Academic Discovery Workshop Agenda

Breakfast Welcome and Registration

Where are we at with RNAi Screening?
Scott Martin, Group Leader, RNAi Screening, NCATS, NIH, United States of America

There has been increased skepticism about the value of RNAi screening. This presentation will discuss many of the pitfalls associated with the technology and how they should shape screening and validation strategies.

Comparative Analysis of Genome-wide RNAi Screens
Bhavneet Bhinder, Bioinformatics Engineer, Memorial Sloan Kettering Cancer Center, United States of America

We conducted genome-wide screens using an EGFP-biosensor image based reporter assay, and utilizing the two flavors of RNAi– the siRNA duplex and the shRNA hairpin technologies. The screens were performed under identical experimental conditions and method of hit nomination. A head-on comparison amongst the two screens exhibited an overlap of only 29 hits, although 15,068 genes overlapped amongst the two libraries.  I would present the results and discuss the reasons for such discrepancy in outputs.

High Throughput Screening Utilizing 3D Multicellular Tumor Spheroids Models
Geoffrey Bartholomeusz, Assistant Professor, MD Anderson Cancer Center, United States of America

We have used a non-matrix scaffold to generate 3D spheroid model of pancreatic cancer to perform high throughput RNAi screens. Validation of our top ranked targets has resulted in the identification of novel regulators of spheroid architecture. 

RNAi Screens to Identify Novel Therapeutic Strategies for Leukemia: Going All The Way
Chris Porter, Assistant Professor / Pediatrics, University of Colorado School of Medicine, United States of America

Genome-scale RNA-interference screens are used as an unbiased approach to address numerous biological questions, including for the identification of novel therapeutic strategies for cancer. We will present our experience with genome-scale RNAi screens, which have led to clinical trial design and implementation.

Coffee and Networking

Genome Editing as an Emerging Technology to Functionalize the Human Genome and Accelerate Targeted Drug Discovery
Arumugham Raghunathan, Sr. Director, Business Development, North America , Horizon Discovery Ltd, United Kingdom

The talk will discuss recent advances in the gene-editing platforms and describe the application of the technology in basic functional genomics research, target ID & validation, lead discovery, production of bio-pharmaceuticals and companion diagnostic development.

CARD: Comprehensive Analysis of RNAi-screen Data
Iain Fraser, Head Laboratory of Systems Biology, National Institutes of Health, United States of America

We will present the features of the CARD software and examples of its application to RNAi screen datasets.

The Functional Biology Toolbox: A Tale of siRNA, miRNA, CRISPR and TALs
Nitin Puri, Associate Director, Product Management, Life Technologies, United States of America

An overview and discussion of the latest CRISPR and TAL cell engineering tools and the siRNA/miRNA technologies for discovery and characterization of gene function.

Lunch and Networking

Expert Panel Discussion on "RNAi and Genome Editing Technologies"
Nitin Puri, Associate Director, Product Management, Life Technologies, United States of America

Session Moderated by Nitin Puri

Panel Participants:
Bhavneet Bhinder, Scott Martin, Chi Yun, Geoffrey Bartholomeusz, Christopher Porter, Arumugham Raghunathan, Iain Fraser

Discussion Topics:

• RNAi Data Reproducibility
• How Many Targets from RNAi Thus Far?
• Novel Genome Editing Technologies and their Impact
• The Future of RNAi Screening

Drinks Reception

Breakfast

Biochemical Assays for Protein-protein Interaction Inhibitor Discovery
Yuhong Du, Assistant Professor and Associate Director, Emory University, United States of America

Modulating Regulators of G Protein Signalling – A Therapeutic Challenge
Richard Neubig, Professor and Chair, Michigan State University, United States of America

RGS4 is an attractive drug target for Parkinson’s disease, neuropathic pain, etc. However, targeting protein-protein interactions is a challenge. Utilizing allosteric mechanisms, protein dynamics, and even covalent ligands may be required in such cases. 

Targeting Protein-protein Interactions in the DNA Damage Response
Kamyar Hadian, Group Leader, Helmholtz Zentrum München, Germany

Historically, the protein-protein interaction (PPI) target class has been rather underexplored with respect to identifying small molecule inhibitors. We have targeted a DUB-E2 enzyme interaction to modulate DNA damage response. We are able to show specific and selective compound effects in vitro and in the cellular context. 

Coffee and Networking

Two Cellular Compartments for the Screening of Antivirals Against Two Processing-related Targets
Roland Wolkowicz, Associate Professor, San Diego State University, United States of America

We have developed two distinct cell-based assays for the search of antivirals targeting viral processing. The assays, adaptable to HTS, monitor the viral protease in a cytosolic environment or host enzymes in the classical secretory pathway. 

Designing High throughput Assay Platforms for Phenotypic Screening in Physiologically Relevant Disease Models
Madhu-Lal Nag, Research Scientist, NCATS, NIH, United States of America

Cytell™ Cell Imaging System - A Simplified Solution for Automated Cell Imaging and Analysis
Steve Titus, Field Application Scientist, GE Healthcare, United States of America

Automated image cytometry technologies offer significant potential to simplify and improve robustness of routine cell assays, but are hampered by inflexibility with respect to the range of compatible assay and sample formats, technical complexity, and lack of integration of the assay and analysis from start to finish. Cytell Cell Imaging System is application-driven through the use of “BioApps” that each enables a specific biological application or assay, and covers all experimental steps from imaging to reporting of the quantitative results. We will present data showing use of Cytell to run a 96-well multi-color cell viability assays complete with full data visualization and report generation. A second example demonstrates rapid quantification of cell cycle blocker effects on phase distribution with accuracy comparable to current benchmarks.

New Dynamic Injection (diSPRTM) Techniques Offer Expanded Biophysical Information in Surface Plasmon Resonance Technology with Concurrent Improvements in Throughput, Reduction of System Error and Simplified Instrument Operation/Data Analysis
Rick Cope, Regional Sales Manager, SensíQ Technologies Inc., United States of America

The FastStep  automated titration method, provides complete, one-pass, kinetics, quickly, with lower systematic noise. OneSteps’ gradient approach captures diffusion coefficients for aggregation assessment, processing 3-4 concentration orders of kinetics, in one pass. SensiQ’s new Pioneer FE(Fragment Edition) with Pharma input improves data and processing time in fragment based drug discovery.

Lunch and Networking

Expert Panel Discussion on "Target vs Phenotypic Based Discovery
Haian Fu, Professor and Director, Emory University, United States of America

Session Moderated by Haian Fu

Discussion Topics:

• Target Based Drug Discovery
• Phenotypic Based Assays
• High Content Assays
• 3D Cell and Physiologically Relevant Model Systems
• Novel and Enabling Screening Technologies in 2014
• Vendor/User Perspectives on HCA in 2014

Close of Day 2

Breakfast

Transactional vs. Collaborative Relationships – Improving Technology Transfer through Academic Entrepreneurship
Charles Sacco, Entrepreneur in Residence and Director of External Relations, Drexel University, United States of America

This session will discuss the development of an Academic Entrepreneurship curriculum that is being created at Drexel University’s Charles D. Close School of Entrepreneurship for the purpose of cultivating a new set of academic entrepreneurs, who can help facilitate the collaborations between universities and companies, who can identify the best opportunities to translate research and discoveries into viable business models, and who can potentially start their own ventures either individually or in collaboration with others.

Themed Alliances as a Model for Academic-industry Drug Discovery Collaborations
Tim Hammonds, Deputy Director Drug Discovery, Cancer Research Technology, United Kingdom

This talk will describe with examples the benefits of focussing discovery alliances in themed areas of oncology which bring together multiple PIs from multiple universities with Pharma partners to form large multi-project discovery teams.  

The Evolution of Academic Technology Transfer
Jeanne Farrell, Business Development Director, Ichan School of Medicine, United States of America

This presentation addresses the changes (and need for change) occurring in the approach by academic institutions to address drug discovery and development, as well as the new partnership opportunities with Pharma, Biotech, and academic spin-out companies.

PhDs, Assets in the Technology Commercialization Engine
Viviane Martin, Associate Director, Penn State Hershey Medical Center, United States of America

Public-Private Partnership can take numerous forms. However in all forms, the science generates the business opportunity, bringing two different worlds to try to work together. I will discuss why and how PhDs are key players in the establishment and management of these collaborations.

The Changing Face of Science and Technology Innovation in Drug Discovery: Envisioning a Path to the Future
Litao Zhang, Vice President/Leads Discovery & Optimization, Bristol-Myers Squibb, United States of America

This talk will focus on recent trends and opportunities to use technology platforms to drive drug innovation. The challenges and gaps to enable target selection and modality optimization will be also discussed. The future perspectives for how to integrate technology platforms across discovery processes will be addressed as well.

Coffee and Networking

CROs and the Changing Paradigm of Drug Discovery
Christine Brideau, Vice President of In Vitro Biology, WuXi, United States of America

This presentation will discuss the changing landscape of outsourcing, its benefits as well as future challenges to meet the increasing demand for high quality work with shorter cycle times and lower cost. CROs are constantly improving their capabilities and offer a wider choice of collaboration models to suit the needs of the customer.

Academic Drug Discovery: The Key for The Future?
Thierry Langer, CEO, Prestwick Chemical, France

Prof. Langer will showcase the results of recent research projects that can be run on academic scale using in silico fragment screening for the discovery of novel attractive starting points for medicinal chemistry programs.

Lunch and Networking

Expert Panel Discussion on "Public and Private Partnerships"
Jeanne Farrell, Business Development Director, Ichan School of Medicine, United States of America

Session Moderated by Jeanne Farrell

Panel Participants:
Tim Hammonds , Litao Zhang, Viviane Martin, Thierry Langer

Discussion Topics:

• Academic Technology Transfer Offices
• Building Alliances between Academia and the Private Sector
• Will Academia - Industry Collaborative Research Enhance Data Reproducibility?
• How to Manage IP Expectations
• Tips for Successful Collaborations with the Private Sector

Close of Workshop