Cambridge Healthtech Instituteの第9回年次会議
Cell Therapy Manufacturing
(細胞治療薬製造)
細胞治療薬のスケーリングと産業化
2023年8月16 - 17日、EDT(米国東部標準時)
8月16日(水)
Registration and Morning Coffee7:30 am
MANUFACTURING CELL THERAPIES BEYOND CAR Ts
CAR-T細胞を超える細胞治療薬の製造
Manufacturing Gamma Deltas
Kate M. Rochlin, PhD, COO, IN8bio, Inc.
IN8bio is a clinical-stage gamma-delta T cell therapy company with two clinical programs in Phase I and one in Phase II, in both solid and hematological tumors. Gamma-delta T cells are part of the innate immune system with the ability to recognize and kill malignant cells and our clinical manufacturing experience has shown that evaluation of the CQA and cellular effector memory profile may help predict expansion and potency.
The (Re)emerging Field of Xenotransplantation
Knut Niss, PhD, CTO, eGenesis, Inc.
Through our transformative research, we are developing HuCo organs and cells to meet the increasing need. Our eGenesis Genome Engineering and Production (EGEN) platform leverages advances in gene editing technologies to address the historical challenges of xenotransplantation.
Optimization of Autologous TCR T Cell Manufacturing Process by Managing the Heterogeneity of the Starting Apheresis Material
Gagan Bajwa, PhD, Senior Scientist, Process Development, Immatics
This presentation will discuss: Heterogeneity of the starting leukapheresis poses challenges for successful manufacturing of autologous TCR T cells; cellular composition of the starting material impacts product characteristics; and optimization of starting material is critical to achieve adequate quantity and quality of the TCR T cell product.
Sponsored Presentation (Opportunity Available)9:30 am
Coffee Break in the Exhibit Hall with Poster Viewing10:00 am
SPECIAL ISCT SESSION - PAT, PROCESS CONTROL, AND SCALE-UP
特別セッション - PAT、プロセス制御、スケールアップ

Process Analytical Technologies for Process Control Strategy Development of Cell & Gene Therapy Products
Daria Marsh, PhD, Head, Bioprocessing, Cell & Gene Therapy Catapult
Advanced control strategies using PAT technologies have the potential to increase product consistency, reduce process variability and increase potential for process automation during the production of cell and gene therapy products. This presentation will discuss examples of real-time monitoring strategies and how an automated PAT lab set up can be utilized for improved process characterization and future manufacturing automation.
Sponsored Presentation (Opportunity Available)12:10 pm
Refreshment Break in the Exhibit Hall with Poster Viewing12:40 pm
SPECIAL ISCT SESSION - PAT, PROCESS CONTROL, AND SCALE-UP (CONT.)
特別セッション - PAT、プロセス制御、スケールアップ(つづき)
Be More Closed-Minded
Ian D. Gaudet, PhD, ISCT Process & Product Committee Member, and Senior Director and Site Head, Process Sciences, Miltenyi Biotec, Inc.
Autologous cell therapy manufacturing costs still limit bringing these impactful medicines to more patients. Open process manipulations requiring expensive engineering controls are a significant driver of the overall manufacturing costs, including significant expenses in labor, equipment, materials, cleaning, validation, and facility footprint. Progress towards fully-closed system design suitable for commercial scale manufacturing in both centralized and point-of-care settings are discussed.
ISCT SESSION - Process Analytics, Automation, and Digitalization
Dominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & CTO, Cell and Gene Therapy, Discovery Life Sciences
- The role of process analytics to inform decisions
- Emerging technologies and modalities
- Automation and AI in cell therapy manufacturing
Ian D. Gaudet, PhD, ISCT Process & Product Committee Member, and Senior Director and Site Head, Process Sciences, Miltenyi Biotec, Inc.
Sponsored Presentation (Opportunity Available)2:30 pm
Refreshment Break in the Exhibit Hall with Poster Viewing3:00 pm
PLENARY KEYNOTE: LEADING TO TOMORROW'S ADVANCES
基調講演:明日の進歩につながる
Current and Future Trends in Biomanufacturing of New Modalities
Konstantin B. Konstantinov, PhD, CTO, Codiak Biosciences
Using exosomes as an example, this presentation examines the current and future trends in biomanufacturing, and the technologies needed to manufacture emerging modalities at scale. Traditional biomanufacturing methods do not provide the industrialized, commercially scalable, highly efficient and reproducible manufacturing process essential for this new class of biotherapeutics- so we built it from the ground up.
The Digitalization of Biomanufacturing
Richard D. Braatz, PhD, Edwin R. Gilliland Professor, Chemical Engineering, Massachusetts Institute of Technology
A fully instrumented testbed is described for the end-to-end integrated and continuous manufacturing of monoclonal antibodies. The testbed consists of parallel bioreactors, simulated moving bed chromatography systems for capture and polishing, bespoke viral inactivation, and a MAST auto-sampling system. Experimental results are compared with a digital twin for continuous runs lasting 30 to 60 days each, which include variations in metabolites and glycosylation profiles in designed experiments. The increased consistency in the glycosylation profile of the monoclonal antibodies being produced is quantified when going from batch to semi-batch to perfusion mode, and when moving from start-up to quasi-steady conditions.
Networking Reception in the Exhibit Hall with Poster Viewing5:00 pm
Close of Day6:00 pm
8月17日(木)
Registration and Morning Coffee7:30 am
LENTIVIRUS PROCESS DEVELOPMENT AND QUALITY
レンチウイルスのプロセス開発と品質
Overcoming the Challenges of Biomanufacturing Lentiviral Vector
Martin Loignon, PhD, Team Leader, Cell Engineering, National Research Council Canada
The demand for lentiviral vectors (LVs) for R&D and engineering cell therapies stems from their efficacy to deliver genes into targeted cells. Current LVs' production bioprocesses vary widely, significantly impacting quantities, quality, and costs. We have used a holistic approach to address challenges of upstream and downstream bioprocesses to increase titers and recovery.
Considerations in Development of Lentiviral-Based in vivo Gene Therapy
Mukesh Mayani, PhD, Head of Process Development, Gene Therapy, National Resilience, Inc.
Lentiviral vectors (LVV) have demonstrated noteworthy clinical success in patients during ex vivo CAR T as well as stem cell therapy gene therapy (GT) applications. The third-gen SIN LVVs have shown improved safety profiles for a conceivable durable treatment of rare disease and cancer indications. However, it’s application as in vivo therapy option is severely limited due to manufacturing, safety, and quality challenges. In this presentation, we will highlight several development and manufacturing considerations for generation of LV vector suitable for in vivo GT use.
Coffee Break in the Exhibit Hall with Poster Viewing9:00 am
Breakout discussions provide an opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. Please visit the breakout discussions page on the conference website for a complete listing of topics and descriptions.
MANUFACTURING IPSCs
iPS細胞の製造
Scalable Production of Pluripotent Stem Cell-Derived Hematopoietic Progenitor Cells and Functional T Cells in Stirred Tank Bioreactors
Liz Csaszar, PhD, Senior Director, Manufacturing Sciences, Tech Operations, Notch Therapeutics
Stirred suspension-based cell manufacturing can be used for scalable and controllable production of cell therapy products. We have developed custom reagents to modulate Notch signaling, which are compatible with suspension culture, and have implemented the production of pluripotent stem cell-derived CD8aß+ T cells in stirred tank bioreactors (STRs). STR-based culture enables process optimization and characterization using bioprocess solutions including automated feeding and in-process monitoring.
Engineering & Manufacturing iPSC-Derived Innate Cells to Provide Globally Scalable, Allogeneic Innate Therapies
Allen Qiang Feng, PhD, Founder and CSO, HebeCell Corp.
Human pluripotent stem cell (PSC)-derived natural killer (NK) cells combine the advantages of PSC and the safety profile of NK cells. At HebeCell we have developed our proprietary technology platform that is bioprocessing friendly and adaptable to GMP standards. Our feeder-free platform utilizes 3D spheroids to mimic the in vivo hematopoiesis to generate cytotoxic protoNK cells in bioreactors. Our platform offers a highly scalable approach for off-the-shelf cell therapies.
Sponsored Presentation (Opportunity Available)11:30 am
Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own12:00 pm
Refreshment Break in the Exhibit Hall & Last Chance for Poster Viewing12:30 pm
OPTIMIZING CELL THERAPY MANUFACTURING
細胞治療薬製造の最適化
Optimising Cell Therapy Manufacturing and Process Understanding
Athanasios (Sakis) Mantalaris, PhD, FAIMBE, Professor, BioMedical Systems Engineering Laboratory, Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology
Analysis of the metabolome can be used for the evaluation of pluripotent and multipotent stem cell cultures, affording early detection of physiological changes indiscernible with traditional culture monitoring techniques. Finally, metabolomics can be used for the evaluation of the quality of stem cell differentiation in 2D and 3D biomaterial cultures, providing a sensitive and robust state-of-the-art technology, guaranteeing high-quality cell therapy manufacturing.
NK and CAR-NK Processing Development
Dongfang Liu, PhD, Associate Professor, Director Immunoassay Development, Pathology & Immunology & Lab Medicine, Rutgers University
Currently available technologies for ex-vivo expansion of NK and CAR-NK cells using feeder cell expansion systems (e.g., K562 cells ) and cytokines (e.g., IL-2 in combination with IL-15) show several limitations telomere shortage, exhaustion, fratricidal killing, and regulatory concerns. Previous studies show that a human 721.221-mIL21 as a feeder cell can rapidly expand primary NK and CAR-NK cells. Based on this technology, we developed a non-feeder cell system to expand NK and CAR-NK cells. The results provided in this study show strong promise with the non-feeder cell expansion approach for functional NK and CAR-NK cells.
Encapsulated Cell Therapy: An Off-the-Shelf, Scalable Approach to Treating Solid Tumors
Lauren E. Jansen, PhD, Director, Process Development, Avenge Bio
Avenge Bio’s LOCOcyte platform consists of polymer encapsulated, allogeneic cells genetically engineered to produce immunomodulatory molecules for the treatment of previously intractable cancers. This presentation will highlight our approach to a successful technology transfer of our lead IL-2 program, AVB-001, into Phase I GMP manufacturing. In addition, we will discuss key considerations for future development and scale-up of this innovative cell therapy.
Networking Refreshment Break2:40 pm
DIGITAL INTEGRATION, DECENTRALIZED MANUFACTURING
デジタルインテグレーション、製造の分散化
The Application of Digital Informatics Methods to Manage Development and Production Data for Cell and Gene Therapies
William E. Janssen, PhD, Principal, WEJ Cell & Gene Therapy Consulting Services LLC
Explosive growth in the fields of informatics and cell and gene therapy (CGT) has occurred over the last four decades. Application of Informatics tools in CGT manufacturing can facilitate record keeping, quality management reviews and communications. An informatics backbone will be essential for deployment of disseminated manufacturing. While electronic records can completely replace paper and ink records, the potential benefits are much greater, including prospective quality management, error mitigation in manufacturing, release and administration, and reduction in redundancies. Implementation of informatics in CGT requires substantial planning, resources, and collaboration between CGT and informatics teams.
Decentralized Manufacturing
Could Your Cell Therapy Manufacturing Facility Be Working Harder for You?
Peter Walters, Fellow of Advanced Therapies, CRB
Optimizing cell therapy manufacturing facilities is challenging due to small-scale cell therapy batch sizes. With batch sizes as small as one patient, manufacturers must scale out rather than up, duplicating every inefficiency in the process. This presentation explores four manufacturing approaches for small-scale cell therapies: dedicated rooms, chasing workstations, segregated unit operations, and process-in-a-box systems. Each approach has its challenges, and manufacturers must balance risk tolerance, capital spending, and growth expectations to achieve manufacturing goals. Case studies comparing facility designs that enable throughput will be presented and compared.
Close of Summit4:25 pm
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