7:30 am Registration & Coffee
8:20 am Chair’s Opening Remarks
Reflecting on iPSC Successes & Learnings to Understand the Prospects of iPSC-Derived Cell Therapies
8:30 am A Case Study to Benchmark the Road to Success For a Precision Engineered iPSC-Derived Effector Cell
Synopsis
- Experiences, learnings and challenges when moving to a PH1 clinical trial
- Laying the regulatory foundations
- Future directions with next-generation programs
9:00 am New Era of Heart Failure Treatment: Regenerative Cell Therapy Using Ventricular Specific Purified Cardiomyocytes
Synopsis
- Scalable method to eliminate undifferentiated iPSCs by difference of metabolism between iPSCs and cardiomyocytes
- Clinical data from FIH study of purified cardiomyocyte spheroids
- Platform technology to develop high quality iPSC-derived product
9:30 am Talk by our Expertise Partner, Cellino
10:00 am Morning Refreshment Break & Networking
DISCOVERY & TRANSLATION TRACK
Diving Into Differentiation to Optimize iPSC-Derived Cell Functions & Fulfill Clinical Potential
11:00 am Directing Differentiation to High Purity Cell Populations Appropriate for Cellular Therapy
Synopsis
- Examining current and novel technologies to successfully characterize undifferentiated cells to reduce product contamination
- Diving beyond karyotypic screening to drive reliable assessment of genomic integrity
- Evaluating the role maturity plays in differentiation to understand why iPSCs do not always fully differentiate into their potential
11:30 am Exploring the Downstream Effects of Genetic Editing on Differentiation to Better Understand the Behavior of Differentiated iPSCs
Synopsis
- Understanding the biological underpinnings causing different responses in differentiated cells to facilitate earlier intervention
- Discussing potential opportunities for earlier detection of functional effects on differentiated cells
- Reviewing options to modify differentiation protocols following unexpected changes due to upstream genetic modifications
12:00 pm Roundtable Discussion: Improving Efficacy in Differentiation Protocols to Enhance Therapeutic Potential
Synopsis
- Exploring best practices to characterizing differentiation success beyond phenotypes to include functionality assessments to produce desired therapies
- Discovering how to differentiate cells into more complex cell types (for example, endoderm) to advance therapeutic capacities
- Debating technological options for assessing functionality post differentiation to boost clinical success
REGULATORY & EARLY CLINICAL DEVELOPMENT TRACK
Improving Clinical Translational Strategies to Streamline Clinical Success
11:00 am Determining the Data Strategy Required for Regulatory Approval of IND Packages
Synopsis
- Learning which findings are of maximum importance to regulatory bodies to ensure they are prioritized in characterization strategies
- Understanding the extent of genetic diversity that is acceptable to regulatory bodies to strengthen IND packages
- Deciding on the necessary information required at the early stage of regulatory approval to create a harmonious development strategy
11:30 am Roundtable Discussion: Considering Clinical Strategy Timelines & Expectations to Improve Clinical Tolerance
Synopsis
- Determining patient selection criteria to target your clinical trial populations
- Benchmarking the best approaches for administration to improve patient compliance
- Understanding the infrastructural and procedural requirements for your selected delivery method to ensure safe and cost-effective administration
12:00 pm Talk by our Program Partner, Catalent
The Future is iPSCs: Re-Evaluating Translational & Strategical Priorities to Drive Next Steps Across the iPSC Industry Chair – Sofia Håkansson Buch, Vice President, Cell Therapy CMC, Novo Nordisk
1:30 pm Genetic Engineering of Allogeneic Donor Cells For Acceptance by the Host’s Immune System
Synopsis
- Overcoming the immune barrier in allogeneic cell transplantation – discussion of the concept of immune evasion
- Understanding the immune response to different iPSC-derived cell types in different transplantation sites
- From mice to patients – various preclinical and translational models to bridge the gap from bench to bedside
2:00 pm Discovering How to Make iPSC Cell-Derived Therapies a Reality: A Pharma Perspective
Synopsis
- Benchmarking pharma priorities when translating a smaller scale process to a larger scale
- Streamlining the process to clinical success
- Highlighting common challenges and solutions of iPSC platform strategies to turbocharge iPSC therapies to the clinic
2:30 pm Afternoon Break & Networking
3:00 pm Evidence-Based Quantitative Decision Making in Cell Therapy Clinical Development
Synopsis
- Developing an evidence-based quantitative decision framework
- Benchmarking endpoints and data package development for decision making
- Discussing criteria for a go/no-go decision based on pre-specified target and/or reference values
3:30 pm Scaling Up Differentiation Process Efficiently: Key Parameters Defined by Cell Biology, Technology, & Regulatory Requirements
Synopsis
- Understanding the type of differentiated derivatives of PSC defines the manufacturing process (including PSC expansion, differentiation, expansion of derivatives) to overcome unique challenges
- Scaling up and compliance with regulatory requirements often requires additional optimization of the process due to impact of raw materials and GMP compliant procedures on cell health and properties
- Appreciating upgraded process needs to produce the same quality drug product (identity, purity, potency, genomic stability, absence of PSC, in vivo efficacy)