Training Seminars - 2024 Archive

2024 ARCHIVES

Cambridge Healthtech Institute Training Seminars offer real-life case studies, problems encountered and solutions applied, along with extensive coverage of the academic theory and background. Each Training Seminar offers a mix of formal lecture and interactive discussions and activities to maximize the learning experience. These Training Seminars are led by experienced instructors who will focus on content applicable to your current research and provide important guidance for those new to their fields.

Training Seminars Will Be Offered In Person Only

Monday, May 13, 2024 8:30 am - 6:05 pm | Tuesday, May 14, 2024 8:00 am - 1:30 pm

TS3A: Introduction to Multispecific Antibodies: History, Engineering, and Application

Detailed Agenda

Introduction to Multispecific Antibodies will be organized as an informative and practical guide to getting up to speed on critical aspects of bispecific antibody therapeutics. Topics will include historical successes, failures, and lessons learned. Specific practical instruction will span mechanisms of action, engineering, developability, regulatory considerations, and translational guidelines. Perspectives on the ideal implementation of multispecifics as targeted and immunomodulatory approaches will be discussed.

G. Jonah Rainey, PhD, Senior Director, Protein Engineering, Eli Lilly and Company

Introduction to Multispecific Antibodies will be organized as an informative and practical guide to getting up to speed on critical aspects of bispecific antibody therapeutics. Topics will include historical successes, failures, and lessons learned. Specific practical instruction will span mechanisms-of-action, engineering, developability, regulatory considerations, and translational guidelines. Perspectives on the ideal implementation of multispecifics as targeted and immunomodulatory approaches will be discussed.

Topics to be Covered:

A brief history of bispecific antibodies: 60 years of progress with critical advances and key pioneers

Bispecific applications and powerful mechanisms-of-action

Engineering bispecific antibodies:100 formats and counting

Bispecific-specific considerations in preclinical development and regulatory landscape

Developability, manufacturing, and analytical considerations

Clinical experience, translation, and regulatory approval

Current trends and future opportunities in regulating immune checkpoints, cell-based therapies, and personalized approaches

INSTRUCTOR BIOGRAPHIES:

G. Jonah Rainey, PhD, Senior Director, Protein Engineering, Eli Lilly and Company

Jonah Rainey holds a PhD in Biochemistry from Tufts University and completed postdoctoral training at the University of Wisconsin and the Salk Institute. He has engaged in discovery, research, and development of bispecific antibodies for more than 15 years. He is an inventor on several patents describing novel bispecific platforms and current clinical candidates that exploit these platforms as well as an author on almost 30 publications. Jonah contributed to research and early development leading to multiple clinical candidates from Phase I and through approved products and led many advanced preclinical programs in oncology, infectious disease, autoimmunity, and other therapeutic areas. Previous industry experience includes MacroGenics, MedImmune/AZ, Oriole Biotech, Gritstone Oncology, and Alivamab Discovery Services. Currently, Jonah is a Senior Director in Protein Science at Eli Lilly & Co.

TS7A: Introduction to Immunogenicity

Detailed Agenda

This 1.5-day training seminar provides a practical, comprehensive overview of immunogenicity—the causes, how to assess, predict, and prevent, and what to do if you observe immunogenicity during preclinical, clinical, and post-market approval. The seminar begins by detailing the science behind immunogenicity, the latest international guidance, followed by assay and bioanalytical assessment strategies for traditional and emerging biologics. Other topics include predictive models and reporting immunogenicity.

Chloé Ackaert, PhD, Senior Scientist, Immunogenicity, ImmunXperts, a Q2 Solutions Company

Sofie Pattijn, Founder & CTO, ImmunXperts, a Q2 Solutions Company

Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting

TOPICS TO BE COVERED INCLUDE:

Part 1: Introduction to Immunology and Immunogenicity

What is immunogenicity?
Immunology and major mechanisms affecting immunogenicity
B cell development
Clinical consequences of ADA
Risk-based approachImmunogenicity in the clinic
Regulations and guidance governing immunogenicity

Part 2: Predictive Immunogenicity

Part 3: Clinical Considerations of Immunogenicity and Regulatory Expectations

Part 4: Assay Methodology and Approaches for Describing Immunogenicity in the Clinic

Assay methodologiesComparison of different methods
Screening, confirmation
Characterization of anti-drug-antibodies
Cut-points Immunogenicity of complex biologics (e.g. multi-domain, biosimilars)
PK/PD and safety and efficacy-- as a measure
Immunogenicity in the clinic – how to report

INSTRUCTOR BIOGRAPHIES:

Chloé Ackaert, PhD, Senior Scientist, Immunogenicity, ImmunXperts, a Q2 Solutions Company

Chloé Ackaert is a pharmacist by training (Catholic University of Leuven 2009) and obtained her PhD at the University of Salzburg (Austria) for the research on the impact of nitration on the immunogenicity of birch pollen allergens in 2013. She first joined ImmunXperts in the start-up phase and continued academic research at the Free University of Brussels (2015-2018) working on the immunogenicity of Nanobodies. Afterwards, she joined ImmunXperts again where she is a senior scientist in the immunogenicity team, collaborating both on the client-based projects as well as on the continuous basic research projects to elucidate immunogenicity-related questions.

Sofie Pattijn, Founder & CTO, ImmunXperts, a Q2 Solutions Company

Sofie Pattijn, CTO and founder of ImmunXperts, has over 20 years of experience in the field of immunogenicity assessment (vaccines and biotherapeutics) and in vitro assay development with a focus on functional assays for immunogenicity, immune oncology, and cell and gene therapy products. She has extensive hands-on lab experience and has managed and coached several in vitro teams over the last decade. From 2008 until 2013, she was Head of the in vitro Immunogenicity group at AlgoNomics (Ghent, Belgium) and Lonza Applied Protein Services (Cambridge, UK). Prior to that, she worked at Innogenetics in Belgium for over 15 years.

Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting

Bonita Rup is a biopharmaceutical development consultant, providing expert advice on bioanalysis, immunogenicity risk assessment, and related regulatory strategy aspects of biopharmaceutical development. Previously she was Research Fellow and lead for the Immunogenicity Discipline at Pfizer, Assistant Vice President of Protein Bioanalytics in Wyeth, and held various positions directing development and application of immuno-ligand binding assay technologies for PK, immunogenicity and protein impurity analysis, and other aspects of biopharmaceutical development. During her career, she has been involved in multiple regulatory filings during preclinical, clinical development and marketing approval of biopharmaceutical products. She has been a member of AAPS, EIP, European IMI ABIRISK consortium, and Biosafe; with these organizations, she has been a co-author for multiple publications related to monitoring immunogenicity and bioanalysis of therapeutic proteins. Bonnie received her B.S. from University of Massachusetts, Amherst, Ph.D. from University of Texas, Austin, and conducted postdoctoral research at Duke University and University of Rochester, NY.

TS9A: Introduction to Protein Engineering

Detailed Agenda

This course presents a comprehensive tutorial in the concepts, strategies, and latest tools of protein engineering applied to biotherapeutic research and development, particularly antibody-related products. The class is for scientists new to industry or working in support roles, academics, and protein scientists wanting a detailed update on the current state of the field.

David Bramhill, PhD, Founder, Bramhill Biological Consulting LLC

Course Outline:

What is protein engineering?
Tools and techniques
Engineering-by-design
Designed libraries, display technologies
Production and manufacturing
Improving manufacturing by protein engineering methods
Other protein modifications
Expression of antibodies and fragments for discovery and testing
Emerging molecule and product formats
Antibody-drug conjugates (ADCs)
Other emerging approaches

INSTRUCTOR BIOGRAPHIES:

David Bramhill, PhD, Founder, Bramhill Biological Consulting LLC

Dr. Bramhill has over 20 years’ experience in biologics, both in large biopharma and startup biotech companies. He has expertise in isolating and improving antibodies using phage display and other display systems and is an inventor on library design for small scaffolds and bi-specific formats. He also has experience in diverse expression systems for producing antibodies, antibody fragments and scaffolds. Additionally, Dr. Bramhill has extensive experience in ADC development including diverse chemical and biochemical conjugation methods. He has taught numerous technical courses for over 15 years at international conferences and served as a Key Opinion Leader for major BioPharma companies.

TS10A: Antibody Drug Discovery: From Target to Lead

Detailed Agenda

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 and development.

Zhiqiang An, PhD, Professor, Robert A. Welch Distinguished University Chair in Chemistry; Director, Texas Therapeutics Institute; Director, CPRIT Core for Antibody Drug Discovery; Vice President, Drug Discovery, University of Texas Health Science Center at Houston

Topics to be covered include:

Different Sources of Antibodies

- Animals: mouse, rat, rabbit, chicken, llama, etc.

- Libraries: immune, synthetic, native, fully human, etc.

- B cells: memory B cells, plasma B cells, human, and animals

Antibody-Based Drug Modalities

- IgGs, IgA, IgM, Bites, nanobody, antibody fragments, etc.

- Naked antibody-ADC

- Bispecific/multispecific

- CAR T

Antibody Engineering

- Affinity maturation

- Humanization

- Fc-engineering: half-life, immune effector function, etc.

Target Selection and Validation

Antibodies Targeting Complex Membrane Proteins

- GPCRs

- Ion channels

- Transporters and membrane-bound enzymes

Delivery of Antibodies

Crossing the Brain-Blood Barrier (BBB)

Case Studies

INSTRUCTOR BIOGRAPHIES:

Dr. Zhiqiang An is Professor of Molecular Medicine, the Robert A. Welch Distinguished University Chair in Chemistry, Director of the Texas Therapeutics Institute, and Vice President of Drug Discovery at the University of Texas Health Science Center at Houston. His laboratory focuses on antibody drug resistance mechanisms, biomarkers for therapeutic antibodies, and antibody drug discovery targeting human diseases. During the last five years, more than 12 novel antibody drug leads discovered in his laboratory were licensed to eight biotechnology companies, and six (6) have advanced to clinical trials for diseases ranging from acute myeloid leukemia (IO-202), breast cancer bone metastasis (ALMB-0168), solid tumor (IO-108), spinal cord injury (ALMB-0166), COVID-19 (IGM6268), and solid tumor (PRTH-101). Previously, he served as Chief Scientific Officer at Epitomics, Inc. and was Director of Biologics Research at Merck Research Laboratories. He has authored over 200 journal articles including more than 30 papers in Nature, Science, and Cell journal series; and two books including the award-winning “Therapeutic Monoclonal Antibodies: from Bench to Clinic.” He is an elected fellow of Society for Industrial Microbiology and Biotechnology (SIMB), the American Academy of Microbiology (ASM), American Association for the Advancement of Science (AAAS), and the National Academy of Inventors (NAI). Dr. An received his PhD from the University of Kentucky and his postdoctoral training at the University of Wisconsin-Madison.

Tuesday, May 14, 2024 3:00 - 6:10 pm | Wednesday, May 15, 2024 8:30 am - 6:10 pm

TS9B: Introduction to Machine Learning for Biologics Design

Detailed Agenda

This course offers an introduction to concepts, strategies, and machine learning methods used for biologics design. It includes presentations and demonstrations of the methods used in the field, covering techniques such as triaging sequences, modulating affinity, and designing antibody libraries, along with increasing manufacturability. The course is directed at scientists new to the field and protein engineers wanting an introduction to how machine learning can aid in guiding biologics design.

Christopher R. Corbeil, PhD, Research Officer, Human Health Therapeutics, National Research Council Canada

Francis Gaudreault, PhD, Research Officer, Human Health Therapeutics, National Research Council Canada

INSTRUCTOR BIOGRAPHIES:

Christopher R. Corbeil, PhD, Research Officer, Human Health Therapeutics, National Research Council Canada

Dr. Christopher Corbeil is a research officer at the National Research Council Canada (NRC) who specializes in the development and application of computational tools for biotherapeutic design and optimization. He is also an associate member of the McGill Biochemistry Department and teaches classes in Structure-Based Drug Design at McGill University. After receiving his PhD from McGill University, he joined the NRC as a Research Associate investigating the basics of protein-binding affinity. Following his time at the NRC he joined Chemical Computing Group as a research scientist developing tools for protein design, structure prediction, and binding affinity prediction. He then decided to leave private industry and rejoin NRC with a focus on antibody engineering. Dr. Corbeil has authored over 30 scientific articles and is the main developer of multiple software programs.

Francis Gaudreault, PhD, Research Officer, Human Health Therapeutics, National Research Council Canada

Francis obtained his PhD in Biochemistry from University of Sherbrooke in 2015, during which he developed a molecular docking program for docking small molecules to flexible protein or RNA targets. While doing his PhD studies, Francis co-founded a successful IT company for automating the management of scientific conferences. Francis joined the National Research Council (NRC) of Canada in 2016, where he has taken part in and led various efforts in the discovery and engineering of antibodies or other biologics. In such efforts are included the structure prediction of antibodies alone or in complex, the affinity assessment of antibody-antigen complexes, and the detection of antibody developability issues. Francis is leading the technical efforts in using artificial intelligence for antibody discovery.

TS10B: Introduction to Antibody-Drug Conjugate Design: Targets, Payloads, and Linkers

Detailed Agenda

In this training seminar, your instructors will take you on a journey through the history of ADC technology, the current status of the ADC field, and the most promising up-and-coming technologies that will shape the ADCs of tomorrow. We will place particular emphasis on design principles that can be applied to next generation ADC programs, whether in oncology applications or in a myriad of other therapeutic applications. We will introduce various assay strategies, experimental approaches, and technical insights that will enable participants to have both a practical and a theoretical understanding of the inner-workings of a successful ADC program. Your instructors are seasoned ADC experts that have been involved in numerous ADC programs in academia, in big pharma, and in biotechnology companies.

Robert J. Lutz, PhD, CSO, Iksuda Therapeutics

Nathan L. Tumey, PhD, Associate Professor, Pharmaceutical Sciences, SUNY Binghamton

Topic areas to be covered include:

An overview of the history of ADCs, emphasizing key developments and discoveries that shape the current therapeutic landscape
Principles and lessons in target selection
Antibody engineering: considerations and current trends
Conjugation and linker chemistry, past and present
Payload design and selection
Designing effective lead identification and screening strategies
Next generation ADC technology: What does the future hold?
ADCs beyond oncology

Who should attend?

Seasoned scientists who are moving into the ADC field
Early career scientists in the ADC field who want a solid grasp of the “big picture” of ADC
Executives and project managers who want a solid grasp of the challenges and opportunities of ADC technology

INSTRUCTOR BIOGRAPHIES:

Robert J. Lutz, PhD, CSO, Iksuda Therapeutics

Bob has been an independent consultant in the biotech/pharma industry since 2015 and has worked with multiple clients providing strategic, tactical and operational input for their research and development efforts. Prior to initiating his consulting practice, Bob held various R&D roles over a 23 year span at ImmunoGen, Inc. In his most recent position at ImmunoGen, Bob was Vice President of Translational Research and Development with responsibility for all early stage antibody drug conjugate (ADC) development programs from lead identification through phase 2. He also served as ImmunoGen’s research lead on Genentech’s FDA-approved trastuzumab emtansine (T-DM1) program. Before the ADC research position, Bob was part of ImmunoGen’s Apoptosis Technology Inc subsidiary where he led the research collaboration leading to the discovery of the BH3 domain of the BCl-2 family of death regulatory proteins. Before ImmunoGen, Bob was a research and postdoctoral fellow at the Eleanor Roosevelt Institute in Colorado. He earned his doctoral degree in Biochemistry at Brandeis University.

Nathan L. Tumey, PhD, Associate Professor, Pharmaceutical Sciences, SUNY Binghamton

L. Nathan Tumey is joined Binghamton University in 2017, following 15 years of medicinal chemistry experience in the pharmaceutical and biotechnology industry. His research focuses on the design of antibody-drug-conjugates (ADCs) for the treatment of cancer, auto-immune disorders, and rare diseases. Specific research projects include immune-stimulating antibody conjugates, glucocorticoid-antibody conjugates, legumain-cleavable linkers, and site-specific conjugation technology. He is a frequent consultant at various biotechnology and pharmaceutical companies, and serves on the scientific advisory board of multiple ADC-related organizations. Dr. Tumey received his PhD in chemistry from Duke University in 2001, under the supervision of Michael Pirrung. During his years in the pharmaceutical industry, Tumey was a key leader in multiple drug-discovery programs including the development of 5HT2c agonists for obesity, PKC-theta inhibitors for asthma, IRAK4 inhibitors for rheumatoid arthritis and antibody-drug-conjugates (ADCs) for the treatment of cancer.

TS11B: Label-Free Biosensor Tools in Biotherapeutic Discovery: SPR, BLI & KinExA

Detailed Agenda

This training seminar will cover the main applications of commonly used commercial label-free biosensors in the interaction analysis of biologics and guidelines for best practices to generate reliable and reproducible data. We will primarily focus on Surface Plasmon Resonance (SPR), Biolayer Interferometry (BLI), and Kinetic Exclusion Assay (KinExA) technologies and their application in drug discovery.

Yasmina Abdiche, PhD, Vice President, Exploratory Research, OmniAb Inc.

Vishal Kamat, PhD, Senior Director, Protein Sciences, Ampersand Biomedicines

Palaniswami (Swami) Rathanaswami, PhD, CEO, PRSwami AbDev Inc.

BINDING AFFINITY and KINETICS- Taught byVishal Kamat

Concept of real-time binding kinetic assays and binding kinetic parameters - ka, kd, KD
Working principle of SPR and BLI biosensors
Designing binding kinetic assays on SPR & BLI biosensors
Best practices to generate reliable binding kinetic data with minimal experimental artifacts on SPR & BLI biosensors

SOLUTION AFFINITY- Taught by Palaniswami (Swami) Rathanaswami

Introducing the solution affinity method and its need
KinExA method and best practices
Data analysis and interpretation
Advanced applications of KinExA

EPITOPE BINNING- Taught by Yasmina Abdiche

Assay formats
Bin definition
Throughput
Antigen heterogeneity
Asymmetric binnings
Antibody displacement

INSTRUCTOR BIOGRAPHIES:

Yasmina Abdiche, PhD, Vice President, Exploratory Research, OmniAb Inc.

Yasmina is an internationally recognized and innovative scientific leader in the field of antibody discovery and biosensors with twenty years of experience in protein engineering and biopharma. To date, she has authored over 45 peer-reviewed publications, had 25 patents granted in the therapeutic antibody space including a market-approved drug (Ajovy), presented over 50 times as an invited speaker globally, and has been involved in numerous industry-wide collaborations and scientific advisory groups. After a twelve-year career at Pfizer where she held positions of increasing responsibility from Principal Scientist to Research Fellow, more recently, Yasmina has held senior management roles at biotechnology companies and contract research organizations including Carterra (CSO), ImmunoPrecise Antibodies (CSO), ALX Oncology (Vice President Protein Science), Revelar (CSO and Co-Founder), and FairJourney Biologics (CTO). During her time as CSO at Carterra, she co-founded its antibody screening biosensor platform (the LSA) which has helped transform label-free interaction analysis in early-stage drug discovery. Yasmina has a Master's degree in Chemistry and a PhD in Biological Chemistry from Oxford University and did post-doctoral studies in biophysical interaction analysis at the University of Utah.

Vishal Kamat, PhD, Senior Director, Protein Sciences, Ampersand Biomedicines

Vishal (Vish) Kamat is an innovative and collaborative leader with 17 years of experience in the field of biotherapeutics and biophysical characterization of antibodies and proteins. He has supported the discovery and development of antibodies for 150+ targets which yielded 20 clinical drug candidates and 3 market-approved drugs - DUPIXENT, LIBTAYO, and EVKEEZA. He also has extensive experience in high throughput screening of antibodies using diverse platforms such as SPR, BLI, KinExA, Gyrolab, MSD and Luminex. His scientific excellence is evident by multiple peer-reviewed publications in high impact journals, patent approvals and IND reports. After a 12-year career at Regeneron Pharmaceuticals where he held positions of increasing responsibility from postdoctoral candidate to Sr. Staff Scientist, he worked as a Director of Antibody Characterization at Twist Biopharma and is currently working as a Senior Director of Protein Sciences at Ampersand Biomedicine. Vishal holds a PhD in Biomedical Engineering from Drexel University and BS in Electronics Engineering from University of Mumbai.

Palaniswami (Swami) Rathanaswami, PhD, CEO, PRSwami AbDev Inc.

World’s leading expert Scientist in Antibody affinity and kinetic characterization, especially for measuring femto molar affinities of antibodies for soluble and on-cell targets. World expert in solution binding measurements and using KinExA technology. Extensive user of SPR and BLI technologies. Experienced SME as Functional Lead for human therapeutic antibody programs and drug product development. Dedicated career to advancing medical research to alleviate disease and make individuals lives better. Consultant for leading biotech organizations on high throughput screening, affinity measurements and antibody drug product development. Did extensive research, over 40 years in the fields of Endocrine Biochemistry, Molecular Endocrinology, Inflammation Immunology-Rheumatology, Human therapeutic antibody generation, engineering and characterization (including immunization, high throughput binding and functional screening and very high affinity kinetic measurements). A key member of a team of 5 Scientists who developed SLAM technology for human therapeutic antibody generation and spun to a Canadian Biotech company – Immgenics Inc., and later acquired by Amgen. First Scientist to generate human antibodies by Single Cell RT-PCR amplification of V genes from antibody producing single B Cells, molecular cloning and express as full antibody. Published over 40 articles in very high impact Scientific Journals and inventor of over 20 scientific patents. Worked for Amgen 25+ years and generated over 100 human therapeutic antibodies of which about 5 are already used as pharmaceutical drugs in market. Invited speaker in national and international conferences, Universities and Research institutes across the globe. Chaired sessions in international conferences and seminars. Dr. Palaniswami Rathanaswami graduated in MSc (Biochemistry, Faculty of Medicine) and a PhD (Biochemistry), University of Madras, India.

Thursday, May 16, 2024 8:45 am - 6:00 pm | Friday, May 17, 2024 8:30 am - 12:30 pm

TS7C: Introduction to Bioassay Design, Development, Analysis, Validation, and Monitoring

Detailed Agenda

This course introduces statistical ideas supporting bioassays (via examples), reviews bioassay properties, and shows how laboratory constraints create the ‘statistical design structure’ of assays. These structures inform analyses and design of experiments (DOE) and its application to development, validation, and monitoring. Strategic combinations of assay design and good assay analysis methods offer new monitoring tools that support a lifecycle approach.

David Lansky, PhD, President, Precision Bioassay, Inc.

Statistical concepts
Biological assay: fundamentals, types, properties, and similarity
Intro to ‘classical’ design of experiments: factorials, response surface designs, blocking
Using DOE with bioassays
Bioassay analysis: addressing violations of assumptions, modeling, why mixed models, similarity
Considerations when setting product specifications
Setting assay and sample acceptance limits in bioassays
Modular assay designs ease development, robustness, qualification, and validation
Qualification and validation design and analysis
Assay monitoring
Managing changes in assays
Assay transfers

INSTRUCTOR BIOGRAPHIES:

David Lansky, PhD, President, Precision Bioassay, Inc.

David has been practicing statistics on bioassays (and other non-clinical applications in Pharma) for 35 years. This includes Searle/Monsanto/Pharmacia (10 years) and as the owner of Precision Bioassay, Inc. (since 2002). Most of his bioassay experience involves helping teams improve and validate cell-based bioassays. His experience includes some early (late 1990’s) success as part of a team that used lab automation for a series of cell-based bioassays. He has been and is still an active participant in the work to revise the USP bioassay chapters. His education includes a year of Electrical and Computer Engineering (University of Michigan), a BS in Botany (San Francisco State), an MS in Entomology (Cornell) and finally both an MS and Ph.D. in Biometry (both Cornell).

TS8C: Bridging the Gap from R&D to Bioprocessing

Detailed Agenda

Carissa L. Young, PhD, Independent Consultant

Marieke Koedood Zhao, PhD, Vice President, Process Sciences, Kudo Biotechnology

Do you seek to better understand end-to-end operations in drug development, gain clarity of biotech-pharma functions and cross-functional teams, phase-appropriate analytics, and improve quantitative Go/No Go decisions in order to accelerate therapies to patients?

This 1.5 day course will focus on Discovery & Drug development processes and operations — providing an overview of the drug pipeline and key milestones towards IND filings, detailed assessments of cross-functional strategies, and in-depth learnings in R&D spanning therapeutic candidate selection, developability assessments, risks & mitigations, and analytical & process development considerations for regulatory submissions.

This interactive course is designed for scientists and engineers in Discovery, NonClinical Development, and CMC, with a desire to increase the probability of success of therapeutic candidates across multiple modalities (biologics, small molecules, cell and gene therapies). Instructors will share best practices, key pitfalls, translational guidelines, data-driven strategies, and regulatory considerations, through case studies, course materials, and supplemental information.

Topics to be covered:

Drug pipeline overview and key milestones towards IND filings
Key Elements of therapeutic candidate selection
Developability assessments
Analytical and process development considerations for regulatory submissions
Risks and mitigations
Cross-functional strategies and integrated timelines

Who should attend: Scientists and engineers in Discovery, NonClinical Development, and CMC, with a desire to facilitate and accelerate successful development of clinical therapeutic candidates across multiple modalities (biologics, small molecules, cell and gene therapies).

INSTRUCTOR BIOGRAPHIES:

Carissa L. Young, PhD, Independent Consultant

Carissa has 18+ years in pharma-biotech spanning nonclinical, CMC, and clinical development across multiple modalities (cell therapies, biologics, small molecules) and disease indications. She has built and managed cross-functional teams in Drug Development and Data Sciences to maximize the value of therapeutic pipelines, portfolio, and platforms. Her versatility enables her to engage across the diversity of translational sciences, process-product development life cycle, clinical/nonclinical study designs to regulatory filings, data quality oversight and compliance. Previous industry experience includes Intellia Therapeutics, Takeda, Applied Biomath, and Johnson & Johnson. Teaching experience as Instructor at Harvard Medical School and the University of Delaware. Carissa received her Chemical Engineer degrees at Georgia Tech (BS) and the University of Delaware (PhD), completing her post-doctoral training in Biological Engineering at MIT.

Marieke Koedood Zhao, PhD, Vice President, Process Sciences, Kudo Biotechnology

Marieke has 20+ years experience in bio-therapeutic development, facilitating collaboration of research, preclinical, CMC, and clinical development teams and had a track record of ushering novel Biologics from pre-development to first-in-human clinical trials with aggressive timelines. She has hands-on experience in drug substance process development, technology transfer, process scale-up, selection and management of Contract Manufacturing Organizations (CMOs), CMC strategy, and regulatory filings for antibody-drug conjugates and mRNA products entering the clinic. Previous industry experience includes GreenLight Biosciences, Mersana Therapeutics, ImmunoGen and Wyeth Biopharma. Marieke received her PhD from the University of Zurich in Switzerland and completed post-doctoral training at Boston University.

TS9C: Analysis and Interpretation of Antibody Deep Sequencing and Single Cell Analysis Data

Detailed Agenda

In this training seminar, participants will learn about recently developed methods for Next-Generation Sequencing (NGS) and single-cell analysis of antibody repertoires. The course will be interactive with case studies, participants will be able to download data and examples. Please bring your computer.

Brandon DeKosky, PhD, Phillip and Susan Ragon Career Development Professor of Chemical Engineering, MIT Core Member, The Ragon Institute of MGH, MIT, and Harvard University

Matias Gutierrez-Gonzalez, PhD, Research Fellow, The Ragon Institute of MGH, MIT, and Harvard

PART I:

Introduction to NGS of antibody repertoires and bioinformatics tools

Introduction to antibody repertoire analysis

Experimental design and considerations

Data analysis overview

Hands-on session: introduction of common computational tools for NGS

PART II:

Preprocessing and analysis of antibody repertoire

NGS data hands-on session: preprocessing data hands-on session

Antibody immune repertoire analysis

Advanced methods for NGS data analysis

PART III:

NGS sequencing of single B cell populations

NGS for analysis of library screening results

Single-B cell functional analyses via NGS

WHO SHOULD ATTEND: This course is designed for individuals in industry and/or academia who are establishing or learning high-throughput NGS-based technologies for antibody repertoire analysis and antibody discovery. Prior knowledge in the areas of antibody biology and command-line programming is very helpful, but not required. By the end of the workshop, course participants should have the ability to understand and evaluate experimental options for antibody NGS data acquisition, perform analysis and visualization of antibody sequencing data, identify related antibody variants from NGS information, and possess a general understanding of the current scope of NGS-enabled antibody discovery techniques.

INSTRUCTOR BIOGRAPHIES:

Brandon DeKosky, PhD, Phillip and Susan Ragon Career Development Professor of Chemical Engineering, MIT Core Member, The Ragon Institute of MGH, MIT, and Harvard University

Dr. Brandon DeKosky is an Assistant Professor in the Department of Chemical Engineering at MIT and a Core Member of the Ragon Institute of MGH, Harvard, and MIT. Research efforts at the DeKosky lab have developed a suite of high-throughput single-cell platforms for comprehensive analyses of adaptive immunity. These efforts are advancing new approaches in biologic drug discovery, and for the comprehensive analyses of genetic and functional diversity in adaptive immune cells. The group seeks to reveal the quantitative principles that govern effective adaptive immunity and provide molecular design strategies for vaccines and biologics to combat global infectious agents including HIV-1, malaria, and SARS-CoV-2. The DeKosky lab is also investigating quantitative principles of immune regulation and establishing new approaches for targeted and personalized cancer therapies. Dr. DeKosky has been awarded several honors for his research program. His PhD research was supported by a Hertz Foundation Graduate Fellowship, an NSF Graduate Fellowship, and a Donald. D. Harrington Graduate Fellowship. In 2016, DeKosky was awarded a K99 Pathway to Independence Award and an NIH Early Independence Award and began a joint faculty appointment at the University of Kansas Departments of Chemical Engineering and Pharmaceutical Chemistry. He has also received the Department of Defense Career Development Award, the Biomedical Engineering Society Rising Star Award, and the AIChE Young Faculty Futures award. In 2021, Dr. DeKosky began as an Assistant Professor in a joint appointment at MIT Chemical Engineering and The Ragon Institute.

Matias Gutierrez-Gonzalez, PhD, Research Fellow, The Ragon Institute of MGH, MIT, and Harvard

Dr. Matias Gutierrez is a Postdoctoral Researcher in the lab of Dr. Brandon DeKosky at the University of Kansas. Dr. Gutierrez’s graduate research studied the development and characterization of biopharmaceuticals, with a focus on monoclonal antibodies. Currently, Dr. Gutierrez works to develop new tools for bioinformatic analysis of single-cell antibody repertoire data, and to establish new techniques for high-throughput B cell sequencing.