Registration
Opening Declaration
Yoji Sato
  • Yoji Sato (Head, Division of Cell-Based Therapeutic Products at National Institute of Health Sciences (NIHS))

    Yoji Sato

    Head, Division of Cell-Based Therapeutic Products at National Institute of Health Sciences (NIHS)

    Dr. Sato is the Head of the Division of Cell-Based Therapeutic Products at the National Institute of Health Sciences. As a graduate student at the University of Tokyo and as a postdoc at the University of Cincinnati, he conducted research on cardiovascular pharmacology and succeeded in establishing a variety of transgenic mouse models to elucidate mechanisms of cardiac excitation-contraction coupling. His current research area is in the field of regulatory science for the quality, efficacy, and safety of cell therapy products. He also serves as a topic leader of ICH Q5A(R2) and a member of the Pharmaceutical Affairs Council of the Ministry of Health Labour and Welfare.

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Opening Remarks
Glenn Wright
  • Glenn Wright (President & CEO of Parenteral Drug Association)

    Glenn Wright

    President & CEO of Parenteral Drug Association

    Glenn E. Wright is the President & CEO of the Parenteral Drug Association and serves as Chairman of the Board for the Product Quality Research Institute. Mr. Wright has more than 30 years industry experience, having served in various technical and senior leadership positions at Eli Lilly, Amgen, and Pfizer. He has extensive technical, regulatory, and quality expertise in pharmaceutical manufacturing and has served on the PDA Board of Directors, Science Advisory Board, and Program Advisory Board. In addition, he has chaired numerous industry meetings, Task Forces, and Steering Committees on topics of importance for the industry.

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Keynote Lecture 1
Keynote Lecture 2: Current Status, Issues, and Direction of Regenerative Medicine Products in Japan

Tomonori Nakayama
Division Head of Medical Device Evaluation Division
MHLW

Regarding regenerative medicine in Japan, the revision of the Pharmaceuticals and Medical Devices Law in 2013 established a system that allows conditional and time-limited approval to be applied to a new category of “regenerative medicine products,” which is expected to be put into practical use at an early stage. As a result, 17 products (as of the end of March 2023), which are included in the regenerative medicine product category, have been approved in Japan, including 4 products that have received conditional and time-limited approval. In the future, it is expected that innovative regenerative medicine products will be put into practical use.

Lunch Break
Session 1: Sterility, Part I
Richard DenkYasuhito Ikematsu

Welcome Remarks by the Moderators

  • Richard Denk (Senior Consultant Aseptic Processing & Containment at Skan AG)

    Richard Denk

    Senior Consultant Aseptic Processing & Containment at Skan AG
  • Yasuhito Ikematsu (Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University)

    Yasuhito Ikematsu

    Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University
Some Efforts by PDA-Japan Chapter for the Practical Use, Dissemination, and Development of Rapid Microbiological Methods (RMM)
Mitsuo Mori

The Aseptic Product GMP Committee of PDA-Japan Chapter has been leading Japan in research and publication on Rapid Microbiological Methods (RMM) continuously and vigorously for about 13 years since 2010 to date. With the release of PIC/S GMP Annex 1 revision 1 in September 2022, this technology, which contributes to improving the quality of pharmaceutical products, is expected to attract even more attention and to expand its practical use globally. In light of the above, some of the research results of the committee, including case studies for the introduction of RMM, will be explained.

  • Mitsuo Mori (Quality Control at Kyowa Kirin Co.,Ltd.)

    Mitsuo Mori

    Quality Control at Kyowa Kirin Co.,Ltd.
Coffee Break
Session 1: Sterility, Part II
Richard DenkYasuhito Ikematsu

Welcome Remarks by the Moderators

  • Richard Denk (Senior Consultant Aseptic Processing & Containment at Skan AG)

    Richard Denk

    Senior Consultant Aseptic Processing & Containment at Skan AG
  • Yasuhito Ikematsu (Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University)

    Yasuhito Ikematsu

    Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University
Contamination Control Strategy and Rapid Microbiological Methods in PIC/S GMP Annex 1 and Annex 2A
Yasuhito Ikematsu

The PIC/S GMP Annex 1 Revision 1 has been released in September 2022, and pharmaceutical and regenerative medicine manufacturing facilities will have been required to develop and implement a Contamination Control Strategy (CCS). Rapid Microbiological Methods (RMM) are effective as a powerful tool for CCS implementation, and a reliable assurance of microbiological contamination control must be established by setting up a rational and effective environmental monitoring program and evaluating its effectiveness. Here, we present the concept of CCS in PIC/S GMP Annex 1 and 2A and the importance of RMM, and introduce several efforts to revise the chapter of RMM in the Japanese Pharmacopoeia.

  • Yasuhito Ikematsu (Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University)

    Yasuhito Ikematsu

    Specially Appointed Associate Professor, Graduate School of Engineering at Osaka University
Strategies and Practical Examples to Ensure Sterility of Cell-Based Health Care Products
Tetsuya Karino

Tetsuya Karino
Sumitomo Pharma Co.,Ltd.

It is extremely difficult to establish sterility assurance by terminal sterilization for biological medical products such as cell-based health care products due to their product characteristics. Therefore, these products must be manufactured using aseptic processing, and an aseptic processing environment must be established and maintained to guarantee sterility in each process.
In particular, aseptic processing environments that require intervention by operators, such as biosafety cabinets, ensure sterility by identifying and controlling the risk of contamination posed by the activities of operators.

This study presents an example of a microbiological contamination risk assessment and associated strategic measures and controls under the condition of a specific cell processing process using a biosafety cabinet.

  • Tetsuya Karino (Production Manager at Sumitomo Pharma Co.,Ltd.)

    Tetsuya Karino

    Production Manager at Sumitomo Pharma Co.,Ltd.
Panel Discussion and Q&A
Networking Reception
Registration
Keynote Lecture 3: PDA Points to Consider for the Engineering and Manufacturing of Cell & Gene “ATMPs”
Richard Denk

The preparation for the commercial manufacturing for Cell & Gene should already start at the development stage of a new novel therapy. This keynote speech will focus on the Points to Consider for the Engineering of the commercial manufacturing of Cell & Gene. Considering equipment and room layout, GMP requirements, disposable technologies, modern technologies like robotics, cleaning and how to work with higher Biosafety Level 2 requirements.

  • Richard Denk (Senior Consultant Aseptic Processing & Containment at Skan AG)

    Richard Denk

    Senior Consultant Aseptic Processing & Containment at Skan AG
Session 2: Process
Masahiro Kino-oka
  • Masahiro Kino-oka (Professor in Department of Biotechnology at Osaka University)

    Masahiro Kino-oka

    Professor in Department of Biotechnology at Osaka University

    Dr. Masahiro Kino-oka is a professor in the Department of Biotechnology, Graduate School of Engineering, Osaka University since 2009, being the leader in the field of manufacturing for cellular therapy products. He has also established the Research Base for Cell Manufacturability since 2021, performing the developments of cell manufacturing system, guidelines for regulation (by PMDA) with standard (by ISO TC198/WG9 and TC276/WG4) as well as human resources, inviting many companies to gather and make up the core consortium for social implementation.

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The Role of Virus Filtration in Achieving Virus Safety of Cell and Gene Therapy Products
Tomoko Hongo-Hirasaki

In recent years, the development of cell and gene therapy products has accelerated, and several products are already in use in the medical field. While viral safety is an important quality item in the safety of these products, there are cases in which clearance processes are not effective in the manufacture of some products and need to be considered. ICH Q5A, the viral safety guideline for biopharmaceuticals, has been revised to reflect technological advances, and the draft revision was released in October 2022, with gene therapy products newly included in the Scope. In this presentation, the role of virus filtration in ensuring viral safety of cell and gene therapy products will be discussed with reference to the three basic principles of ICH Q5A, including the following study cases: (1) Application of virus filtration for contaminated virus clearance in the AAV manufacturing process for gene therapy products, and (2) Application of filtration treatment to raw materials used for product manufacturing further upstream as a risk mitigation strategy.

  • Tomoko Hongo-Hirasaki (Lead Expert on Virus Filtration at Asahi Kasei Medical Co., Ltd.)

    Tomoko Hongo-Hirasaki

    Lead Expert on Virus Filtration at Asahi Kasei Medical Co., Ltd.
Production and Quality Control of Clinical-grade iPSCs and Differentiated Cells Using an Automated Closed Culture System.
Masayoshi Tukahara

Human iPSCs have great potential as source cells for cell therapy. We are investigating the production and quality control of human iPSCs for clinical use using an automated closed culture system in order to establish and culture human iPSCs stably at low cost. We believe that the use of a closed culture system eliminates the need for aseptic manipulation in a safety cabinet and reduces facility management costs. At the same time, we expect to stabilize production and reduce quality control costs by conducting in-process quality control.Until now, iPSCs have generally been established under 2D conditions, but we have succeeded in establishing iPSCs from whole blood under 3D conditions. This enables the establishment of iPSCs, expansion culture, and differentiation under 3D conditions in a closed culture system, and is expected to bring us closer to the realization of low-cost cellular medicine. In this presentation, I will discuss the characteristics of 3D-iPSCs and the 3D culture process, as well as the challenges and expectations for the closed culture system.

  • Masayoshi Tukahara (Center for iPS Cell Research and Application at Kyoto University)

    Masayoshi Tukahara

    Center for iPS Cell Research and Application at Kyoto University
Introduction and Process & Quality design for Alofisel Manufacturing Facility in Japan
Seitaro Mizukami

Alofisel® Injection is a suspension of expanded allogeneic (donor-derived), adipose-derived mesenchymal stem cells (eASC) , which was approved in Japan in Sep 2021 as the first cellular & Gene Therapy product in Takeda Japan. It has been supplied to patients in Japan since 2022. Alofisel Injection is expected to contribute to the improvement of QOL of patients as a therapeutic product for perianal fistulas in Crohn's disease. I would like to introduce the facilities introduction and tech transfer and validation for this product into Japan, and process & quality design for newly introduced isolator.

  • Seitaro Mizukami (Takeda Pharmaceuticals Company Limited)

    Seitaro Mizukami

    Takeda Pharmaceuticals Company Limited
Lessons Learned from Clinic to Commercialization for Cell and Gene Therapy Product
Sang Yoon

Building a C&GT startup company for the first approved Allogeneic Cell Therapy product and separately a CDMO business comes with lessons learned that can benefit others on a similar journey. The lessons learned can help other C&GT companies accelerate their success to scale up and to commercialize their C&GT product. Also, he will provide unique and fresh insights from a leadership and culture perspective, to improve employee engagement and retention in a highly competitive talent market for C&GT professionals.

  • Sang Yoon

    Sang Yoon

Lunch Break
Session 3: Quality, Part I
Shingou Sakurai
  • Shingou Sakurai (Professor of Facility, Pharmaceutical Sciences at Tokyo University of Science)

    Shingou Sakurai

    Professor of Facility, Pharmaceutical Sciences at Tokyo University of Science

    Dr. Sakurai is the professor of facility of Pharmaceutical Sciences in Tokyo University of Science. After retiring from PMDA as an operating officer of inspection departments of GMP, QMS and GCTP in 2020, he has opened the laboratory of Pharmaceutical Quality Design and GMP in the Univ. His research is the international harmonization of Japanese guidelines in these areas. For the human resource development of students and industries, he conducts develop research on the educational materials of GMP and GCTP. He is also a director of PDA Japan chapter and chief director of NPO Drug and Food Quality Assurance Support Center.

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What Does the ICHQ5A Revision Mean for your Cell & Gene Therapy Product?
Dr. Alison Armstrong

Any cell & gene therapy (CGT) manufacturing strategy requires extensive in-process testing to assess quality safety and efficacy of the intended final product. Many national regulatory agencies (i.e. FDA, EMA, NMPA, etc.) provide guidance on what type and degree of testing is expected, however, recommendations may not align between different national agencies. To address this, The International Council of Harmonisation (ICH) is a body of pharmaceutical regulatory experts tasked with harmonizing these quality, safety, and efficacy guidelines to benefit the global development of new pharmaceuticals while still maintaining safeguards to protect public health. The ICH’s quality chapter on viral safety testing for biological products (ICHQ5A) has recently been revised and now brings into scope most CGT products currently in production. While the revision is still in draft the final revision is expected in 2024 and has significant implications to the quality control testing accepted and expected for most CGT products. This presentation provides a description of (1) the CGT products now within scope of the ICHQ5A guidance, (2) where alternative technologies can replace traditional testing strategies, (3) the additional viral clearance studies some vector products are expected to complete, and (4) case studies on how these changes will influence the viral safety testing strategies for future CGT products.

  • Dr. Alison Armstrong (Senior Director and Global Head of the Technical and Scientific Solutions at Merck)

    Dr. Alison Armstrong

    Senior Director and Global Head of the Technical and Scientific Solutions at Merck
Topic (TBC)

Akiyoshi Kunieda
Office of Cellular and Tissue-based Products
PMDA

Coffee Break
Session 3: Quality, Part II
Shingou Sakurai
  • Shingou Sakurai (Professor of Facility, Pharmaceutical Sciences at Tokyo University of Science)

    Shingou Sakurai

    Professor of Facility, Pharmaceutical Sciences at Tokyo University of Science

    Dr. Sakurai is the professor of facility of Pharmaceutical Sciences in Tokyo University of Science. After retiring from PMDA as an operating officer of inspection departments of GMP, QMS and GCTP in 2020, he has opened the laboratory of Pharmaceutical Quality Design and GMP in the Univ. His research is the international harmonization of Japanese guidelines in these areas. For the human resource development of students and industries, he conducts develop research on the educational materials of GMP and GCTP. He is also a director of PDA Japan chapter and chief director of NPO Drug and Food Quality Assurance Support Center.

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Topic (TBC)

Kazuji Omori
Office 0f Manufacturing Quality for Drugs
PMDA

Topic (TBC)
Yoji Sato
  • Yoji Sato (Head, Division of Cell-Based Therapeutic Products at National Institute of Health Sciences (NIHS))

    Yoji Sato

    Head, Division of Cell-Based Therapeutic Products at National Institute of Health Sciences (NIHS)

    Dr. Sato is the Head of the Division of Cell-Based Therapeutic Products at the National Institute of Health Sciences. As a graduate student at the University of Tokyo and as a postdoc at the University of Cincinnati, he conducted research on cardiovascular pharmacology and succeeded in establishing a variety of transgenic mouse models to elucidate mechanisms of cardiac excitation-contraction coupling. His current research area is in the field of regulatory science for the quality, efficacy, and safety of cell therapy products. He also serves as a topic leader of ICH Q5A(R2) and a member of the Pharmaceutical Affairs Council of the Ministry of Health Labour and Welfare.

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Panel Discussion and Q&A
Invited Lecture
Masahiro Kino-oka

Regenerative medicine and cell therapy use the cell transplants which are manufactured, requiring interdisciplinary activities not only from medicinal and biological fields but also from manufacturing field. In cell production for regenerative medicine and cell therapy, process development is essential to improve the efficiency and stability of cell product quality. Unlike conventional pharmaceutical manufacturing,
The quality of cell products tends to be unstable due to the inherent characteristics of cells, which is an obstacle to mechanization and scale-up of manufacturing. Therefore, it is essential to develop a unique concept.
We proposed the discipline for cell production, which is ‘cell manufacturability’ to be defined as capability of cell manufacturing through the processes by bridging between biological and process aspects. And ‘Design for cell manufacturability’ is to be manufacturing design of cell-based products in such a way that they are easy to manufacture through simple, safe and efficient (cost-saving) process with stable product quality and secure to customer by considering transportation and preparation outside factory, acting practically the system optimization for the efficiency and stability of the process by understanding the requirement gap between biological and engineering aspects and reducing output fluctuation. This action will lead to stable process and cost saving of the products through the process simplification and governing the kinetics of cell behavior.

  • Masahiro Kino-oka (Professor in Department of Biotechnology at Osaka University)

    Masahiro Kino-oka

    Professor in Department of Biotechnology at Osaka University
Summary and Closing Remarks
Richard Denk
  • Richard Denk (Senior Consultant Aseptic Processing & Containment at Skan AG)

    Richard Denk

    Senior Consultant Aseptic Processing & Containment at Skan AG

    Richard Denk is working at SKAN AG, headquartered in Allschwil Switzerland as a Senior Consultant Aseptic Processing & Containment. Richard is Member of the PDA ATMP Advisory Board and chairs the PtC of the Manufacturing of ATMPs. Richard also chaired the 2022 PDA ATMP Conference in Brussels and is a Member of the Program Committee of the PDA 2022 Annex 1 workshops. Richard is a member of the PDA Isolator Expert Group and publisher of the PDA Paper “Isolator Surfaces and Contamination Risk to Personnel and Patient”. Furthermore, Richard is member of the ISPE Annex 1 and PIC/s Annex 2A commenting group. Richard is a global recognized subject matter expert on Containment and has developed the containment pyramid.

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