7:50 am Registration & Coffee

8:40 am Chair’s Opening Remarks

Reaffirming the Future of iPSC-Derived Cell Therapies to Benchmark the Road to Clinical Success

8:50 am iPSC Leaders Panel: Highlighting the Next Steps for the Future of iPSC-Derived Cell Therapies


  • Reflecting on how far the field has come to reaffirm iPSCs as the cornerstone of regenerative cell therapies
  • Understanding the prospects of iPSC and laying out the trajectory to unite industry experts to propel regulatory approval
  • Discussing the progression of, and clinical readiness for, iPSC technologies to identify areas in need of greater attention

9:30 am A Phase 1/2a Study Using Autologous iPSC-Derived Retinal Pigment Epithelium for Treatment of Advanced Dry Age Related Macular Degeneration

  • Kapil Bharti Senior Investigator, National Institute of Health Science


  • What is the biological need for RPE replacement as a potential therapy for AMD?
  • How to translate an autologous iPSC-based therapy from bench-to-bedside?
  • What are the future disease specific applications of a RPE-based cell therapy?

10:00 am Roundtable Discussion: To Auto or to Allo? Evaluating the Future of iPSC-Derived Cell Therapies to Inform Our Next Steps

  • Armin Rath Head of New Product Development & Research Alliances, Cytovia Therapeutics
  • Irina Klimanskaya Senior Director, Translational Science & Development, Astellas Institute for Regenerative Medicine


  • Considering the practicalities of a scaled-up autonomous approach to re-evaluate its clinical acceptance
  • Understanding the transection between autologous and allogeneic translational approaches to promote a holistic iPSC strategy
  • Identifying how industry, service providers and academia can collaborate to support the advancement of iPSCs to the clinic

10:30 am Investigated Stem Cell Genetic Integrity Using NGS Testing: Turning a Deep Analysis Into a Straightforward Interpretation

  • Reda Zenagui Manager, Research & Development, Genetics and Bioinformatics, Stem Genomics


  • Introducing the latest Next-Generation Sequencing (NGS) genomic stability assay range
  • Enabling the detection of copy number variations (CNVs) such as the 20q amplicon and single nucleotide variations (SNVs) in genes such as TP 53 and BCOR amongst many more.
  • Providing an end-to-end workflow solution that includes personalized bioinformatics analyses for comprehensive interpretation of the obtained data.

10:40 am Morning Refreshment Break & Speed Networking


Optimizing Gene Editing Protocols for Improved iPSC Product Safety

11:40 am Improved Anti-Tumor Immune Function of TGFβR2 Knock Out and IL-15 Knock In iPSC-Derived NK (iNK) Cells by TALEN® Editing


  • The off-target effect of TALEN pairs were detected, and these TALEN®-edited iPSCs kept their pluripotency, exhibited normal morphology and karyotype.
  • Engineered iNK cells with IL-15 KI and TGFβR2 KO consist of a homogeneous population of CD56+ NK cells (>99%) with the expression of several typical NK markers.
  • The iNK cells with TGFβR2 KO are resistant to TGFβ-mediated suppression of activating receptors and conferred resistance to TGFβ-mediated suppression of cytotoxicity against HCC tumor cells.

12:10 pm Talk by our Innovation Partner, Cell Microsystems

12:20 pm Panel Discussion: Benchmarking Safety Considerations During Gene Editing Protocols to Ease Regulatory Burdens

  • AnPing Chen Associate Director, Cytovia Therapeutics
  • CiCi Shi Head of Technology Incubation Engine, Oncology Cell Therapies, Takeda
  • Yinpeng Zhan Scientist, Sorrento Therapeutics Inc.


  • Balancing therapeutic potential of immortalized cells with safety considerations
  • Discussing best practices for combatting offsite mutations to reduce adverse effects
  • Monitoring cell behavior to prevent product contamination


Refining Manufacturing Strategies to Increase Efficiency in CMC Protocols

11:40 am Developing a Scalable Process Using a Quality by Design Approach for High Yield Bioprocessing of Cell Therapy Products in Stirred Suspension-Based Bioreactor

  • Shri Joshi Director, Process Development, Notch Therapeutics


  • Scalable and controllable manufacturing of pluripotent stem cells with iterative process development
  • Aligning cell manufacturing with QbD
  • Process optimization and characterization using bioprocess solutions including automated feeding and in-process monitoring.

12:10 pm Talk by our Program Partner, PBS Biotech

12:40 pm Exploring Technologies to Enable Population-Wide Personalized iPSC Production

  • Leo Lei Associate Professor, Penn State


  • Reaffirming the fundamental biology underpinning iPSCs to enable personalized medicine and therapy
  • Benchmarking manufacturing challenges preventing the commercial development of iPSCs based personalized medicine and therapy
  • Discuss technologies and innovation to enable population-wide personalized iPSC production

1:10 pm Lunch Break & Networking


Strengthening Preclinical Models to Improve Translation of Data to the Clinic

2:10 pm Tailoring the Preclinical Approach for Effective Translation of a Cell (-Derived) Therapy for Ischemic Stroke

  • Dang Dao Director, Research & Development, Astellas Institute for Regenerative Medicine


  • Aligning in vitro potency with relevant disease biology to inform potential therapeutic efficacy
  • Using in vivo models to understand facets of translation that lead to improved efficacy
  • Overcoming challenges in clinical development through better understanding of optimal delivery

2:40 pm Roundtable Discussion: Combatting Limitations with Preclinical Models to Improve Translation of iPSC-Derived Therapies to the Clinic

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


  • Acknowledging current limitations with existing animal models to identify areas for greater discovery
  • Discussing alternative approaches to enhance quality of preclinical efficacy, safety and toxicology data
  • Benchmarking critical qualities of preclinical models to ensure accurate reflection of disease indications


Discussing Data Requirements for Regulatory Approval of iPSC-Derived Cell Therapies

2:10 pm Determining Stability of Genome Editing Components to Streamline Regulatory Strategies

  • Alex Chialastri Analytical Development Genetic Characterization & Molecular Assay Scientist, Century Therapeutics


  • Identifying critical components – the role of the ribonucleoprotein complex
  • In-silico estimation of intra- and extra-cellular dilution of residuals
  • In-vitro and ex-vivo stability of the ribonuclease complex and individual CRISPR components

2:40 pm Roundtable Discussion: Outlining High-Quality Efficacy & Potency Data to Boost Regulatory Approval


  • Optimizing collection of efficacy data in a multidose process to create a strong data package for regulatory bodies in a manageable time frame
  • Highlighting the importance of early consideration of potency assays and reference materials to accelerate phase II approval and increase confidence in clinical processes
  • Considering characteristics that are fundamental in animal models to boost agency approval

3:15 pm Afternoon Break & Networking

Tackling In Vivo Survivability to Improve Clinical Tolerane

3:45 pm Exploring Delivery & Formulation Challenges of iPSC-Derived Cell Therapies to Improve In Vivo Survivability & Clinical Success

  • Louise Winkel Director, Global Search & Evaluation, Novo Nordisk


  • Benchmarking indication specific challenges of cell therapy delivery to improve efficacy
  • Enhancing engraftment and immune-protection to improve in vivo survivability
  • Understanding dose control considerations and optimizing routes of administration to improve clinical trial outcomes

Strengthening Preclinical Models to Improve Translation of Data to the Clinic

4:15 pm Roundtable Discussion: Re-Evaluating Necessary Genetic Knock-Outs to Reduce Immune Burdens


  • Reviewing current evidence determining the necessity of immune knock-outs to elevate regulatory approval
  • Comparing persistency of cells in immune knock-out models to non-knock out models to assess essential edits
  • Outlining data requirements to justify knock-out selection to unite the iPSC space

Learning the Landscape: Investors’ Perspectives on iPSC Therapies to Inform Successful Funding Proposals

3:45 pm Early Stage Venture Investing in iPSC Technology

  • Meg Wood Managing Director, NYBC Ventures


  • Why do we think this space is so important to fund?
  • What type of advancements in technology do we, as early-stage investors, want to see?
  • How can you as a company/syndicate investor work with NYBC Ventures to build/fund gamechanging companies?

Discussing Data Requirements for Regulatory Approval of iPSC-Derived Cell Therapies

4:15 pm Panel: Bridging the Gap Between Investors and Investigators to Establish a Mutual Understanding and Facilitate Meaningful Relationships


  • Developing mutual agreements and understandings of the future of iPSCs to inform smart investments
  • Analyzing costs of clinical plans to provide insight into how to lower costs and reduce investment burdens
  • Mapping out plans to produce iPSC therapies faster and cheaper to increase investment excitement

5:00 pm Close of Conference Day One