Original Agenda
We are actively working with our speakers to confirm their availability for the virtual event. Initial response from our speakers has been very positive, and we are optimistic we will have the new programs ready to share here soon.

Cambridge Healthtech Institute’s 17th Annual

Target Identification and Validation - Part 2

Genomics-Based Target Discovery

September 17 - 18, 2020

Finding novel, druggable targets for therapeutic intervention remains a top priority for the pharma/biotech industry. It also remains a formidable challenge and companies continue to invest a lot of time and resources in identifying and validating targets that will yield viable drugs. The second part of the Target Identification and Validation conference focuses on Genomics-Based Target Discovery. It includes applications of RNAi, CRISPR, chemical genetics, single-cell sequencing, and other genomics-based tools to identify and validate new cellular pathways and drug targets. Complementary use of these technologies integrated with emerging data analysis and AI/ML approaches will also be discussed.

Thursday, September 17

11:20 am Conference Registration for Part B Programs


12:20 pm Event Chairperson's Opening Remarks
An-Dinh Nguyen, Team Lead, Discovery on Target 2020, Cambridge Healthtech Institute
12:30 Plenary Keynote Introduction (Sponsorship Opportunity Available)

Tackling Undruggable Oncoproteins: Lessons from the VHL Tumor Suppressor Protein

William G. Kaelin, Jr., MD, Professor and Investigator, Howard Hughes Medical Institute, Oncology, Dana-Farber Cancer Institute

VHL tumor suppressor protein (pVHL) inactivation is common in kidney cancer and upregulates the HIF2 transcription factor. PT2977/MK-6482 is an allosteric HIF2 inhibitor now in Phase 3 testing. Thalidomide-like drugs (IMiDs) bind to cereblon which, like pVHL, is the substrate-binding unit of a ubiquitin ligase. IMiDs redirect cereblon to destroy the myeloma oncoproteins, IKZF1 and IKZF3. We have developed new assays for identifying drugs that can destabilize oncoproteins of interest.


De-Risking Early Drug Discovery

Panel Moderator:
Nadeem Sarwar, PhD, Founder & President, Eisai Center for Genetics Guided Dementia Discovery, Eisai, Inc.
  • Data Sciences
  • ​Novel Chemical Modalities
  • Investment and Partnering Models
  • COVID-19 Progress as Examples of Successful Partnerships
Anthony A. Philippakis, PhD, Chief Data Officer, Data Sciences & Data Engineering, Broad Institute; Venture Partner, GV
Andrew Plump, MD, PhD, President, Research & Development, Takeda Pharmaceuticals, Inc.
2:00 Close of Plenary Keynote Program
2:00 Dessert Break in the Exhibit Hall with Poster Viewing


2:45 Organizer's Welcome Remarks

Cambridge Healthtech Institute

2:50 Chairperson's Remarks

Narender Gavva, PhD, Director, Early Target Discovery, Takeda California, Inc.


Where Are We with Human Genetics-Based Drug Discovery?

Narender Gavva, PhD, Director, Early Target Discovery, Takeda California, Inc.

Human genetics supported targets are sought after for higher success in the clinic during the last decade. While population genetics studies are revealing disease associated locations in the genome, biobank genotyping, whole exome, and whole genome sequencing efforts are in full swing to find rare causal and protective variants that would become drug discovery targets. This presentation willcover the status of current efforts and future directions.


Applications, Challenges and Opportunities of Functional Genomics in Drug Discovery

Lauren Drowley, PhD, Head, Functional Genomics, UCB Pharma
3:55 Sponsored Presentation (Opportunity Available)
4:25 Refreshment Break in the Exhibit Hall with Poster Viewing

Translating the Genetic Vulnerabilities of Cancer into Therapeutics: Lessons from the Cancer Dependency Map Project

Brenton Paolella, PhD, Research Scientist, Cancer Dependency Map, Broad Institute of Harvard and MIT and Dana-Farber Cancer Institute

Recent technological advances have enabled the systematic identification of genes that cancer cells rely on for their survival. However, translating these biological insights into new cancer therapeutic targets remains challenging. To facilitate cancer target discovery and advancement, we have developed computational pipelines to prioritize targets from the Cancer Dependency Map datasets and also cell-based assays to validate the most promising gene dependencies towards drug discovery to accelerate precision cancer medicine.


Small Molecule Target Identification Using CRISPR-Cas9 Functional Screens

Susanne E. Swalley, PhD, Principal Scientist, Chemical Biology & Proteomics, Biogen

While phenotypic approaches allow for the discovery of new targets and mechanisms, target deconvolution represents a key challenge due to the intrinsic difficulty, time, and expense. Compound-sensitized genome-wide screens using CRISPR/Cas9 are a powerful tool to aid in target identification. We will describe the development of an arrayed screening platform, and its application to generation of target hypotheses for a small molecule that enhances antisense oligonucleotide (ASO) uptake.


End-to-End Target Discovery Capabilities in AstraZeneca

Davide Gianni, PhD, Associate Director, Functional Genomics, AstraZeneca

Identification and validation of the “right” target is critical to reduce clinical attrition and to make drug discovery more sustainable. We have invested in the establishment of “end to end” target discovery capability to enable the identification of novel drug targets with better attrition rates, building on advancements in genome editing, translatable models and computational power. I will describe a couple of case studies that epitomise this capability.  

6:30 Dinner Short Course Registration (Premium Package or separate registration required)
7:00 Dinner Short Courses 10-12 (see Short Courses page for details)
9:30 Close of Day

Friday, September 18

7:00 am Registration
7:30 Interactive Breakfast Breakout Discussion Groups
Grab a cup of coffee and join a breakout discussion group. These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic. Visit the conference website for discussion topics and moderators.
8:30 Transition to Sessions


8:40 Chairperson's Remarks

John Doench, PhD, Director R&D, Genetic Perturbation Platform, Broad Institute of Harvard and MIT


Deeper, Finer and Wider with CRISPR Screens for Gene Function

John Doench, PhD, Director R&D, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

Genome-wide CRISPR screens have revitalized functional genomics. Large-scale data sets enable rapid hypothesis generation, and focused screening efforts can provide detailed mechanistic insights into the function of any gene of interest. Here I will discuss how CRISPR screens are being employed in gene function discovery projects, with an emphasis on the latest technological advances.


Biases and Blind Spots for CRISPR Screens in Target Finding

Traver Hart, PhD, Assistant Professor, Department of Bioinformatics and Computational Biology; Department of Cancer Biology, The University of Texas MD Anderson Cancer Center

CRISPR knockout screening can identify essential genes in cancer cell lines, and when these genetic vulnerabilities are strongly associated with a specific oncogene or tissue, they present ideal candidate therapeutic targets. However, functional buffering by related genes masks these vulnerabilities. We discuss how this blind spot in CRISPR screens can mislead drug targeting efforts, and discuss new CRISPR multiplex perturbation technologies to fill in the gap.


On-Target and Off-Target Cancer Drugs

Jason Sheltzer, PhD, Principal Investigator, Cold Spring Harbor Laboratory

We have recently discovered that many anticancer drugs function through off-target interactions. By deploying a variety of spontaneous and directed mutagenesis approaches, we can identify resistance-granting mutations, and thereby uncover their true targets. Using these techniques, we have recently discovered the first potent and specific inhibitor of the cyclin-dependent kinase, CDK11.

10:15 Coffee Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced

In vivo T Cell CRISPR Screen for Immunotherapy Target Discovery

Sidi Chen, PhD, Assistant Professor, Department of Genetics and Systems Biology Institute, Yale University; Member, Yale Cancer Center and the Yale Stem Cell Center

In vivo CRISPR screen is a powerful means for discovering therapeutic targets in physiologically relevant settings. Here, we describe recent advancements in in vivo T cell CRISPR screen for immunotherapy target discovery and characterization of example targets.


Cellular Target Engagement – De-Risking Preclinical Development

Elmar Nurmemmedov, PhD, Assistant Professor, Translational Neurosciences & Neurotherapeutics, John Wayne Cancer Institute

Discovery of therapeutics is a long and expensive process that requires biological, medicinal and clinical precision. Cellular target engagement is a new paradigm in drug discovery – it provides a physiologically relevant cellular environment for rigorous interrogation of candidate molecules. This emerging technology can be used for a variety of targets to test a variety of drug molecules, and can be combined with other emerging technologies, such as CRISPR.

12:25 Session Break
12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own
1:05 Refreshment Break in the Exhibit Hall with Poster Viewing


1:50 Chairperson's Remarks

Richie Kohman, PhD, Senior Research Scientist and Lead, Synthetic Biology Platform, Wyss Institute for Biologically Inspired Engineering, Harvard University


The State-of-the-Art in Highly Multiplexed Multi-in situ OMICs

Richie Kohman, PhD, Senior Research Scientist and Lead, Synthetic Biology Platform, Wyss Institute for Biologically Inspired Engineering, Harvard University

Biological tissues are immensely complex containing a huge diversity of chemical motifs in specific, three-dimensional locations. Most OMICs techniques, such as single-cell transcriptomics, do not retain the location of the targets they are analyzing. This talk will cover the state of the art in in situ OMICs, where molecules are analyzed within their endogenous environment, providing a crucial insight into the content of healthy and diseased tissues.


Multi-Scale Network Biology Approach to Identify Novel Targets for Parkinson's Disease (PD)

Bin Zhang, PhD, Professor, Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai

Molecular mechanisms underlying idiopathic PD, which account for 80% of the PD cases, remain elusive. We performed multi-scale gene network analysis of a large gene expression dataset in the substantia nigra from 83 PD cases and 70 controls, and systematically identified and prioritized co-expressed gene modules and key regulators in PD. This study lays down a foundation for developing a comprehensive signaling map and novel therapeutics for PD.


Detecting Virus and Deep Learning Its Integration Sites in the Human Genome

Zhongming Zhao, PhD, Chair, Center for Precision Health, Professor of Biomedical Informatics, The University of Texas Health Science Center at Houston

I will introduce our virus detect software, VirusFinder, which incorporates an algorithm for detecting virus under its rapid evolution model (mutations). I will introduce the Viral Integration Site DataBase (VISDB, https:/bioinfo.uth.edu/VISDB). In VISDB, we systematically collected and manually curated all virus integration sites (VISs) reported in the literature and publicly available resources. Finally, I will report DeepVip, a deep learning-based method for virus integration site prediction in the host genome.


Developing Standards for Potential Prognostic Complex Multicellular Models

Madhu Lal-Nag, PhD, Program Lead, Research Governance Council, Office of Translational Sciences, Center for Drug Evaluation & Research, U.S. Food and Drug Administration
Marc Ferrer, PhD, Leader, Biomolecular Screening and Probe Development, Division of Pre-Clinical Innovation, National Center for Advancing Translational Sciences, National Institutes of Health (NIH)

There has been much progress towards the experimental development of robust, scalable, and reproducible 3D cellular models, including spheroids, organoids, biofabricated tissues and microphysiological on chip systems, as assay platforms for preclinical drug testing.  However, there is a need for systematic physiological and pharmacological validation and benchmarking of the different 3D cellular models to establish their true clinical predictability and use for decision making in the drug discovery and development pipeline.

3:55 Close of Conference

Please click here to return to the agenda for Target Identification and Validation - Part 1

Choose 2 Short Courses and 2 Conferences/Training Seminars
Sept. 15 Short Course: SC8: Artificial Intelligence and Machine Learning in Drug Discovery and Development
Sept. 16-17 Conference: Target Identification and Validation – Part 1
Sept. 17 Dinner Short Course: SC11: Insights into PROTACs and Targeted Protein Degradation
Sept. 17-18 Conference: Target Identification and Validation – Part 2