Finding novel, druggable targets for therapeutic intervention remains a top priority for the pharma/biotech industry, especially when it comes to building a robust drug discovery pipeline. It also remains a formidable challenge and companies continue to invest a lot of time and resources in identifying and validating good drug targets to pursue. What are the challenges in target discovery? What tools and strategies are being used and how well are they working? What’s being done to ensure that validated targets lead to better and safer therapies? Cambridge Healthtech Institute’s conference on Target Identification and Validation will bring together leading experts to discuss some of these critical questions. The conference will help attendees meet and interact with experts and peers from around the world to share ideas and hear about new strategies and technologies helping target discovery.

 

Part 1: Focus on Functional Genomics and Computational Screening

This part of the Target Identification and Validation conference will highlight some of the existing and emerging functional genomics, artificial intelligence and machine learning tools that are being used for generating novel drug targets. The use of such tools and screens for validating existing targets or associating them with new disease indications will also be discussed.

Preliminary Agenda

USE OF CRISPR, RNAi AND OTHER GENOMIC SCREENS FOR FINDING NOVEL TARGETS

Leveraging Functional Genomics to Identify and Validate Novel Targets

Lauren Drowley, PhD, Director of Molecular Genetics, Translational Medicine, UCB Pharma

Up, Down, and Out: Complementary CRISPR Technologies for Genetic Screens

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

Investigating Cancer Drug Specificity with CRISPR/Cas9 Mutagenesis

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

Exploring New Biology and Targets Identified through Big CRISPR Data

Scott Martin, PhD, Group Lead, Functional Genomics, Genentech

Efficient Tools to Enable Drug Target and Biomarker Discovery

Paul Diehl, PhD, COO, Cellecta, Inc.

Cellecta’s technologies allow for precise and convenient analysis of the genes responsible for driving biological responses. Using NGS with multiplex RT-PCR in our DriverMap™ Genome-wide Expression Profiling Assay enables rapid association of gene expression changes in large numbers of cell, tissue, and blood samples. Examples of this approach will be discussed.


Development of High-Throughput Cellular Thermal Shift Assays: Cellular Mechanistic Assays in Early Drug Discovery

Marc Holbert, PhD, Scientific Leader Protein, Cellular and Structural Sciences, GSK

A problem in early drug discovery is lack of rank-order correlation between biochemical potencies from initial screens and diminished potency and efficacy in cellular phenotypic assays. Cellular thermal shift assays bridge this gap, enabling assessment of drug target engagement in live cells based on ligand-induced changes in protein thermal stability.

NEW FUNCTIONAL GENOMICS ASSAYS, LIBRARIES & PLATFORMS FOR TARGET IDENTIFICATION

Building CRISPR/Cas9-Based Platforms for Therapeutic Target Identification and Validation

Rui Wang, PhD, Senior Scientist, Department of Immunology, AbbVie Bioresearch Center

Target Deconvolution Using CRISPR Mutagenesis Scanning

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

A Membranome cDNA Library for Biologics Discovery and Phenotypic Screening

Xi Ai, PhD, Associate Principal Scientist, Screening & Compound Profiling, Merck Research Laboratories

TECHNOLOGY PANEL: Do We Have the Right Tools to Find Drug Targets That Are Difficult to Identify and Validate?

This panel will bring together speakers and 3-5 technical experts from leading technology and service companies to discuss improvements and limitations in assays, platforms, and data analysis tools available for target discovery and validation.

CRISPR Technology Mimicking the Fibrosis Process


Ann-Marie Zuurmond, Associate Director, Biology, Charles River

CRISPR technology is an asset for target validation in the drug discovery process with its ability to generate full gene knockouts. Using an adenovirus delivery system, efficient CRISPR-mediated gene knockout was obtained in a complex cellular assay mimicking the fibrosis process.

COMPLEMENTARY USE OF TECHNOLOGIES FOR TARGET VALIDATION

Talk Title to be Announced

Roderick Beijersbergen, PhD, Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

MACHINE LEARNING AND COMPUTATIONAL TOOLS FOR FINDING NOVEL TARGETS

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

Arvind Rao, PhD, Assistant Professor, Department of Bioinformatics and Computational Biology, The University of Texas MD Anderson Cancer Center

Computational Approaches for Target Identification

Deepak Kumar Rajpal, PhD, Director, Computational Biology, GlaxoSmithKline