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


  • 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


  • 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


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


  • 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

  • Amy Wesa Senior Vice President, Preclinical & Translational Research, Vita Therapeutics


  • 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


  • 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


Improving Clinical Translational Strategies to Streamline Clinical Success

11:00 am Determining the Data Strategy Required for Regulatory Approval of IND Packages

  • Pinky Doshi Vice President, Regulatory Affairs, Shoreline Biosciences


  • 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


  • 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

  • Sonja Schrepfer Senior Vice President, Hypoimmune Platform, Sana Biotechnology


  • 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

  • Stefan Frank Associate Director, Induced Pluripotent Stem Cell & Platform Strategy, Bayer


  • 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

  • Weidong Zhang Vice President, Head of Biometrics, Sana Biotechnology


  • 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

  • Irina Klimanskaya Senior Director, Translational Science & Development, Astellas Institute for Regenerative Medicine


  • 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)

4:00 pm Close of Conference Day Two