iPSC SCIENTIFIC SIGNATURE SERIES: PROGRAM SCHEDULE
Saturday, June 1, 2024
08:00–08:15
Co-Chairs Welcome
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
08:15–09:15
Manufacturing Considerations for Autologous and Allogeneic iPSC-derived Therapies & Opportunities for Cross-industry Alignment
This session will survey current manufacturing paradigms for iPSC-derived therapies with an emphasis on the impact of different starting materials, such as autologous or allogeneic cells. The speakers will discuss key steps in the iPSC manufacturing process, such as iPSC banking and differentiation, while highlighting differences and similarities between autologous and allogeneic therapies. Following these presentations, there will be a panel session to explore opportunities for alignment of pre-competitive manufacturing practices across the nascent field of iPSC-derived therapeutic development.
CHAIR
Bruno Marques, PhD
Century Therapeutics
United States
Lise Munsie, PhD
CCRM
Canada
Kim Raineri, MBA
Aspen Neuroscience, Inc.
United States
Priye Iworima, PhD
PBS Biotech, Inc.
Canada
Learning Objectives:
- Highlight key differences and similarities in current applications of iPSC-derived therapies.
- Identify opportunities for cross-industry alignment and collaboration, such as white papers, to streamline manufacturing operations, influence regulators, and ultimately accelerate development of iPSC-derived therapies.
09:15–10:30
CMC: Developing Effective Strategies to Control and Assess Identity & Potency Throughout the iPSC-derived Product Lifecycle
This session will look at practical and regulatory considerations underpinning CMC (Chemistry, Manufacturing and Controls) strategy for the manufacture of iPSC derived products. Manufacturing processes and product specifications must be designed and controlled in order to ensure the generation of safe and consistent products. iPSC-derived products can be particularly challenging due to the nature of the starting material and the potential to treat many patients from a single batch. The speakers will share a range of perspectives on this critical and rapidly evolving area of GMP.
CHAIR
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Jacqueline Barry, PhD
CGT Catapult
United Kingdom
Ricardo Baptista, Eng PhD
SmartCella
Sweden
Nobuaki Shiraki, PhD
Tokyo Institute of Technology
Japan
Learning Objectives:
- Identify major considerations when developing an effective CMC plan for an iPSC-derived product, and where to find guidance and advice.
- Highlight the importance of understanding your process and product in order to a) develop relevant and useful potency and functional assays, and to b) de-risk the product in early development.
10:30–11:30
Characterization of iPSC-derived Cell Therapies – Going in Depth
This session will immerse attendees in the latest advancements shaping the deep characterization of iPSC-derived cell therapies, such as genomic stability testing, to ensure the quality and consistency of the product and manufacturing process. Experts will share their insights into the current efforts and challenges in standardizing characterization protocols, including technologies such as WGS, omics-based testing, and functional assays.
CHAIR
Xiaokui Zhang, PhD
Aspen Neuroscience, Inc.
United States
Jason 'Jay' Mills, PhD
Century Therapeutics
United States
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
Daniel Fremgen, MS
Aspen Neuroscience, Inc.
United States
Learning Objectives:
- Provide a comprehensive understanding of the complexities involved in the characterization process and its critical role in expediting clinical entry for iPSC-derived cell therapies.
- Explore the latest initiatives aimed at driving industry-wide consensus on characterization standards, ultimately paving the way for widespread adoption.
11:30–12:00
Discussion - Morning Sessions
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
12:00–12:30
Lunch
12:30–13:45
Regulatory Requirements and Heterogeneity Across Jurisdictions
In this session, we will engage with regulatory authorities from different jurisdictions to unpack the complex landscape governing iPSC-derived product development. Our aim is to illuminate the regulatory variances and challenges encountered when aligning product development with international standards. The session will provide insights into navigating this multifaceted environment, facilitating a clearer understanding of how to harmonize international regulatory strategies for cell therapy development and highlight the regulatory nuances in starting material choices, CMC strategies, and genomic stability testing.
CHAIR
Wanxing Cui, PhD
Georgetown University
United States
Yusuke Nozaki
PMDA
Participation Supported by the Japanese Society for Regenerative Medicine
Japan
Zhaohui Ye, PhD
FDA
United States
Learning Objectives:
- How do regulatory expectations for starting materials and genomic characterization in iPSC-derived product development differ across FDA, PMDA, and TGA jurisdictions?
- What are the common regulatory hurdles faced during the CMC process for iPSC products, and how can developers navigate these across different regions?
- In the context of global harmonization efforts for iPSC-derived products, how are jurisdictions addressing the heterogeneity in iPSC potency and functional assays, and what collaborative steps could be taken to streamline this process?
13:45–15:15
Ongoing Clinical Trials
Speakers will offer insights into their target disease or indication, the current status of their ongoing clinical trials, and their roadmap for securing regulatory approval. Following these presentations, invited KOLs will share experiences with their clinical trials, including challenges and learnings that are specific to iPSC-based cell therapies.
CHAIR
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
Narihito Nagoshi, PhD
Keio University; Japanese Society for Regenerative Medicine
Japan
Kilian Kelly, PhD
Cynata Therapeutics Limited
Australia
Dan Kemp, PhD
Shinobi Therapeutics, Inc.
United States
Kiyoko Bando, PhD
Sumitomo Pharma Co., Ltd.
Japan
Learning Objectives:
- Explore benchmarks for start-ups with iPSC-based products in terms of exit strategies.
- Determine key considerations for cost of goods of iPSC-based products.
- Identify CMC guidelines for iPSC clones as a starting material.
15:15–16:00
Closing Panel:
Building a Roadmap for iPSCs: How do we Move Forward from Here?
Session Chairs join closing panel to summarize takeaways from each session presentations and discussions and build a roadmap for getting iPSCs to the clinic successfully with an eye to eventual adoption.
Joanne Mountford, PhD
Scottish National Blood Transfusion Service
Scotland
Shin Kawamata, MD, PhD
Cyto-Facto
Japan
16:00–17:30
ISCT Scientific Signature Series Cocktail Reception