- 표적 식별과 타깃 밸리데이션 전략 -

표적 식별과 타깃 밸리데이션 전략을 테마로 한 이 컨퍼런스 프로그램에서는 새로운 Drug Discovery 표적의 발견, 새로운 적응을 위한 기존 표적의 밸리데이션, 표적의 저해 및 활성화가 세포 경로에 미치는 영향에 대한 이해 촉진 등을 도모하기 위해 기능 게노믹스 및 표현형 스크리닝, 화학생물학을 활용하는 방법에 초점을 맞춥니다.

Final Agenda

Recommended All Access Package:

11월 27일: Drug Discovery를 위한 인공지능과 기계학습

11월 27일 Dinner Course: 쇼트코스 3:장기 칩 시스템의 기점, 최적화, 응용

11월 28-29일: 표적 식별과 타깃 밸리데이션 전략

11월 29-30일: 중추신경계 모델과 중개연구 전략

11월 29일 Dinner Course: 쇼트코스 5:인간화 마우스 모델:기술 개요와 암 면역치료의 전임상 평가에 대한 응용

11월 28일(수)

7:00 Registration and Morning Coffee

새로운 표적의 발견을 위한 CRISPR, RNA 간섭, 각종 기능 게노믹스 스크리닝의 활용

8:20 Welcome Remarks

Tanuja Koppal, PhD, Conference Director, Cambridge Healthtech Institute

8:25 Chairperson’s Remarks

John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

8:30 Up, Down, and Out: Multiplexing CRISPR Modalities for Genetic Screens

John Doench, PhD, Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

The ease of programming Cas9 with an sgRNA presents an abundance of potential target sites, but the on-target activity and off-target effects of individual sgRNAs can vary. We will discuss the design and use of libraries for CRISPR-based knockout screens, activation (CRISPRa) and interference (CRISPRi) technologies, and combinatorial approaches.

9:00 Development of New CRISPR/Cas9-based Tools to Study Drug Interactions Through Knockout and Directed Evolution

Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University

9:30 Sponsored Presentation (Opportunity Available)

10:00 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Geometry and Genome editing: A Comparative Analysis of Targeted siRNA and CRISPR Arrayed Screens in 3D

Madhu Lal-Nag, PhD, Group Leader, Trans-NIH RNAi Facility, National Center for Advancing Translational Sciences, National Institutes of Health

11:15 PANEL DISCUSSION: Exploiting the Complementarity of Different Genomic Screening Platforms - Best Practices

Panelists:

Michael Bassik, PhD, Assistant Professor, Department of Genetics, Stanford University

John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

Madhu Lal-Nag, PhD, Group Leader, Trans-NIH RNAi Facility, National Center for Advancing Translational Sciences, National Institutes of Health

11:45 Sponsored Presentation (Opportunity Available)

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:45 Session Break

표현형 스크리닝용 각종 모델, 어세이, 플랫폼 연구

13:45 Chairperson’s Remarks

Ulrich Elling, PhD, Principle Investigator, Institute of Molecular Biotechnology Austria (IMBA)

13:50 CRISPR-UMI: Single Cell Lineage Tracing of Pooled CRISPR/Cas9 Screens

Ulrich Elling, PhD, Principle Investigator, Institute of Molecular Biotechnology Austria (IMBA)

Pooled CRISPR screens are a powerful tool to assess gene function. We developed CRISPR-UMI (Unique Molecular Identifiers), a single cell lineage tracing methodology for pooled screening to account for cell heterogeneity. The added value of CRISPR-UMI will be presented for positive and negative selection paradigms including synthetic lethal screens. CRISPR-UMI controls for inherent noise in genetic screens, increases reproducibility, and enables quantitative phenotyping.

14:20 Unbiased Compound-Target Interface Mapping through Forward Genetics

Moritz Horn, PhD, Postdoctoral Fellow, Laboratory of Dr. Martin Denzel, Max Planck Institute for Biology of Ageing

We present a chemical mutagenesis approach to map compound binding interfaces in forward genetic screens and exemplify this for several compounds. We leverage haploid embryonic stem cells as genetic tool to reveal loss-of-function, gain-of-function, and separation-of-function mutations, since chemical mutagenesis in haploid cells can uncover recessive traits. In summary, chemical mutagenesis combined with deep sequencing allows to identify drug targets at amino acid resolution in an unbiased genome-wide scale.

14:50 Interactive Breakout Discussion Groups

This session features various discussion groups that are led by a moderator/s who ensures focused conversations around the key issues listed. Attendees choose to join a specific group and the small, informal setting facilitates sharing of ideas and active networking. Details on the topics and moderators are available on the conference website.

16:00 Refreshment Break in the Exhibit Hall with Poster Viewing

16:45 A High Density CRISPR Tiling Mutagenesis Genetic Screen for Target Deconvolution

Dirk Daelemans, PhD, Associate Professor, Rega Institute - Laboratory of Virology and Chemotherapy, KU Leuven

Deconvoluting the direct molecular target of hits from a complex phentotypic high-throughput screen or validating the target of hits from a biochemical screen in a more complex system is still a major challenge but required for further drug development. The discovery of mutations that confer resistance is recognized as the gold standard proof for target engagement. Exploiting the error prone non-homologous end joining after CRISPR-induced double strand breaks, we have developed a high-density tiling CRISPR genetic screen to rapidly deconvolute the target protein and binding site of small-molecule inhibitors based on resistance mutations. The CRISPR guides directly annotate the resistance mutations avoiding the need for whole transcriptome sequencing.

17:15 Target Deconvolution and Validation Strategies for Antibodies from Target-Agnostic Phenotypic Screens

Alan Sandercock, PhD, Scientist II, Department of Antibody Discovery and Protein Engineering, MedImmune

17:45 Concurrent Toxicity and Efficacy Evaluations of Lead Compounds Utilizing Multi-organ Functional Human-on-a-chip Systems

James J. Hickman, PhD, Founding Director, NanoScience Technology Center and Professor, Nanoscience Technology, Chemistry, Biomolecular Science, Material Science and Electrical Engineering, University of Central Florida

Phenotypic screening systems for lead optimization have the advantage of providing useful information without a known target. Human-on-chip systems can potentially establish a therapeutic index before evaluation in animal models or clinical trials and also gives the possibility of target identification. Examples of multi-organ systems being developed for cancer and other diseases and conditions as well as the results of six Workshops held at NIH for validation and qualification will be presented.

18:15 Welcome Reception in the Exhibit Hall with Poster Viewing

19:15 Close of Day

11월 29일(목)

8:00 Registration and Morning Coffee

새로운 화학생물학과 케모게노믹 스크리닝 플랫폼

8:25 Chairperson’s Remarks

Paul Brennan, PhD, Associate Professor, Medicinal Chemistry, University of Oxford; Principal Investigator, Target Discovery Institute, Structural Genomics Consortium


8:30 KEYNOTE PRESENTATION: Chemical Probes in Target Discovery

Paul Brennan, PhD, Associate Professor, Medicinal Chemistry, University of Oxford; Principal Investigator, Target Discovery Institute, Structural Genomics Consortium

Chemical probes are selective small molecule inhibitors that can be used in cellular assays to induce a phenotype and link it to a small set of protein targets. Chemical probes have been developed for many bromodomains, the principal epigenetic readers of histone lysine acetylation, and been used to decipher the biology of bromodomains in cancer and inflammation. We are currently developing chemical probes for new protein families.

9:30 Sponsored Presentation (Opportunity Available)

10:00 Coffee Break in the Exhibit Hall. Last chance for poster viewing.

10:45 Large-Scale Chemogenomics Data: An Enabler for Early Drug Discovery

Florian Nigsch, PhD, Chemical Biology and Therapeutics Informatics, Novartis Institutes for BioMedical Research

This talk will showcase the impact of the integration of large-scale chemogenomics data on a range of activities in early drug discovery. Topics covered will include pre- and post-screening activities, tool compound discovery, chemoproteomics, as well as applications that link compounds and their transcriptional profiles.

11:15 Chemo-Genomic Screening in AML: A New Approach to Identify Therapeutic Strategies in Cancer

Anne Marinier, PhD, Principal Investigator and Director of Medicinal Chemistry, IRIC and Associate Professor, Department of Chemistry, Université de Montréal

Capitalizing on new leukemia stem cell culture conditions, we developed a chemo-genomic screening approach using genetically and clinically characterized acute myeloid leukemia (AML) specimens and a structurally diverse compound collection. Clustering of hits demonstrating similar specimen inhibition patterns generated CCCs (Compound Correlation Clusters) which reveal sensitized target pathways essential to tumor survival. The CCCs therapeutic relevance will be exemplified by the identification of a novel target for poor prognosis AML.

11:45 A High-Throughput Imaging-Genetic Screen Identifies MEK-PI3K Modulation for TNBC

Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan

As combination therapies enter mainstream clinical oncology, there is now a need for infrastructure to integrate multiple modalities of data to prioritize drug combinations rationally. In this vein, we examine a scenario using machine learning methods to prioritize drug combinations selected based on phenotypic screening via RNAi, coupled with known genetic vulnerabilities in Triple Negative Breast Cancer cells. Such a strategy leverages imaging and genetic information to prioritize the MEKi+PI3Ki combination as a possible regimen.

12:15 Brief Session Break

12:20 Bridging Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:50 Close of Conference

* 주최측 사정에 따라 사전 예고없이 프로그램이 변경될 수 있습니다.

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