Cambridge Healthtech Institute (CHI) 培训讲座除了将进行实际案例研讨、介绍目前所面临课题时的对策之外，并网罗了作为各项议题根基的基础知识。将由经验丰富的专家主持，透过讲义、互动讨论、作业等内容，可吸收多方面知识。透过培训讲座，您可以聆听制药产业各个面向深入解说，对于企业及学术研究团体中刚入足新领域的研究人员、协助人员而言十分有助益。
Day 1: 9:00 am - 5:00 pm | Day 2: 9:00 am - 12:00 pm
TS1A: Introductory Immunology for Autoimmune and Cancer Drug Discovery
This 1.5-day training seminar will provide an introduction to human immunology for discovery pharmacologists, biologists and chemists working in the biopharmaceutical industry on inflammation, autoimmune, or immune-oncology programs. This training seminar will focus on how the immune system is organized and gives rise to both normal and pathogenic immune responses. This overview will also serve as a basis for discussions of how the immune system can be modulated through biopharmaceutical intervention to either suppress pathogenic inflammation or enhance anti-tumor immunity.
Tim Bauler, PhD, Assistant Professor, Department of Biomedical Sciences, Western Michigan University Homer Stryker M.D. School of Medicine
Tom Sundberg, PhD, Senior Research Scientist I, Center for the Development of Therapeutics, Broad Institute of MIT and Harvard
TS2A: Applying Pharmacology to New Drug Discovery: The System-Independent Quantification of Molecular Drug Properties for Prediction of Therapeutic Utility
Over the past six years, the primary cause of new drug candidate failures (50%) has been failure of therapeutic efficacy. Put another way, drug discovery programs do everything right, get the defined candidate molecule, only to have it fail in therapeutic trials. Among the most prevalent reasons proposed for this shortcoming is the lack of translation of in vitro and recombinant drug activity to therapeutic in vivo whole systems. Drug activity in complete systems can be characterized with the application of pharmacological principles which translate drug behaviors in various organs with molecular scales of affinity and efficacy.
Pharmacological techniques are unique in that they can convert descriptive data (what we see, potency, activity in a given system) to predictive data (molecular scales of activity that can be used to predict activity in all systems including the therapeutic one, i.e. affinity, efficacy). The predicted outcome of this process is a far lower failure rate as molecules are progressed toward clinical testing.
This course will describe pharmacological principles and procedures to quantify affinity, efficacy, biased signaling and allostery to better screen for new drugs and characterize drug candidates in lead optimization assays. In particular, new concepts that have entered the fabric of discovery over the past few years, namely biased signaling and allosteric drug function, will be emphasized as new ways forward to reduce new candidate attrition in the drug discovery process.
Terry Kenakin, PhD, Professor, Department of Pharmacology, University of North Carolina School of Medicine
TS3A: Genome Editing with Targeted Nucleases: Theory and Practice of CRISPR/Cas9 Technology Applications from Basic Research to Precision Therapies
This rigorous day and a half program is ideal for discovery and translational scientists who are looking for a balanced knowledge of gene editing applications in drug discovery and development. Taught by experts in the CRISPR/Cas9 field, this program was designed as a succinct introduction into the technology "nuts and bolts". Beginning from describing the basic aspects, key molecular features, strengths and shortcomings of CRISPR/Cas9 technology, the workshop instructors will advance towards sharing in-depth knowledge touching upon all facets of state-of-art genome editing applications, e.g. constructing of experimental cell culture based systems, engineering disease in vivo models supporting preclinical drug development workflows, rational design and functional screening of sgRNA libraries, and many others. Instructors will furthermore strive to achieve a balance between presenting theory information and conducting hands-on exercises in exploring available digital frameworks for designing and resourcing CRISPR/Cas9 studies, as well as troubleshooting complex experimental scenarios and conducting Q&A sessions.
Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader PTO, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)
Danilo Maddalo, PhD, Lab Head, ONC Pharmacology, Novartis Institutes for BioMedical Research, Novartis Pharma AG
Day 1: 2:30 - 5:30 pm | Day 2: 8:00 am - 4:00 pm
TS4B: Introduction to Small Molecule Drug Discovery and Development
This 1.5-day lecture-based interactive seminar focuses on strategies for identifying drug discovery targets, discovering and characterizing small molecule hits, and developing structure-activity relationships to advance hits through lead optimization, preclinical development, and clinical evaluation. Participants will learn the stages and processes required to advance programs from idea to clinic, through examples and case studies. This seminar is intended for scientists in either academia or industry who would like to become more familiar with small molecule drug discovery and development.
H. James Harwood Jr., PhD, Founder and CEO, Delphi BioMedical Consultants, LLC
TS5B: Practical Introduction to PKPD Modeling in Drug Discovery and Development: Better, Faster, Cheaper
Model-based drug development (MBDD) has evolved into a set of practical techniques that dramatically reduce pharma R&D costs without sacrificing quality. Pharmacokinetic/Pharmacodynamic (PKPD) modeling lies at the heart of MBDD. Part 1 of the seminar will teach the concepts of MBDD and its application in drug R&D. Part 2 will provide an in-depth introduction to key concepts in PKPD modeling, and teach hands-on implementation of PKPD modeling on real and simulated datasets.
Arijit Chakravarty, CEO, Fractal Therapeutics
Ryan Nolan, PhD, Research Fellow, Takeda Pharmaceuticals International Co.
TS6B: Patient-Derived ASC-Organoids for Drug Development and Screening, Patient Stratification and Regenerative Medicine
Recently, a number of technological advances in stem cell biology have prompted the development of better in vitro models for biological systems. The seminar will describe the scientific breakthroughs from the lab of Hans Clevers that lead to the development of the organoid technology based on adult stem cell (ASC)-derived human epithelial 'mini-organs'. The seminar will describe in detail how organoid models generated from tissues of patients with various diseases including cancer and cystic fibrosis can be used for drug development or patient stratification. These culture models stably maintain the genotype/phenotype of the patient's diseased tissue, thereby representing an in vitro platform for preclinical drug discovery and validation and a tool for precision medicine. Some attention will also be given to the impact that this technology could have in the area of regenerative medicine.
Sylvia F. Boj, PhD, Scientific Director, Hubrecht Organoid Technology
Additional Instructor to be Announced