7:00 am Registration & Coffee

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

7:50 am Chair’s Opening Remarks

  • Stefan Frank Director, Cell & Gene Therapy Platform Strategy, Bayer

8:00 am iPSC Leaders Panel: Highlighting the Next Steps for the Future of iPSC-Derived Cell Therapies

Synopsis

  • 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

9:00 am Investigating Stem Cell Genetic Integrity Using NGS Testing: Turning a Deep Analysis Into a Straightforward Interpretation

  • Reda Zenagui Director, Research & Development, Stem Genomics

Synopsis

  • 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

9:10 am Roundtable Discussion: To Auto or to Allo? Evaluating the Future of iPSC-Derived Cell Therapies to Inform Our Next Steps

  • Irina Klimanskaya Senior Director, Translational Science & Development, Astellas Institute for Regenerative Medicine
  • Wei Li Chief Scientific Officer, Cytovia Therapeutics

Synopsis

  • Considering the practicalities of a scaled-up autologous 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

10:00 am Improved Anti-Tumor Immune Function of TGFβR2 Knock Out & IL-15 Knock In iPSCDerived NK (iNK) Cells by TALEN® Editing

Synopsis

  • 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

10:30 am Morning Refreshment Break & Speed Networking

DISCOVERY & TRANSLATION TRACK

  • Toby Deuse Scientific Co-Founder, Shinobi Therapeutics

Optimizing Gene Editing Protocols for Improved iPSC Product Safety

  • Toby Deuse Chief Scientific Officer, Shinobi Therapeutics

12:00 pm Overcoming Manual Bottlenecks to iPSC Reprogramming to Improve Efficacy & Efficiency

Synopsis

  • Patient-specific induced pluripotent stem cells (iPSCs) are a valuable resource in the development of models for studying unique disease or drug responses
  • Despite the potential of these cells, reprogramming has low efficiency (<1%), instability of pluripotency, and higher chance for mutations. Reprogramming is long, labor intensive and manual, and requires additional screening to derive a monoclonal population
  • The CellRaft® Array offers a favorable culture environment for fragile iPSCs by providing flask-like culture conditions combined with single-cell segregation, and CellRaft AIR® Technology can accelerate reprogramming by improving efficiency, automation and cell viability

12:15 pm Panel Discussion: Benchmarking Safety Considerations During Gene Editing Protocols to Ease Regulatory Burdens

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

Synopsis

  • 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

REGULATORY & EARLY CLINICAL DEVELOPMENT TRACK

  • Stefan Frank Director, Cell & Gene Therapy Platform Strategy, Bayer

Refining Manufacturing Strategies to Increase Efficiency in CMC Protocols

12:00 pm Suspension Culture of Autologous Human Induced Pluripotent Stem Cells in a Single- Use Vertical-Wheel® Bioreactor System Improves Stem Cell Quality, Quantity & Function

  • Nidheesh Dadheech Senior Research and Process Development Lead, University of Alberta

Synopsis

  • Vertical-wheel® bioreactor system provides most optimal and scalable platform for human iPSC expansion
  • Bioreactor-driven 3D iPSC culture enables prime to naive pluripotency transition
  • Suspension iPSCs form more mature teratomas with defined tri-germ layer tissues

12:30 pm Promises & Challenges of Translating PSC-Derived Natural Killer Cells Manufacturing CMC Protocols

  • ShiYu Wang Director, Research and Development, HebeCell

Synopsis

  • Developing a Platform for a Scalable Solution For Off-The-Shelf NK Cell Therapies
  • Safety Considerations of HebeCell’s PSC-NK
  • NK-Specific Challenges and Solutions for CMC protocol

1:00 pm Lunch Break & Networking

DISCOVERY & TRANSLATION TRACK

Strengthening Preclinical Models to Improve Translation of Data to the Clinic

2:00 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

Synopsis

  • 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

2:30 pm Roundtable Discussion: Combatting Limitations With Preclinical Models to Improve Translation of iPSC-Derived Therapies to the Clinic

  • Toby Deuse Scientific Co-Founder, Shinobi Therapeutics

Synopsis

  • 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

REGULATORY & EARLY CLINICAL DEVELOPMENT TRACK

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

2:00 pm Investors Deep Dive: Bridging the Gap Between Investors & Investigators to Establish a Mutual Understanding & Facilitate Meaningful Relationships

Synopsis

An introductory panel from leading investors in the cell therapy space uncovering the nonnegotiables and current perspectives on investing into iPSC-derived products. Rounding off with a group discussion on how to unify investor expectations with clinical reality

  • 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

3:00 pm Afternoon Break

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

  • Toby Deuse Chief Scientific Officer, Shinobi Therapeutics

3:40 pm Determining Stability of Genome Editing Components to Streamline Regulatory Strategies

Synopsis

  • 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

4:10 pm Roundtable Discussion: Re-Evaluating Necessary Genetic Knock-Outs to Reduce Immune Burdens

  • Toby Deuse Scientific Co-Founder, Shinobi Therapeutics

Synopsis

  • 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

5:00 pm Drinks Reception & Poster Session

6:00 pm Close of Day 1