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2023年5月14日（日） 2:00 - 5:00 pm

SC1: Antibody Drug Discovery: From Target to Lead
SC1：抗体創薬 - ターゲットツーリード（T2L）から

At least 100 antibody therapies have been approved for the treatment of cancer, immune disorders, metabolic, cardiovascular, and infectious diseases, and among the top 20 bestselling prescription medicines in 2020, 14 are antibody-based. This trend will continue as about 50% of the new drugs in various stages of clinical development are antibodies. This course will review state-of-the-art concepts, methodologies, and current trends in therapeutic antibody discovery.

2:00 pm

Antibody Drug Discovery: From Target to Lead

Zhiqiang An, PhD, Professor, Molecular Medicine, University of Texas Health Science Center at Houston

Topics to be covered include:

Different sources of antibodies (animals, libraries, and B-cells)
Various antibody-based drug modalities (ADC, bispecific, naked antibody, and CAR T)
Antibody engineering (affinity maturation, humanization, isotypes, and formats)
Target selection and design including complex membrane proteins (GPCRs, ion channels, transporters, and membrane-bound enzymes)
Case studies

INSTRUCTOR BIOGRAPHIES:

Zhiqiang An, PhD, Professor, Molecular Medicine, University of Texas Health Science Center at Houston

Dr. Zhiqiang An is Professor of Molecular Medicine, the Robert A. Welch Distinguished University Chair in Chemistry, and Director of the Texas Therapeutics Institute at the University of Texas Health Science Center at Houston. His laboratory focuses on cancer antibody drug resistance mechanisms, biomarkers for cancer therapeutic antibodies, and antibody drug discovery targeting human diseases. Dr. An also directs the Therapeutic Monoclonal Antibody Lead Optimization and Development Core Facility funded by the Cancer Prevention and Research Institute of Texas (CPRIT). Previously, he served as Chief Scientific Officer at Epitomics, Inc. and was Director of Biologics Research at Merck Research Laboratories. He started his biotech career at Millennium Pharmaceuticals. Dr. An received his Ph.D. degree from the University of Kentucky and his postdoctoral training at the University of Wisconsin-Madison. Dr. An is well published in the field of antibody drug discovery including the award-winning book “Therapeutic Monoclonal Antibodies: from Bench to Clinic”. He started his biotech career at Millennium Pharmaceuticals. He is an elected fellow of Society for Industrial Microbiology and Biotechnology (SIMB), an elected fellow of the American Academy of Microbiology (ASM), an elected fellow of American Association for the Advancement of Science (AAAS), and an elected fellow of the National Academy of Inventors (NAI).

SC2: Introduction to Lipid Nanoparticle Characterization and Formulation
SC2：脂質ナノ粒子の特性評価と構築入門

2:00 pm

Introduction to Lipid Nanoparticle Characterization and Formulation

Jan Jezek, CSO, Arecor, United Kingdom

With increasing focus on nucleic acid-based therapies, particularly mRNA, lipid nanoparticles are emerging as the non-viral vectors of choice for their efficient delivery. The short course will review the field of lipid nanoparticle formulation and characterization, including (a) lipid nanoparticles in the broader context of lipid nanocarriers, (b) key structural features, (c) stability, (d) characterization, (e) factors influencing transfection efficiency, (f) factors influencing tissue/cell targeting, (g) manufacture and (h) lessons from the COVID vaccines development. Recent developments in patent landscape will also be covered.

INSTRUCTOR BIOGRAPHIES:

Jan Jezek, CSO, Arecor, United Kingdom

Jan Jezek is the Chief Scientific Officer at Arecor Ltd. He has been trained as a biophysical chemist. He was the principal scientist at Insense Ltd, leading the development of a range of novel medical devices from the proof-of-concept all the way to market. During his time at Insense, he and his team developed a novel formulation platform to achieve superior stability of proteins and other biological molecules. His inventions related to protein stabilization led to inception of Arecor Ltd as a separate company focusing on commercialization and further development of the stabilization platform.

SC3: In silico and Machine Learning Tools for Antibody Design and Developability Predictions
SC3：抗体設計と開発可能性予想へのin silicoと機械学習ツール

In silico developability predictive platforms offer promising screening support to identify optimal properties of a candidate biotherapeutic at early stages. Predicting your biologic’s developability can help avoid instability problems during later development and impede significant economic consequences.

2:00 pm

Structure and Machine Learning (ML) Guided Tools for Antibody Design and Developability Predictions

Philip M. Kim, PhD, Professor, Molecular Genetics & Computer Science, University of Toronto

Vinodh B. Kurella, PhD, Biotherapeutic Computational Modeler, Takeda Pharmaceuticals, Inc.

Christopher J. Langmead, PhD, Director of Digital Biologics Discovery, Amgen

Topics to be covered include:

Revealing a Global Biologics Research Informatics Platform integrating in silico prediction and lab experiment data to fast-forward biologics drug design and development
Putting the platform into practice with biologic discovery use cases
Elucidating process development best practices with use cases from Takeda

INSTRUCTOR BIOGRAPHIES:

Philip M. Kim, PhD, Professor, Molecular Genetics & Computer Science, University of Toronto

Philip M. Kim is a Professor at Donnelly Centre at the University of Toronto. He leads a research laboratory that integrates machine learning, physics-based modeling and wet/experimental methods for engineering of biologics. He is a Co-founder of Resolute Bio, a drug development startup company. He authored over 70 publications, 7 invention disclosures and 3 patent applications. Before to setting up his lab in 2009, he was a postdoctoral fellow at Yale University where he pioneered structural analyses of protein interactions networks and an associate with McKinsey & Co. He holds a Ph.D. from the Artificial Intelligence Laboratory and Department of Chemistry at the Massachusetts Institute of Technology and a B.S. in Biochemistry and Physics from the University of Tuebingen.

Vinodh B. Kurella, PhD, Biotherapeutic Computational Modeler, Takeda Pharmaceuticals, Inc.

Vinodh Kurella is a Senior Scientist (Biologics computational modeler) in Biotherapeutic Engineering Group (BE) within Global Biologics at Takeda (Cambridge, USA). Experienced in structure-based antibody/protein design and optimization. Previously had range experiences at various biotech companies in different modalities such as gene therapy, biologics, and CAR T designs. Post-doctoral training at Harvard Medical School/Dana Farber in Dr. Wayne Marasco laboratory in antibody engineering and graduate training from Louisiana State University (LSU-HSC) in the field of protein X-ray crystallography in David Worthylake laboratory.

Christopher J. Langmead, PhD, Director of Digital Biologics Discovery, Amgen

Christopher James Langmead is the Director of Digital Biologics Discovery at Amgen. His team at Amgen develops and uses AI/ML methods to design and optimize of biologics. He earned his PhD in computer science from Dartmouth and then spent the next 18 years as faculty in computer science and computational biology at Carnegie Mellon University where his group developed a variety of generative models relevant to protein design, including GREMLIN. Dr. Langmead joined Amgen at the beginning of 2022.

SC4: An Introduction to Protein Degraders: A Focus on PROTACs
SC4：プロテインデグレーダー入門 - PROTAC（タンパク質分解誘導キメラ分子）への注目

This seminar will give an overview of proteolysis targeting chimeras (PROTACs) and will introduce some important topics relevant to how they work as well as the challenges of developing them as oral therapeutics. Topics to be covered include examples of what they can accomplish that small molecules cannot, importance of ternary complex formation, and how proteomics is essential for their development. Strategies to increase their selectivity, such as antibody PROTACs, will be examined. PROTACs lie in Bro5 space, and we will cover the significance of this for developing them as oral drugs. Finally, the mechanism of action of PROTACs give rise to some unique drug safety issues which will also be discussed.

2:00 pm

An Introduction to Protein Degraders: A Focus on PROTACs

John Erve, PhD, Senior Principal Scientist, Jerve Scientific Consulting

Topics to be Covered:

Advantages of PROTACs compared to small molecules
Importance of ternary complex formation
Strategies to enhance PROTAC selectivity
Why proteomics is essential for developing PROTACs
Challenges of developing PROTACs in the context of Bro5
Safety issues unique to PROTACs

INSTRUCTOR BIOGRAPHIES:

John Erve, PhD, Senior Principal Scientist, Jerve Scientific Consulting

John Erve is from Chicago and studied Chemistry (BS, MS) at the University of Chicago and earned a PhD in Toxicology at Oregon State University. Following postdoctoral work at Vanderbilt (1995-1999) he joined BD-Biosciences (Woburn, MA) as a Study Director. In 2002, he joined AstraZeneca (Sweden) where he characterized reactive metabolites. In 2004 he joined Wyeth (Collegeville, PA) as a Principal Scientist responsible for metabolite identification. In 2010, John joined Novartis (Cambridge, MA) as a Lab Head in Analytical Sciences. John returned to drug metabolism at Elan Pharmaceuticals (San Francisco, CA) in 2012 and later formed Jerve Scientific Consulting, Inc to help small biotech companies in the Bay area with their drug discovery efforts. John was a certified D.A.B.T. from 2004 to 2019.

2023年5月16日（火） 6:30 - 9:00 pm

SC5: Introduction to Gene Therapy Product Manufacturing and Analytics
SC5：遺伝子治療製品製造と分析入門

This short course introduces concepts that can be used to facilitate CMC development for gene therapy products. The instructors will review regulatory guidance and present phase-appropriate control strategies. Several CMC challenges unique to this modality will also be discussed, along with different manufacturing platforms. The workshop will include an interactive session on developing an integrated control strategy.

6:30 pm

Introduction to Gene Therapy Product Manufacturing and Analytics

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

Scott Dooley, Senior Scientist, Analytical Development, Sanofi

Topics to be covered include:

Overview of Manufacturing for Gene Therapy Products and Parallels with Protein Therapeutics
Analytical Development Strategies
Phase-Appropriate Control Strategy
Regulatory Guidance and Expectations for Gene Therapy Products
Specific Challenges in Comparison to Protein Therapeutics
Potential for Leveraging Platforms and Benefits
Interactive Workshop on Developing a Control Strategy
Non-Viral Vectors Including Lipid Nanoparticles

INSTRUCTOR BIOGRAPHIES:

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

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 20 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.

Scott Dooley, Senior Scientist, Analytical Development, Sanofi

After obtaining his master’s degree from the University of Massachusetts Amherst in 2013, Scott Dooley began working for the Analytical Development department at Sanofi. Over the past 8 years, Mr. Dooley has supported analytical method development and validation for enzyme-replacement therapies, antibodies, and gene therapy products, and acted as the analytical team leader for several products. Currently, Mr. Dooley acts as the analytical team leader within CMC for two products in clinical development.

SC6: Developability of Bispecific Antibodies
SC6：二重特異性抗体の開発可能性

Bispecific antibodies are a rapidly growing and clinically validated class of antibodies with marketed drugs and multiple candidates in clinical trials. Targeting multiple antigens in a synergistic manner can confer enhanced therapeutic benefits and potentially uncover novel biological mechanisms. However, multiple formats and a tedious candidate selection process to select functional and developable bispecific antibodies make such programs cumbersome. This short course highlights the rapid growth in the field, therapeutic applications, and it focuses on challenges with discovery and development of bispecific antibodies. We will use an approved bispecific antibody as a case study to understand the varied aspects of discovery and development of bispecific antibody programs.

6:30 pm

Developability of Bispecific Antibodies

Nimish Gera, PhD, Vice President, Biologics, Mythic Therapeutics

Topics to be Covered Include:

Introduction to bispecifics and bispecific formats
Therapeutic applications of bispecific antibodies
Developability of bispecifics
Case study: discovery and development of an FDA-approved bispecific antibody

INSTRUCTOR BIOGRAPHIES:

Nimish Gera, PhD, Vice President, Biologics, Mythic Therapeutics

Nimish Gera is the Vice President of Biologics at Mythic Therapeutics leading multiple projects to engineer and develop novel antibody and antibody-based drugs in oncology and immuno-oncology. Prior to Mythic, Nimish has over ten years of experience in antibody and protein engineering with five years leading bispecific antibody programs in several disease areas such as rare diseases, oncology and immunology at Alexion Pharmaceuticals and Oncobiologics. Nimish received his PhD degree in Chemical and Biomolecular Engineering from North Carolina State University and a B.Tech degree in Chemical Engineering from Indian Institute of Technology, Guwahati.

SC7: Use and Troubleshooting of Eukaryotic Expression Systems
SC7：真核生物発現システムの使用とトラブルシューティング

Eukaryotic expression systems are extensively used for the generation of recombinant proteins thereby becoming an essential protein engineering tool. The choice of a suitable eukaryotic expression system depends mainly on the biological and biochemical properties of an individual protein. The course will focus on both the insect and mammalian expression systems, which have demonstrated the ability to express complex proteins for a wide variety of applications. We will discuss the concepts, uses, and optimization of these systems along with sharing experimental troubleshooting lessons learned. The course combines instruction and case studies in an interactive environment.

6:30 pm

Use and Troubleshooting of Eukaryotic Expression Systems

Richard Altman, MS, Field Application Scientist, Life Science Solutions, Thermo Fisher Scientific

Henry C. Chiou, PhD, Senior Director General Manager, Biosciences, Thermo Fisher Scientific

Dominic Esposito, PhD, Director, Protein Sciences, Frederick National Laboratory

6:30 pm Opening Remarks/Introductions

Richard Altman, MS, Field Application Scientist, Protein Expression, Biosciences Division, Life Sciences Solutions Group, Thermo Fisher Scientific

6:45 pm Insect Expression Systems

Dominic Esposito, PhD, Director, Protein Expression Laboratory, Frederick National Laboratory for Cancer Research

7:15 pm Mammalian Expression Systems

Henry C. Chiou, PhD, Senior Director, Cell Biology, Life Science Solutions, Thermo Fisher Scientific

7:45 pm Coffee Break

8:00 pm Implementing and Optimizing Eukaryotic Expression Workflow

Richard Altman, MS, Field Application Scientist, Protein Expression, Biosciences Division, Life Sciences Solutions Group, Thermo Fisher Scientific

8:30 Q&A

INSTRUCTOR BIOGRAPHIES:

Richard Altman, MS, Field Application Scientist, Life Science Solutions, Thermo Fisher Scientific

Rich Altman has 30 years of experience in protein expression and production. In early 2019, he joined Thermo Fisher Scientific as a Field Application Scientist. Previously, he worked for several pharmaceutical companies, including Amgen, Alexion, Bayer, and Upjohn, on the cloning, expression, purification and characterization of recombinant proteins. This work supported both small-molecule high-throughput screening and protein therapeutic efforts. He received his MS degree from the University of Pittsburgh School of Medicine in the Department of Molecular Biology and Biochemistry.

Henry C. Chiou, PhD, Senior Director General Manager, Biosciences, Thermo Fisher Scientific

Henry Chiou is Senior Director and General Manager for the Delivery and Protein Expression business within Biosciences at Thermo Fisher Scientific. He and his teams have developed products such as the Expi family of 293, CHO and Sf9-based expression systems, Lipofectamine 3000, and other Lipofectamine-family transfection reagents, production systems for cell and gene therapy viral vectors such as AAV MAX system. Henry has authored multiple publications on mammalian transient expression and frequently teaches courses and lectures on this subject. Prior to joining Thermo Fisher, Henry worked in small to mid-sized biotech companies on non-viral gene therapy. Henry received his doctorate from Harvard University in Molecular Pharmacology, following which he completed a postdoctoral fellowship in viral expression systems at the University of Pennsylvania.

Dominic Esposito, PhD, Director, Protein Sciences, Frederick National Laboratory

Dr. Esposito is currently the Director of the Protein Expression Laboratory (PEL) and Project Lead for the RAS Reagents Core at the Frederick National Laboratory for Cancer Research in Frederick, Maryland. The 33 employees in the PEL clone, express, and purify proteins from a variety of host organisms in support of the NCI RAS Initiative and for investigators at the National Institutes of Health. In addition, the PEL invents and develops novel technologies for improving protein expression and production, focused heavily on baculovirus expression technology and combinatorial cloning. Prior to his role as director, Dr. Esposito led the Clone Optimization Group in the PEL for nine years and was responsible for the generation of over 15,000 expression clones, 400 new expression vectors, and several technological innovations in protein expression. Dr. Esposito received his B.A. in Chemistry at La Salle University in Philadelphia, and his Ph.D. in Biochemistry in the laboratory of Dr. John Scocca at the Johns Hopkins University Bloomberg School where he studied bacteriophage site-specific recombination. Dr. Esposito then worked as a postdoctoral fellow in the laboratory of Dr. Robert Craigie at the NIDDK, where he studied the protein-DNA interactions involved in the HIV integration reaction. Prior to joining the FNL in 2001, Dr. Esposito worked for three years as a Staff Scientist in the Protein Engineering group at Life Technologies, where he helped to develop the Gateway recombinational cloning system. Dr. Esposito has published more than 70 peer-reviewed manuscripts in the fields of protein expression and DNA recombination.

SC8: CAR T Cells: Improving Safety While Retaining Therapeutic Activity
SC8：CAR T細胞 - 治療活性を保持する間の安全性の向上

CD19 CAR T cells have revolutionized the treatment of B cell malignancies. However, CD19 CAR T cells have their drawbacks in that there can be serious adverse events and not all patients go into complete remission. Furthermore, CAR T cells have not as effective for other cancers, especially solid tumors. In this course, we will provide an overview of the current research/clinical landscape for CAR T cells will be reviewed, and next potential steps will be discussed.

6:30 pm

CAR T Cells: Improving Safety While Retaining Therapeutic Activity

Nasheed M. Hossain, MD, Assistant Professor of Medicine, University of Pennsylvania - Perelman School of Medicine, Department of Medicine - Division of Hematology/Oncology, Cell Therapy & Transplant Program

Michael I. Nishimura, PhD, Professor, Surgery, Loyola University Chicago

Rimas J. Orentas, PhD, Scientific Director, Caring Cross, Inc.; Professor, University of Washington School of Medicine

Considerations for target selection and results in the clinic using CAR T cells against that target
Adverse events encountered in patients treated with CAR T cells
Manufacturing hurdles and practical considerations for CAR T products
New approaches for making better CAR T products

INSTRUCTOR BIOGRAPHIES:

Dr. Nasheed M. Hossain is an Assistant Professor of Medicine at the University of Pennsylvania. During his BMT fellowship at Stanford and as an Assistant Professor at Loyola and the University of Pennsylvania, he was instrumental is designing and implementing CAR T clinical protocols and caring for CAR T patients. Dr. Hossain’s research focuses on understanding the factors leading to improving the safety of CAR T cells while retaining their therapeutic efficacy. Dr. Hossain’s research also focuses on leveraging insights in T-cell biology on the prevention and treatment of Graft versus Host Disease in Allogeneic Stem Cell Transplant patients.

Michael I. Nishimura, PhD, Professor, Surgery, Loyola University Chicago

Dr. Michael I. Nishimura is a Professor in the Department of Surgery at Loyola University Chicago. He has devoted most of the past 30 years of my career to studying TCR genes and developing novel strategies to use them for the purpose of treating patients with advanced malignancies and viral infections. More recently, we have applied our T cell engineering expertise to CAR T cells. We have conducted clinical trials using TCR and CAR T cells. We have objective clinical responses without associated toxicity from the gene modified T cells. Our clinical and laboratory studies are guiding the development of new and safer products for treating cancer patients.

Rimas J. Orentas, PhD, Scientific Director, Caring Cross, Inc.; Professor, University of Washington School of Medicine

Dr. Rimas J. Orentas is an investigator at the Ben Towne Center for Childhood Cancer Research, Seattle Children’s Research Institute and a Professor in the Department of Pediatrics, University of Washington School of Medicine. Research in his academic laboratory focuses on CAR T approaches towards treating pediatric solid tumors such as rhabdomyosarcoma, Ewing sarcoma, and osteosarcoma. He continues to develop CAR T approaches to hematologic malignancies and has created 5 first-in-human CAR T products. Dr. Orentas is also a co-founder of the non-profit organization, Caring Cross, Inc., whose goal is to bring cell and gene therapy approaches to sickle cell disease, HIV, and malignancy to low- and middle-income countries.

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