancillary materials early in development
• Fit products to process: optimize workflows with consistent, animal-free and GMP-grade raw materials with future need and flexibility built in
• Build from the beginning to scale: solutions for a smooth transition from research to clinical applications
1:00 pm Panel Discussion: How to Optimize Supply and Storage of Off-the-Shelf Cell Therapies
Synopsis
• Assess the supply chain complexities when building a large-scale trial and commercial product
• Discuss the regulatory challenges when utilizing donor materials in different countries, how can this effect a product from being truly off-the-shelf
• Describe novel cryobiology to improve the feasibility of transport and storage over time
1:45 pm Stem Cell Therapy Standardisation
Synopsis
• The use of ethically approved and quality controlled stem cell lines is crucial to support high quality research and healthcare developments
• Appropriate stem cell standards and controls, as well as suitable qualification assays are essential to support the translation of scientific discoveries into effective advanced stem cell-based therapies
2:25 pm Refreshment Break
3:00 pm Roundtable Discussion Session – Innate Vs. Adaptive Immune Cell Types for Allogeneic Therapy
Synopsis
• Assess the different characteristics of NK, GDT and T cells and how this can impact their success as an allogeneic therapy
• Discuss current clinical and preclinical results, specifically assessing the potential to persist and safety profiles
3:45 pm Overcoming Safety and Tolerability Risks Cas-Clover™ Gene-Editing of TRBC and B2M Yields a Safe and Effective Off-the-Shelf BCMA CAR-T Product
Synopsis
• Explain how the Cas-Clover activity is efficient and highly specific in multiplexed editing of TRBC and B2M
• Combination of Cas-Clover gene-editing with Poseida’s proprietary piggyBac® gene delivery, booster, and process, yields a highly effective and durable allogeneic BCMA CAR-T product
• The genotoxicity assessment of GMP products reveals that Poseida’s P-BCMA-ALLO has an exceptionally safe profile: consistently low off-target editing and translocations frequencies (<2%), as determined by industry standard methods
4:10 pm Engineering Off-the-Shelf T Cell Receptor Fusion Construct (TRuC™) T cells
Synopsis
• T Cell Receptor Fusion Construct (TRuC™) T cells are engineered to express a fusion protein that comprises an antibody-derived binder tethered to the CD3ε signalling subunit
• Upon integration of the TRuC into the TCR, it recognizes tumor surface antigens independent of HLA and uses the complete receptor complex to trigger a comprehensive T cell response
• Describe the engineering of off-the-shelf TRuC-T cells directed against mesothelin, a validated solid tumor antigen, with engineering strategies to eliminate alloreactivity and reduce the risk of GvHD
3:40 pm Autologous vs. Allogeneic; Key Considerations for a Manufacturing Process
Synopsis
• Meeting product demand
• Building cost-effectiveness
• Ensuring process robustness and control strategy
3:40 pm Clinical Delivery Considerations to Realize the Power of RED™: A New Era in Cellular Medicine
Synopsis
• Describe the RED™ allogeneic cell therapy product and delivery to clinical sites
• Explore the factors for success in implementing a new cellular therapy – pharmacy delivery, allogeneic, off the shelf, liquid delivery, no lymphodepletion regimens
4:10 pm Harmonizing Regulatory CMC Challenges for Allogeneic Cell Therapies
Synopsis
• Update on the current regulatory guidance
• Address the donor cell test requirement issues
• Outline ARM’s efforts to streamline regulatory CMC challenges across the US and EU