Cambridge Healthtech Instituteの第5回年次会議

Cell Therapy CMC and Analytics
(細胞治療薬のCMCとアナリティクス)

新しい技術の導入とアナリティクス戦略の強化

2023年3月15 - 16日、CET(中部欧州標準時)

Cambridge Healthtech Instituteの「細胞治療薬のCMCとアナリティクス」会議では、バイオ医薬品業界、学界、政府から有数の科学者が集まり、アナリティクスの特性評価、遺伝子編集、CMC・規制戦略における最近の進歩について議論します。バルセロナに参加すると、製品開発プロセスの最適化や、市場投入までの時間の短縮方法に関する洞察を得ることができます。

3月15日(水)

Registration Open10:30

PLENARY SESSION: EMERGING MODALITIES, PLATFORMS, AND TECHNOLOGIES - FROM mRNA TO PROTEINS
基調講演:新興のモダリティ、プラットフォーム、技術 - mRNAからタンパク質へ

11:15

Chairperson's Opening Remarks

Margit Holzer, PhD, Owner, Ulysse Consult

11:20 PLENARY PRESENTATION:

Overcoming CMC and Supply Chain Challenges for mRNA Technologies

Gregory Troiano, Chief Manufacturing Officer, mRNA Center of Excellence, sanofi

Thanks to the rapid development of mRNA vaccines for COVID-19, the industry now has the momentum and resources to overcome many of the early CMC challenges and realize its enormous potential. This presentation will discuss the strategies in place to overcome CMC and supply chain challenges for mRNA technologies already and future innovations primed to take it to the next level.

11:50 PLENARY PRESENTATION:

Affinity Proteins for Biotechnological and Medical Purposes

Sophia Hober, PhD, Professor, School of Biotechnology, KTH Royal Institute of Technology

Affinity proteins are crucial for life, for building structures, performing reactions, and for signaling purposes. In life sciences and medicine, affinity proteins are used to generate knowledge, but also for diagnostic and therapeutic purposes. This talk will cover how antibodies and small affinity molecules can be used to map the human proteome, develop diagnostic tools for in vivo visualization as well as efficiently purify therapeutics based on antibodies.

Transition to Sessions12:20

Sponsored Presentation (Sponsor Opportunity Available)12:30

Networking Lunch (Sponsor Opportunity Available)13:00

CMC & EMERGING ANALYTICAL METHODS
CMCと新興のアナリティクス手法

14:00

Chairperson's Remarks

Christiane Niederlaender, PhD, Vice President, Technical CMC, Parexel

14:05

CMC Challenges and Opportunities for Cell and Gene Therapies

Paulo Fernandes, PhD, Associate Director Cell & Gene Therapy Technologies, Technology Operations & Global Tech Development, Orchard Therapeutics

Developing next generation products to release current bottlenecks in CGT. Technical overview on CMC strategies for real CGT products, presentation of actual scientific data showing the progress done to release current bottlenecks in the manufacture and COGs of CGT products. The critical points in the CMC strategy for CGT products, with real examples. Current bottlenecks in the manufacture of CGT products and development areas to focus on.

14:35

A Consortium Investigation for Extensive Analytical Characterisation of T Cell Immunotherapy Processes

Rhys Macown, PhD, Lead Scientist, Cell & Gene Therapy Catapult

There are longstanding challenges in the commercialisation of ATMPs that can be addressed by the implementation of process analytical technologies (PATs). Cell and Gene Therapy Catapult (CGTC) has led a consortium project with 23 collaborators to investigate the application of PAT and advanced analytics for a T cell immunotherapy process. We will discuss the extensive approach we applied to analytical characterisation of the process and the opportunities and challenges it presented.

15:05

Cell Sorting and Analytical Development Considerations for GMP Manufacture of Cell Therapies

Harish Adoni, Scientist, Analytics Cell Therapy, ElevateBio

Over the past decade, the development of cell therapies has gained significant momentum. Processes executed in a hospital setting such as the isolation of tumor infiltrating lymphocytes (TILs), transformed clones of hematopoietic stem cells (HSCs), or other rare cells, are challenging to replicate in a cGMP environment. We will discuss the cell sorting process and analytical development considerations when implementing fluorescence activated cell sorting (FACS) in a cGMP environment.

Sponsored Presentation (Opportunity Available)15:35

Refreshment Break in the Exhibit Hall with Poster Viewing16:05

16:40

Establishment of Process Intensification and Automated Process Control Strategies for CAR-T Production

Qasim A. Rafiq, PhD, Associate Professor, Biochemical Engineering, University College London

Manufacturing clinically-relevant cell numbers with desired phenotypes is a major hurdle for ex vivo genetically modified cellular immunotherapies, such as CAR-T. The high cost of goods and inherent variability of gene-modified cell therapies presents a translational and manufacturing challenge. This talk will focus on providing process optimisation and process intensification case studies for CAR-T cell manufacture and how applying these strategies may prove essential for commercial viability. Specifically, this talk will focus on: Identifying critical process parameters for improved, consistent CAR-T expansion, scaling up critical parameters to the stirred-tank bioreactor process and optimizing perfusion parameters to maximize growth and function?. 

17:10 PANEL DISCUSSION:

CMC & Emerging Analytical Methods

PANEL MODERATOR:

Christiane Niederlaender, PhD, Vice President, Technical CMC, Parexel

PANELISTS:

Rhys Macown, PhD, Lead Scientist, Cell & Gene Therapy Catapult

Harish Adoni, Scientist, Analytics Cell Therapy, ElevateBio

Qasim A. Rafiq, PhD, Associate Professor, Biochemical Engineering, University College London

Interactive Discussions17:40

Interactive Discussions are informal, moderated discussions, allowing participants to exchange ideas and experiences and develop future collaborations around a focused topic. Each discussion will be led by a facilitator who keeps the discussion on track and the group engaged. For in-person events, the facilitator will lead from the front of the room while attendees remain seated. To get the most out of this format, please come prepared to share examples from your work, be a part of a collective, problem-solving session, and participate in active idea sharing. Please visit the Interactive Discussion page on the conference website for a complete listing of topics and descriptions.

Close of Day18:15

3月16日(木)

Registration and Morning Coffee08:00

OPTIMISING COMPARABILITY STRATEGIES
比較可能性戦略の最適化

08:25

Chairperson's Remarks

Christopher Bravery, PhD, Consulting Regulatory Scientist, Advanced Biologicals Ltd.

08:30 KEYNOTE PRESENTATION:

Implementing Risk-Based Approaches for Cell Therapies

Florence Salmon, PhD, Vice President, Regulatory Affairs and Preclinical Development, Ridgeline Discovery

Genetically modified cells are remarkably complex medicinal products that require smart, risk-based manufacturing and control strategies to meet regulatory expectations. Nonetheless, changes are inevitable at each stage of development of cell therapies, sometimes late or even post-approval. Comparability is therefore a key exercise and should be accounted for from the very beginning. Having the right analytical and regulatory tools at hand at the right time is the key to success.

09:00

Life-Cycle Comparability Approaches for Genetically Modified Cells

Christiane Niederlaender, PhD, Vice President, Technical CMC, Parexel

Comparability of biological products is always challenging, but even more so when the product is a complex mixture of cells of which a proportion have been genetically modified. The frequently short development timelines for cell and gene therapies mean that comparability is often considered too late during the implementation of process changes. The talk outlines key considerations that ensure that an appropriate comparability plan can be put in place and executed, and that can be adapted throughout the product lifecycle.

REGULATORY CONSIDERATIONS
規制上の考慮事項

09:30

Regulatory Strategies for Cell Therapy

Raj Puri, MD, PhD, Executive Vice President, Regulatory Strategy and Translational Medicine, Iovance Biotherapeutics

Tumor-infiltrating lymphocytes (TIL) are circulating cytotoxic and helper T-cells that have infiltrated a tumor, recognize tumor-specific neoantigens unique to each patient, and mediate tumor cell death. TIL cell therapy involves the adoptive transfer of billions of polyclonal, patient-specific TIL recovered from a patient’s tumor tissue that are amplified and reinvigorated using a proprietary process. I will discuss unique opportunities and challenges in TIL cell therapy manufacturing, logistics and development.

10:00

Ensuring Quality of Cell Therapy Products

Maura Kibbey, PhD, Director, Biologics Marketing, USP

Assessment of cell therapy quality from raw materials to final product is critical to ensuring their safety and efficacy. Lack of standards for cell therapy has slowed down product development since the path and tools to commercialization are not clear. USP continues to publish best practices and methods that support their innovation. Existing standards as well as new work related to plasmid DNA and rapid microbial methods will be shared.

Coffee Break in the Exhibit Hall with Poster Viewing10:30

11:00 FEATURED PRESENTATION:

Common Issues Identified with Cell Therapy Applications

Janet Glassford, PhD, Expert Quality Assessor, MHRA

This presentation will outline common issues relating to cell therapy applications from the perspective of a quality assessor with over a decade of experience as an MHRA assessor and as a former EMA expert. Updated information regarding UK specific regulatory routes and scientific guidance will also be provided.

11:30

Achieving Global Regulatory Harmonization and Centralized QA in a Decentralized Model

Chaya Mazouz, Vice President, Quality (IL) & Global Regulatory Affairs, Orgenesis, Inc.

Decentralized manufacturing (DCM) strategy consists of a platform comprising manufacturing and testing units, installed to the same specifications at multiple locations placed close to or at medical centers, networked and managed by a central remote Quality Management System. A regulatory framework is required to enable the development of point of care (POCare) manufacture, providing control measures equivalent to those currently in place, confirming that POCare products have appropriate quality, safety, and efficacy attributes. To ensure compliance to quality and regulatory DCM requirements, Orgenesis has established a comprehensive approach supporting oversight of POCare centers.

12:00

Sterility Control for Cell Therapies from a Regulatory Perspective

Christopher Bravery, PhD, Consulting Regulatory Scientist, Advanced Biologicals Ltd.

The nature of cell therapy products means they cannot be terminally sterilised or even sterile-filtered; this puts a greater onus on other aspects of sterility control. This talk will discuss how a holistic approach is needed to ensure safety of these products, including facilities, testing and methods of sterilisation.

Sponsored Presentation (Opportunity Available)12:30

Networking Lunch (Sponsor Opportunity Available)13:00

NOVEL PLATFORMS, RISK ANALYSIS, AND MACHINE LEARNING
新規プラットフォーム、リスク分析、機械学習

13:45

Chairperson's Remarks

Ricardo Baptista, PhD, CTO, Alder Therapeutics

13:50

Development of a Novel Costimulatory Platform to Improve Adoptive Cell Therapies

John Bridgeman, PhD, Director, Cell Therapy Research, Instil Bio

Utilizing a novel platform to overcome poor endogenous costimulation and leverage the diverse TCR repertoire of TILs while amplifying antitumor activity.

14:20

Raw Materials and Reagents Supply to Develop Scalable Manufacturing Processes - Considerations for Pluripotent Stem Cell Therapies

Ricardo Baptista, PhD, CTO, Alder Therapeutics

Evaluation of critical raw materials should start at early stages of product cycle. This talk will focus on risk analysis and mitigation strategies to support process and analytical development for the manufacture of allogeneic therapies from pluripotent stem cells.

14:50

Replacing Manual Gating of Flow Cytometry Data in Cell Therapy Manufacturing

Ryan Brinkman, PhD, Head, Flow Cytometry R&D, Dotmatics; Distinguished Scientist, BC Cancer; Professor Emeritus, Medical Genetics, University of British Columbia

Flow cytometry data analysis is extremely time-consuming and the principal source of variation in the application of the technology. As an alternative, we have developed the best performing automated approach for cell population identification in the peer-reviewed literature. It consistently achieves up to 94% accuracy, and is in use by pharma for clinical studies and cell therapy manufacturing. Looking to the future our preliminary data supports that machine learning will exceed even this, providing a scalable solution for all users of the technology.

Close of Summit15:20

* 不測の事態により、事前の予告なしにプログラムが変更される場合があります。

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Prelim Agenda Now Available

3月14 - 15日

CELL CULTURE AND BIOPRODUCTION

ADVANCES IN RECOVERY & PURIFICATION

GENE THERAPY CMC AND ANALYTICS

CELL THERAPY MANUFACTURING

ANALYTICS AND CHARACTERIZATION

3月14 - 15日

CELL LINE DEVELOPMENT

INTENSIFIED & CONTINUOUS PROCESSING

GENE THERAPY MANUFACTURING

CELL THERAPY CMC AND ANALYTICS

FORMULATION AND STABILITY