iPSC SCIENTIFIC SIGNATURE SERIES: PROGRAM SCHEDULE
Saturday, May 10, 2025
08:00–08:15
Opening Remarks
This session will introduce the key challenges and objectives in ensuring the safety and Quality of iPSC-derived therapies. Co-Chairs will provide an overview of the structure of the event and the objective of addressing the need for standardized and adaptable safety characterization frameworks.
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
08:15–08:45
Session I: 2018 Guidelines on Safety Characterization: How Have These Guidelines Held Up?
This session will assess the impact of the 2018 Global Alliance for iPSC Therapies (GAiT) guidelines, examining how well they have supported the field over the past seven years. The session will also explore emerging challenges and evolving regulatory expectations, setting the stage for discussions on how the guidelines should adapt to support the next phase of iPSC therapeutic development.
CHAIR & SPEAKER
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Presentation Sponsored by GAiT
Key Learning Objectives:
- How have the 2018 GAiT guidelines influenced the development and standardization of iPSC-based therapies?
- What gaps or challenges have emerged in applying these guidelines, and how have they been addressed in practice?
- What changes can we anticipate from the next phase of iPSC clinical translation?
08:45–12:00
Session II: Technologies and Approaches to iPSC Safety Characterization
As iPSC-derived therapies progress toward clinical applications, selecting the most effective methods for to demonstrate safety and quality are key challenges. This session will explore the strengths and limitations of eight core technologies that are currently used to assess genomic stability, tumorigenicity, pluripotency, residual undifferentiated cells and biological potential. These include karyotyping, tumorigenicity testing, whole genome sequencing (WGS), pluripotency assays, flow cytometry, targeted mutational analysis, assay for residual pluripotent cells, and imaging techniques. The discussion will examine current and emerging approaches, highlighting key considerations such as sensitivity, reproducibility, and clinical relevance. Discussion will explore opportunities for consensus regarding best practices for ensuring the safety and reproducible quality of iPSC-derived products.
CO-CHAIR
Wanxing Cui, MD, PhD
Georgetown University Hospital
United States
CO-CHAIR & SPEAKER
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
CO-CHAIR
George Muschler, MD
Cleveland Clinic
United States
SPEAKER
Tenneille Ludwig, PhD
WiCell
United States
SPEAKER
Lise Munsie, PhD
BlueRock Therapeutics
United States
SPEAKER
Natacha Agabalyan, PhD
CGT Catapult
United Kingdom
SPEAKER
Terri Gaskell, PhD
Rinri Therapeutics
United Kingdom
SPEAKER
Anthony Asmar, PhD
National Institute of Standards & Technology
United States
SPEAKER
Deborah Hursh, PhD
Hursh Cell Therapy Consulting
United States
Session includes a corporate presentation by Ajinomoto
Key Learning Objectives:
- What are the key advantages and limitations of each technology?
- Are there alternative approaches that are better or cheaper?
- Can these technologies be improved (accuracy, efficiency, cost)?
- What new technologies will replace or augment current methods?
- Where will AI models integrate these tools for stage specific QA?
- At what stages are models most needed for QA?
12:00–13:00
Lunch
Sponsored by:
13:00–16:00
Session III: Building Frameworks Using iPSC Products
Building on previous discussions, this session will apply key safety characterization methodologies to real iPSC-derived products across different therapeutic applications. Experts will evaluate how existing technologies can be integrated into safety assessment frameworks, considering critical factors such as autologous vs. allogeneic approaches, gene-edited vs. non-edited products, and varying disease indications. Through case studies, this session will develop practical strategies for selecting and optimizing safety characterization methods tailored to specific product types and regulatory requirements.
CO-CHAIR
Hiroto Bando, PhD, MBA
Minaris Advanced Therapies
Japan
CO-CHAIR
Vladislav Krupalnik, PhD
RenewalBio
Israel
CO-CHAIR & SPEAKER
Xiaokui Zhang, PhD
Aspen Neuroscience
United States
SPEAKER
Masayo Takahashi, MD, PhD
Vision Care
Japan
SPEAKER
Bruna Paulsen, PhD
Gameto
United States
SPEAKER
Arina Perez, PhD
Century Therapeutics
United States
SPEAKER
Takehiko Kaneko, MD
Heartseed
Japan
Key Learning Objectives:
- How can safety characterization technologies be applied effectively across different iPSC-derived products and therapeutic applications?
- What specific considerations should be made for autologous vs. allogeneic iPSC therapies and gene-edited vs. non-edited products?
- How can a standardized yet adaptable framework be developed to guide safety assessment while meeting regulatory expectations?
16:00–16:30
Closing Remarks
This final session will consolidate insights from previous discussions to establish clear frameworks for selecting and applying safety characterization technologies in iPSC-based therapies. Experts will outline which methodologies are best suited for different product types, balancing sensitivity, cost-effectiveness, and regulatory expectations. The session will provide actionable guidance on integrating these approaches into clinical development, ensuring robust and scalable safety assessment strategies for the future of iPSC therapies.
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Key Learning Objectives:
- What have these discussions concluded?
- What next steps need to be taken to advance the field?
- Where is there consensus, where is there still debate?