The RNAi for Functional Genomics Screening will cover the latest in the use of RNA interference (RNAi) screens for identifying and validating drug targets and exploring unknown cellular pathways. It will cover everything from assay design to data analysis for the use of in vitro and in vivo siRNA (small interfering RNA) and shRNA (short hairpin RNA) screens. There is growing interest in combining RNAi screens with gene editing, chemical genomics, overexpression studies and phenotypic screens, which will all be discussed here. Screening experts from industry and academia will share their experiences leveraging the utility of these diverse screening platforms for a wide range of applications.

Final Agenda

Day 1 | Day 2 | Download Brochure 

Tuesday, September 22

7:00 am Registration and Morning Coffee


8:00 Chairperson’s Opening Remarks

Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech, Inc.

8:10 Harnessing High-Throughput RNAi for Target Identification Utilizing 3D Cell Culture Models

Geoffrey Bartholomeusz, Ph.D., Associate Professor and Director, siRNA Core Facility, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas M.D. Anderson Cancer Center

It is now well accepted that 3D cell culture models although varied and complex, replicate clinically relevant outcomes associated with the tumor microenvironment. They are thus relevant models for target discovery and drug screens. It is imperative to select a relevant 3D model when performing high-throughput screens. The development of 3D models used in high-throughput screening to address relevant questions in cancer biology will be discussed.

8:40 Development of Physiologically Relevant Assay Platforms to Probe Various Disease Pathologies

Madhu Lal-Nag, Ph.D., Team Leader, RNAi Screening, National Center for Advancing Translational Sciences, National Institutes of Health

There is a tremendous need for biologically relevant cancer models that can accurately predict the clinical efficaciousness of various drugs. Here we describe the development of In vitro model platforms that are designed to be biologically relevant to fill a critical gap between the cellular and animal model domains and offer the opportunity to study compounds that are therapeutically attractive.

9:10 Expanding the Screening Toolbox for the Interrogation of Gene Function

Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech, Inc.

It is increasingly evident that different approaches towards functional genomics screening (e.g., CRISPR, RNAi, and small molecules) have their own advantages and disadvantages. In that regard, a combined approach can be more informative than any one approach alone. This talk will attempt to highlight the need for a flexible approach towards functional genomics screening, and the contexts in which certain platforms may be more appropriate.

9:40 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing


10:25 Identifying Regulatory Interactions Among Genes Linked to Alzheimer’s Disease

Michael Ollmann, Ph.D., Principal Scientist, Genome Analysis Unit, Amgen, Inc.

10:55 High-Throughput RNAi Screening for Immune Modulatory Ligands on Tumor Cells

Philipp Beckhove. Ph.D., Head, Translational Immunology Division, German Cancer Research Center Heidelberg; Director, Regensburg Center for Interventional Immunology, University of Regensburg

Immune checkpoint inhibition has lately revolutionized cancer therapy. We have established the first high-throughput screening approach to characterize the entities of immune modulatory molecules on different tumors - the immune modulatome of cancer. We identified many novel candidates derived from gene families that were hitherto not reported to play a role in the immune system and will report functional validation data for some of them.

11:25 Functional Genomic Screening to Accelerate the Discovery of Combination Therapies

Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

Single drug cancer therapies meet with limited success due to drug resistance from reactivation or activation of redundant pathways. Due to the complexity of the targeted signaling networks in addition to tissue-specific characteristics, the prediction of the best combination therapy remains a major challenge. We apply large scale functional genomic screening in clinically relevant models to identify such interactions with the goal to accelerate the discovery of clinically active combinations.

11:55 High Content RNAi Screening with Persomics: Discover More Faster With Turnkey Printed Libraries

Neil Emans, Ph.D., CEO, Persomics USA, Inc.

RNA interference is routinely used in High Content and Phenotypic screening. However, set-up and operational costs are beyond the reach of individual labs and limit core facilities. Persomics technology miniaturizes, accelerates and de-industrializes RNAi screening. Preprinted libraries integrate with conventional HCS platforms and image analysis to enable off-the-shelf screening in individual labs and now allow core facilities to do more.

12:10 pm Sponsored Presentation (Opportunity Available)

12:25 Session Break

12:35 Luncheon Presentation to be Announced

1:15 Refreshment Break in the Exhibit Hall with Poster Viewing


1:50 Chairperson’s Remarks

2:00 Development and Application of CARD, a Comprehensive Integrated Web-Based Platform for Analysis of RNAi Screening Data

Iain Fraser, Ph.D., Investigator, Laboratory of Systems Biology, National Institute of Allergy and Infectious Diseases, National Institutes of Health

CARD is a comprehensive web-application for integrated analysis and interactive visualization of RNAi screening data. CARD combines both existing and novel algorithms for data pre-processing, reducing false positive hits through gene expression and off-target filtering, implementing network/pathway enrichment of high-confidence hits and predicting active miRNAs. We will discuss the application of CARD to several datasets and demonstrate both increased hit validation rates and improved hit overlap between related screens.

2:30 RNAi for Rare Disease Drug Discovery: Signal or Noise?

Christopher Gibson, Ph.D., Co-Founder and CEO, Recursion Pharmaceuticals

RNAi has increasingly well-described off-target effects. High-content imaging assays, for which vast quantities of morphological data are collected, are particularly prone to convolution due to such effects. Findings will be presented from 1000+ feature high-content imaging data in various human cell types using up to 6 RNAi for each of more than 100 disease-related targets. A discussion of the usefulness and limitations of the data resulting from such RNAi-based results will also be discussed.

3:00 Presentation to be Announced


3:30 Refreshment Break in the Exhibit Hall with Poster Viewing and Poster Winner Announced

4:10 3D Phenotypic Screening for Target Identification and Drug Discovery

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

A high-throughput kinome siRNA screen (880 kinases) was carried out to study their effects on tumor architecture and hypoxic response induced in 3D tumor spheroids. We present a workflow to identify and interpret gene function in such large scale 3D RNAi experiments by analyzing such image-derived data in the context of associated molecular data. Apart from describing the components of this integrative workflow, we will also share some “lessons learnt” during this process.

4:40 The Path from Arrayed RNAi to Arrayed CRISPR Screens: Lessons Learned and Challenges

Eugen Buehler, Ph.D., Group Leader, Informatics, National Center for Advancing Translational Sciences, National Institutes of Health

The use of CRISPR for whole genome functional screens has now been demonstrated by several groups. However, these screens have been performed only in a pooled format, which severely limits the range of biological functions that can be interrogated. We will detail our experiments in arrayed CRISPR screening and discuss the challenges and opportunities for future work.

5:10 Interactive Breakout Discussion Groups

6:10 Welcome Reception in the Exhibit Hall with Poster Viewing

7:15 Close of Day

Day 1 | Day 2 | Download Brochure 

Wednesday, September 23

7:30 am Registration and Morning Coffee

8:00 Chairperson’s Remarks

Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

8:10 Pooled shRNA, Arrayed siRNA and CRISPR-Cas9: Three Essential Tools towards Understanding Gene Function in Cancer and Disease Biology

Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

The addition of CRISPR-Cas9 systems to the incumbent technologies of arrayed siRNA and pooled shRNA have created an unparalleled gene perturbation toolbox for investigators to deploy. Specific examples, published and unpublished, of each of these types of experimental designs will attest to the power of these endeavors, particularly for cancer biology. Respective strengths and weaknesses of each technology will be presented.

8:40 Parallel shRNA and CRISPR/Cas9 Screens Reveal Biology of Stress Pathways and Identify Novel Drug Targets

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

We have developed high-complexity shRNA libraries (25 shRNAs/gene) that greatly reduce false negatives/false positives, and have adapted these libraries to knock down gene pairs to perform systematic genetic interaction maps in mammalian cells. We have used these maps to study ER-trafficking toxins, and identified novel protein complexes as well as insights into retrograde trafficking. We are now using this strategy together with the CRISPR/Cas9 system to study stress signaling and identify novel drug targets.

9:10 TECHNOLOGY PANEL: Finding the Right Functional Genomics Tool to Address Your Biological Question

(Opportunities Available For Sponsoring Panelists)

This panel will bring together 4-5 technical experts from leading technology and service companies to discuss screening trends and improvements in assay platforms and reagents that users can expect to see in the near future.

9:40 Coffee Break in the Exhibit Hall with Poster Viewing

10:25 Screening the Kinome: Lessons from Using Functional Screens in Glioblastoma Stem Cells

Brent Cochran, Ph.D., Professor, Department of Developmental, Molecular and Chemical Biology, Tufts University School of Medicine

It is possible to isolate and maintain in culture glioblastoma cell lines with stem cell properties. We have conducted shRNA screens of three different GBM stem cell lines in both arrayed and pooled screening format for growth and survival of these cells, under normoxia and hypoxia. We have found that there are considerable differences in the kinase requirements between these cell lines. These results argue for a personalized therapeutic strategy for glioblastoma.

10:55 From Model Systems to Mammalian Applications: Learning from Functional Genomics Analyses in Drosophila

Stephanie Mohr, Ph.D., Lecturer, Genetics & Director of the Drosophila RNAi Screening Center, Harvard Medical School

Drosophila cell and in vivo systems are exemplary platforms for functional genomics. We have developed algorithms for the design of RNAi and CRISPR reagents; platforms for efficient RNAi reagent production; and mature methods for large-scale screening and data analysis. Our workflows for CRISPR knockout and RNAi screens in human disease-relevant sensitized backgrounds will be discussed. Emphasis will be given to approaches, algorithms and analyses relevant to mammalian systems.

11:25 Enjoy Lunch on Your Own

12:55 pm Plenary Keynote Program

2:40 Refreshment Break in the Exhibit Hall with Poster Viewing

3:25 Close of Conference

Day 1 | Day 2 | Download Brochure 

Suggested Event Package: 

September 21 Short Course: Setting Up Effective RNAi Screens: From Design to Data to Validation 

September 21 Short Course: Setting Up Effective Functional Screens Using 3D Cell Cultures 

September 23 Short Course: A Primer to Gene Editing: Tools and Applications 

September 22-23 Conference: RNAi for Functional Genomics Screening 

September 23-24 Conference: New Frontiers in Gene Editing 


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2015 Discovery on Target Brochure  





Molecular Sensing 

Rosa Drug Development Advisors







The exhibit hall was sold out in 2014, so please contact us early to reserve your place. To customize your sponsorship or exhibit package for 2015, contact:

Jon Stroup
Sr. Business Development Manager


September 21 

Next-Generation Histone Deacetylase Inhibitors Symposia 

Strategies for Rare Diseases Symposia 

September 22 

Developing CRISPR-Based Therapies Symposia 

September 22 - 23 

Targeting Epigenetic Readers and Chromatin Remodelers 

Targeting the Ubiquitin Proteasome System 

Targeting the Microbiome 

GPCR - Based Drug Discovery - Part 1 

Antibodies Against Membrane Protein Targets - Part 1 

RNAi for Functional Genomics Screening 

Gene Therapy Breakthroughs 

Targeting Ocular Disorders 

September 23 - 24 

Targeting Histone Methyltransferases and Demethylases 

Targeting the Unfolded Protein Response 

Kinase Inhibitor Discovery 

GPCR-Based Drug Discovery - Part 2 

Antibodies Against Membrane Protein Targets - Part 2 

New Frontiers in Gene Editing 

Quantitative Systems Pharmacology 

Short Courses 

SC1: Cancer Metabolism: Pathways, Targets and Clinical Updates 

SC2: Leveraging Data and Analytics for Drug DiSCovery 

SC3: Setting Up Effective Rnai SCreens: From Design to Data to Validation 

SC4: Phenotypic SCreening and Chemical Probe Development 

SC5: GPCR Structure-based Drug Discovery 

SC6: Targeting of GPCRs with Monoclonal Antibodies 

SC7: Setting Up Effective Functional SCreens Using 3D Cell Cultures 

SC8: Targeting Protein-protein Interactions: Biophysical Approaches 

SC9: Preclinical Animal Models for Ocular Indications 

SC10: Introduction to Allosteric Modulators and Biased Ligands of GPCRs 

SC11: Introduction to Targeted Covalent Inhibitors 

SC12: Assays and High-throughput SCreening for Novel Epigenetic Inhibitors 

SC13: Gamification and Drug Target Challenges 

SC14: A Primer to Gene Editing: Tools and Applications 

SC15: Using Mechanistic Physiological Models In Drug Development