ショートコース

6月1日 (月) 18:00~21:00

SC1: Particles in Biotherapeutics: Characterization & Impact
( ショートコース1:生物学的製剤に含まれる粒子:特性評価と影響 )

Presenters:

Dean C. Ripple, PhD, Group Leader, Bioprocess Measurements Group, NIST

Srivalli Telikepalli, PhD, Research Chemist, Biomolecular Measurement Division, NIST

Presentations will include:

Introduction: 

This short course will give an introduction to current issues surrounding particle formation & characterization in biotherapeutics. Regulatory expectations provide the context of why particle loads are characterized and controlled. The basics of why and how proteins can aggregate will be presented along with a discussion of other particle types. An overview of the recent technology to accurately characterize various classes of aggregates and particles will be discussed. Studies from the current literature will be used to highlight various key points throughout the course.

Topics covered include:

1. Introduction to Protein Aggregates & Other Particles

  • Overview of particles in biotherapeutics: protein aggregates & other types
  • Current regulatory expectations
  • Rationale for characterization & control
  • Recent experiences & case studies

2. Causes & Mechanisms behind Aggregation/Particle Formation

  • Causes of protein aggregation
  • Mechanisms behind formation of aggregates
  • Sources of other types of particles
  • Methods to minimize aggregation & particle formation
  • Case studies

3. Technologies & Tools for Measuring & Characterizing Aggregates & Particles

  • Visible particles (manual and automated inspection)
  • Sub-visible particles (microflow imaging, light obscuration, etc.)
  • Sub-micrometer aggregates (size exclusion SEC, AUC, FFF, etc.)  
  • Approaches to standardize particle counts
  • Comparisons between technologies regarding limits of detection

4. Strategies for Particle Control & Characterization

5. Discussion with Q&A

Presenter Biographies

Dean received a Ph.D. from Cornell University in 1991 and began a post-doctorate position at the National Institute of Standards and Technology the same year. After a successful career at NIST supporting improved methods and standards in thermometry, in 2010 he became Leader of the Bioprocess Measurements Group. His research focuses on the development of new standards for protein particles. Dean has presented workshops on measurement issues to many industrial groups, ranging from petroleum producers to vaccination program coordinators. He is an active member of ASTM and USP committees and has received awards from the Department of Commerce, ASME, and ASTM.

Srivalli N. Telikepalli received a B.A. degree in Chemistry at Cornell University in 2008 and a Ph.D. in Pharmaceutical Chemistry from the University of Kansas in 2014. Her graduate work was focused on understanding the formation of protein aggregates/particles, their characterization, and their possible correlations to immunogenic responses. In 2014, she started working as a Research Chemist at the National Institute of Standards and Technology. She has contributed to the development of the NIST Monoclonal Antibody Reference Material and a subvisible protein-like particle reference material. Her work focuses on developing protein-like particle standards that enable more robust characterization of proteinaceous particles in biotherapeutics. She received a United States Department of Commerce Gold Medal Award in 2017 for her contributions.

6月1日 (月) 18:00~21:00

SC2: Advanced Analytical Technologies for Developability and Early Formulation Assessments
( ショートコース2:開発可能性と早期製剤評価のための先進的な分析技術 )

Danny K. Chou, PhD, Founder & CEO & President, Formulation Development & Protein Characterization, Compassion BioSolutions LLC

Introduction:

For biopharmaceuticals, drug design, lead selection and formulation/manufacturing process development constitute significant areas of risk because of their decisive influence on product quality, biological activity and safety, as well as cost of goods. The purpose of this short course is to introduce how a range of advanced analytical technologies, along with the concept of Quality by Design (QbD) may be incorporated at the interface of drug discovery and development in order to both select drug candidates with the best inherent stability and deliver the most suitable formulation for these molecules. Part of the course will be focused on the practical tools (both conceptual tools and analytical tools) one can use to achieve this objective.

Topics covered include:

  • What are developability and formulability and how they relate to the principles of Quality by Design?
  • How to assess formulability of a new therapeutic protein drug candidate: Define the suitability of a given drug candidate to be formulated for a desired route of administration or delivery method.
  • What are some off-the-shelf tools that are available (statistical, time-saving approaches, and analytical tools that can help us assess molecules on attributes include, among others, solubility, aggregation, viscosity or product stability.
  • A real case study of how one implements some of the tools mentioned above to evaluate new therapeutic protein drug candidates.

Who Should Attend?

Scientists, Project leaders, or Heads of departments for drug discovery, formulation, R&D, analytical development, process development, technical and pharmaceutical development.

Presenter Biography

Dr. Danny K. Chou is a biopharmaceutical industry veteran with expertise in biopharmaceutical characterization, formulation development, and emerging technologies for protein aggregate/subvisible particle analysis. Currently, Dr. Chou is the Founder and President of Compassion BioSolution, a biopharmaceutical consultancy and Contract Development Service provider that serves clients throughout the world. Dr. Chou has over 20 years of experience in the pharmaceutical industry, both as a pharmacist and pharmaceutical scientist. Over the past 15 years he has led the development of formulations for numerous therapeutic modalities ranging from peptides, growth factors, mAbs, ADCs, and bispecific antibodies. Prior to starting Compassion BioSolution, Dr. Chou was a Senior Scientist and Group Leader at Gilead Sciences, where he successfully built up state-of-the-art analytical capabilities for the company and converted IV formulations of monoclonal antibodies to high concentration formulations that are more stable and can be easily administered by subcutaneous injection. Prior to this, Danny was employed by Genzyme and Amgen, where he played critical roles in drug product process development, manufacturing technical support, and pharmaceutical development. Since founding Compassion BioSolution, Danny has developed stable pharmaceutical dosage form for clients ranging from small start-up biopharmaceutical companies to Fortune 500 pharmaceutical companies. Danny received his PhD from the University of Colorado Center for Pharmaceutical Biotechnology under a NIH Fellowship and his PharmD from the University of Florida.

6月4日 (木) 18:00~21:00

SC3: Critical Quality Attributes and Testing Strategy for Biotherapeutics Development
( ショートコース3:重要品質特性と生物学的製剤開発のための試験戦略 )

Christine P. Chan, PhD, Director, Global Manufacturing Science & Technology, Sanofi

Introduction:

Biotherapuetics are challenging to develop due to the complexity of the molecular structure as well as the manufacturing process. Identification of product critical quality attributes (CQAs) is an important component in the development of a robust control strategy using the Quality-by-Design approach.  In this short course, we will discuss the key concepts of CQA risk ranking based on potential impact on safety and efficacy, defining control strategies, the common analytical characterization technologies used, and the considerations for development of an integrated testing strategy.

Topics covered include:

  • CQA criticality assessment: leveraging prior knowledge and gathering product structure-function relationship (SAR) information; 
  • Acceptable ranges through development stages
  • Analytical characterization strategy and test methods selection: commonly used assays and emerging tools
  • Conducting forced degradation studies; product stability profile
  • Linkage of process to product quality: defining control strategies, comparability studies
  • Towards a streamlined testing strategy: evolving the test panel and specifications through the product lifecycle
  • Practical examples and discussions

Presenter Biography

Dr. Christine Chan is a protein biochemist with broad experience in the biopharmaceutical industry, including prior experience at Sandoz Pharmaceuticals and Merck & Co., Inc. She specializes in the analysis of recombinant products produced from mammalian cells for vaccines and biologics development. Her application experience includes expression cell line selection, drug substance & drug product process development, manufacturing tech transfers and lifecycle management of commercialized products.

6月4日 (木) 18:00~21:00

SC4: Gene Therapy Products: Phase-Appropriate Analytical Development Strategies
( ショートコース4:遺伝子療法薬:臨床試験の各段階に応じた分析法の開発戦略 )

Presenters:

Francis Poulin, PhD, Scientific Director, Analytical Development, Sanofi

Claire Davies, PhD, Associate Vice President, Bioanalytics, Sanofi

Presentations will include:

Introduction:

This short course introduces concepts that can be used to facilitate analytical development for gene therapy products.  The instructors will review regulatory guidance and present phase-appropriate analytical development and control strategies. Several challenges unique to this modality will also be discussed. The course combines instruction and an interactive workshop on platform method development.

Topics covered include:

  • Overview of Manufacturing for Gene Therapy Products
  • Analytical Development Strategies
  • Phase appropriate Control strategy
  • Comparability and Stability
  • Regulatory guidance and unique challenges for gene therapy
  • Interactive workshop on platform method development

Who Should Attend:

Research Associates, CMC and analytical project leads, regulatory affairs specialists and managers, process and analytical development scientists.

Presenter Biographies

Francis Poulin is a Director of Analytical Development at Sanofi in Framingham, Massachusetts, where he is responsible for the development, qualification and validation of bioassays and molecular assays to support the release of biologics in preclinical and clinical development, including gene therapy products. Prior to joining Sanofi, Francis was a postdoctoral fellow at HHMI/University of California, Berkeley and the Lawrence Berkeley National Laboratory. Francis received his PhD in Biochemistry from McGill University.

After obtaining her PhD from the University of London in 2001, Claire Davies performed her postdoctoral work at the William Harvey Research Institute (WHRI) at Bart's and The London, Queen Mary's School of Medicine and Dentistry and Joslin Diabetes Center, Harvard Medical School, Boston. Over the last 18 years, Dr Davies has led analytical and CMC teams in product development and analytical method development and validation. Currently, Dr Davies leads Bioanalytics, a group responsible for developing methods and strategies to support process development, product characterization and release and stability testing for therapeutic proteins and gene therapy products in preclinical and clinical development.

* 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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更新履歴
2020/02/25
アジェンダ更新


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