Cambridge Healthtech Instituteの第8回年次会議
Cell Therapy CMC and Analytics
2023年8月14 - 15日、EDT（米国東部標準時）
Registration and Morning Coffee8:00 am
CMC STRATEGIES FOR CELL THERAPIES
Innovative CMC Strategies for Cell and Gene Therapies
Zhimei Du, PhD, Vice President, Translational Research and Early Development, LandMark Bio
Most cell and gene therapy (CGT) programs have their origins in academic research and have been developed in academic settings. However, this has resulted in challenges with establishing robust manufacturing controls and ensuring consistency in quality. Another prevalent issue in the field is the rush to bring products to market without adequate product and process understanding. This presentation will delve into the importance of developing a new roadmap and strategy for cell and gene therapy (CGT) to strike a balance between the speed to clinic and the success rate of commercialization.
Raw Material Qualification for Cell Therapies
Ben Clarke, PhD, Senior Scientist, USP
USP is continuing to develop reference standards, informational chapters, and compendial analytical methods to safeguard raw, starting, and ancillary materials for cell therapies. USP’s standards give best practice guidance to developers and manufacturers, simplify risk assessments, accelerate analytical development, and support raw material qualification and release. This presentation will describe existing standards and USP's recent development related to plasmid DNA and rapid microbial methods.
Standards Development and Control Strategies for Cell Characterization and Cell Viability
Sumona Sarkar, PhD, Biomedical Engineer, Biosystems and Biomaterials Division, Biomaterials Group, National Institute of Standards and Technology
The manufacturing and release of cellular therapy products (CTPs) requires high quality, robust, and validated analytical methods. Here I will describe recent efforts in standards development and public-private partnerships to support the development of critical analytical methods used in advanced therapies. A key aspect of analytical development for these new class of products is the need for a fit-for-purpose approach when identifying CQAs and designing appropriate analytical methods. Cell viability is a fundamental measurement for cell-based therapies which also requires a fit-for-purpose approach. A framework for establishing fit-for-purpose cell viability assays will be described as well as ongoing standards efforts.
Sponsored Presentation (Opportunity Available)11:30 am
Session Break12:30 pm
BUILDING QUALITY INTO CELL THERAPY DEVELOPMENT
Building Quality into the CMC - Real World Perspectives from an Academic GMP Facility
Patrick J. Hanley, PhD, Associate Professor, Pediatrics; Chief & Director, Cellular Therapy Program, Children's National Hospital
In this presentation we will discuss real world examples of how to build quality into the CMC and how real world deviations and incidents have led to a better quality program. Examples will include improvements to change control, training, operations, and testing.
Networking Refreshment Break2:25 pm
Genetically-Modified Cell Therapies and Multi-Edited iPSCs-Derived Products
Session Break and Transition to Plenary Keynote Session3:40 pm
PLENARY KEYNOTE: SOLVING TODAY'S CHALLENGES
Overcoming the Challenges of Bioprocesses: The Future of Biomanufacturing
Glen R Bolton, PhD, Executive Director, Late Stage Bioprocess Development, Amgen Inc
Novel therapies and technologies are emerging to meet the needs of patients; however, the manufacturing of biopharmaceuticals remains a complex and challenging process. As demand for biopharmaceuticals grows, the industry faces new challenges in terms of scalability, cost, and process robustness. The implementation of innovative technologies to improve process efficiency and the importance of process control and data analytics in ensuring process robustness are key levers to meet these challenges.
Commercializing Gene Therapies - The Combined Power of Patient Advocacy and Cost-Effective Manufacturing
Rachel Salzman, DVM, Founder, The Stop ALD Foundation & Global Head, Corporate Strategy, Armatus Bio
There is only a very small handful of FDA-approved gene therapies. This presentation will examine the development of an FDA-approved gene therapy where patient advocacy played a critical role resulting in the first-ever clinical use of a lentiviral vector. Although manufacturing continues to represent a significant challenge throughout the entire R&D journey, there are opportunities for advocacy and manufacturing communities to seek alignment and combine their collective powers to achieve the common goal of increasing patient access to transformative medicines.
Welcome Reception in the Exhibit Hall with Poster Viewing5:30 pm
Close of Day6:30 pm
Registration and Morning Coffee7:30 am
TECH TRANSFER, MOVING FROM ACADEMIC TO COMMERCIAL
Technology Transfer for Cell Therapies
Scott R. Burger, Principal, Advanced Cell & Gene Therapy LLC
We explore how an objective technology transfer approach should be applied for cell therapy products and provide detail of the mechanisms and tools which should be used in order to make sure that the transition from development to cGMP is correctly achieved.
Accelerating T Cell Therapy Product Development through a Joint Industry-Academia Collaboration
Therese Choquette, PhD, Director, Analytics, Tigen
Tigen Pharma and CHUV in Lausanne, Switzerland, have formed a unique and close collaboration in development of tumor-infiltrating lymphocytes for treatment of solid tumors. The deep scientific knowledge of the Coukos and Harari teams at CHUV and the industrial expertise in Tigen accelerates the path of product to patient. This talk presents examples of this collaboration for progress in manufacturing and analytics for TIL in future clinical trials.
Critical Quality Attribute Assessment and Product Characterization for Base Editing Cell Therapy Drug Products
Bo Yan, PhD, Director, Analytical Research & Development, Beam Therapeutics
Base editing offers a fundamentally new strategy for precise gene editing. We combine base editing technology with available delivery methods to provide innovative gene editing and cell therapies. Multiple critical materials (such as gRNA, mRNA, plasmid, etc,), and delivery vehicles (such as lentivirus, adeno-associated virus AAV, and lipid nanoparticles) mean the processes and products are very complex. As a result, unprecedented analytical needs arise from such complex processes and drug products. Here, I will discuss the overall analytical development strategy in developing base editing drug candidates.
Coffee Break in the Exhibit Hall with Poster Viewing10:00 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.
LENTIVIRAL QUALITY AND ANALYSIS
New Programs, New Opportunities; Utilizing a Phase II Trial to Rethink Potency Workflow
Eric Bolf, PhD, Scientist, Analytical Development, 2SeventyBio
As we have gained experience with analytical methods, we have learned more about the pain-points associated with our assays. With renewed focus on efficiently bringing our pipeline into the clinic, we have taken this opportunity to redevelop how we measure lentiviral vector potency. This talk will discuss our new methodology, including approaches to improve assay throughput, improve consistency of the readouts, and reduce assay complexity.
Optimization of Nuclease Digestion in a Lentiviral Vector Process for Improved Reduction of DNA Impurities
James Xin, Scientist, Vector Process Development, ElevateBio
In the C> field, process-related DNA impurity levels are a safety focal point. Here, we provide the statistical analysis results based on several DoE studies or reduction of hcDNA and pDNA levels in our Lentiviral Vector (LVV) platform process. Focusing on several critical parameters including nuclease concentration, supplement concentration, incubation time, and incubation conditions (ph/temperature), this data shows the optimal factors and ranges in the nuclease digestion step.
Irfan Bashir, PhD, Anglia Ruskin university
- Formulation of LNPs using High Pressure Homogenizer and Probe Sonication Method
- Characterizations of formulations i.e. Osmolality, Size, Zeta Potential, PDI, DSC, Drug entrapment efficiency, Stability
- Statistical optimization of LNPs using osmolality centered approach
Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own1:00 pm
Refreshment Break in the Exhibit Hall with Poster Viewing1:30 pm
FLOW CYTOMETRY AND CHARACTERIZING IPSCs
Sensitivity, Precision, and Misconceptions when Flow Cytometry Is Not Performed by You
Ruud Hulspas, PhD, Technical Director, Process Development, Dana-Farber Cancer Institute
Rare cell analysis on large numbers of cells is important in characterization of cell products as unwanted effects can be caused by just a few cells. Conventional flow cytometers are designed to accommodate assay development, allowing operators to fit instrument settings to their specific needs. The 'open' design of this instrument brings a significant level of complexity to flow cytometry and challenges reproducibility in manufacturing processes of therapeutic cells.
Moving toward Meaningful Characterization of iPSCs by Live Imaging to Accurately Monitor Cell Behavior
Anthony Asmar, PhD, Biologist, National Institute of Standards and Technology
Live-cell imaging can provide quantitative dynamic and spatial characteristics of iPSCs in culture. Our research is geared toward developing systematic workflows quantifying the effects of culture and other handling conditions on iPS cell characteristics such as rates of mitosis, changes in morphology, and expression of relevant transcription factors to predict and evaluate the effects of manufacturing conditions and other perturbations on the state of the population.
Characterizing CARs from the Cell Surface Using Immunoprecipitation-Mass Spectrometry
Nicolle Serrano SantoDomingo, Research Scientist II, Novartis
CAR-T cells are engineered T cells expressing a chimeric antigen receptor (CAR) on the cellular surface. CARs allow the T cell to engage an antigen on tumor cells and activate downstream signaling that leads to destruction of the tumor cell. Surface expression of the CAR is critical for therapeutic efficacy, but often differences in efficacy are observed between constructs that show similar surface expression. We developed a workflow using immunoprecipitation of the CAR from the cell surface to characterize post-translational modifications by LC-MS (IP-MS) to gain insights on differences in efficacy.
Refreshment Break in the Exhibit Hall with Poster Viewing3:45 pm
Cell Therapy Analytical Toolkit
Victor Muthu, PhD, Principal Scientist, Analytical Development, National Resilience
My presentation will highlight the most recent analytical development methods in the cell therapy field and how our team at Resilience is pushing the boundaries of developing the cell therapy analytical toolbox by utilizing advanced platforms for assay development and qualification.
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.
Close of Cell Therapy CMC and Analytics Conference5:30 pm