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TVD 09

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Recommended Short Courses*

12:00 pm - 3:00 pm Strategies for Effective RNAi Screens (SC1)*

3:30 pm - 6:30 pm Strategies to Optimize RNAi Delivery (SC3)*

*Separate Registration Required



12:30 pm Registration

Challenges in RNAi Delivery

2:00 Chairperson’s Opening Remarks

Christina Rondinone, Ph.D., Director, Research, Metabolic Diseases, Hoffmann-La Roche Inc.

2:10 Delivery is in the Details

Catherine J. Pachuk, Senior Director, Delivery Biology, Pfizer Research Technology Center

2:40 Stem Cells as a Non-Immunogenic Vehicle for Delivery of siRNA

Peter R. Brink, Ph.D., Professor and Chair, Department of Physiology and Biophysics, Stony Brook University

Although siRNA has the potential to be a unique therapeutic agent, delivery has been hampered by immune responses for siRNAs in the vascular or interstitial spaces. One potential delivery system are human mesenchymal stem cells (hMSCs) because they are not immunogenic and it has been shown that hMSCs can be injected into an organ (the heart) where they integrate into the tissue and form gap junctions with the target cells. Gap junctions are also capable of mediating transfer of siRNA from one cell to another. These features make for a viable cellular delivery system.                

3:10 Nanoparticles for Controlled and Targeted Delivery of siRNA

Mark Saltzman, Ph.D., Chair, Department of Biomedical Engineering, Yale University

3:40 Networking Refreshment Break in the Exhibit Hall

4:20 A Biodegradable, Biocompatible, Sustained Release System for RNAi Delivery

Seth D. Feuerstein, M.D., J.D., President, Carigent Therapeutics

While cationic and lipid delivery vehicles allow for encapsulation of high levels of RNAi material such as siRNA and miRNA, they are generally limited by cytotoxicity for direct applications as well as preference for delivery to the liver. Recently, polymer vehicles have begun to show promise for delivery of RNAi and can have specific and clear advantages such as delivering to different organs and less (or no) cytotoxicity. With high loading in non-cationic polymers that are FDA approved sustained release delivery can be achieved for direct applications such as, skin or systemically in organs such as the brain and lungs.

Biovex-Neurovex4:50 Highly Efficient Delivery of RNAi to Neurons in Vivo Using NeuroVex

Anna-Maria Anesti, Ph.D., Product Development Director, NeuroVex, BioVex Inc.

Suzanne Thomas, Ph.D., Director of Project Management, NeuroVex, BioVex Inc.

At BioVex we have developed a highly efficient method for gene silencing in neurons in vivo using NeuroVex disabled HSV-1 vectors. NeuroVex vectors allow delivery of shRNAs, artificial miRNAs, which are the new generation RNAi triggers with improved safety profiles for gene silencing in the nervous system, and multiple miRNAs in tandem for simultaneous silencing of multiple genes. Here, we show that NeuroVex delivery of shRNA/miRNA to sensory neurons in vivo results in highly effective and specific silencing of targeted genes. This has not been feasible using other vector systems and allows new approaches to in vivo gene target validation.

Mitoprod5:05 Conception of Circular Interfering RNA and Production’s Strategy
Guillaume Plane, CEO, MitoProd SA
Interfering RNAs represent a powerful tool for the Pharmaceutical Industry. Indeed, the stability, quality, in vivo efficiency, and delivery of these molecules represent major challenges for their development. Basically, by circularizing the primary transcript and thus removing extremities, MitoProd brings an innovative solution to face most of these challenges. In association with MitoProd innovative technology for RNA manufacturing, based on the fermentation of Saccharomyces cerevisiae yeast, Circular Interfering RNAs (ciRNA®s) offer some major advantages for the development of siRNA-based drugs.

 Cell Type-specific siRNA Delivery and the Treatment of Disease
Derek M. Dykxhoorn, Ph.D., Assistant Professor, University of Miami School of Medicine
The delivery of siRNAs to the appropriate cell types and tissues in vivo remains one of the biggest hurdles being faced for the development of RNAi-based therapeutic approaches. Cell type-specific approaches have been developed which facilitate the targeted delivery of siRNAs to specific cell populations by taking advantage of the affinity of various molecules (antibodies, aptamers, etc) for cell surface antigens. These approaches have taken the promise of RNAi-based therapeutics one step closer to reality.  





5:50 Combinatorial Development of Synthetic siRNA Delivery Systems

Daniel Anderson, Ph.D., Research Associate, Massachusetts Institute of Technology

High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing siRNA delivery systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery siRNA, both in vitro and in vivo. A number of siRNA delivery formulations have been developed with in vivo efficacy, and show potential therapeutic application for the treatment of genetic disease, viral infection, and cancer.               

6:20 Close of Day

Day 1  |  Day 2 |  Short Courses 



Suggested Event Package (November 1 – 4):

November 1

Pre-Conference Short Courses:

Strategies for Effective RNAi Screens (SC1) AND Strategies to Optimize RNAi Delivery (SC3)

November 2 - 3


RNAi for Screening Cellular Pathways and Targets 

November 3 - 4


RNAi for Developing Targeted Therapeutics




Arrow Overshort courses




The exhibit hall was sold out in 2015, so please contact us early to reserve your place. To customize your sponsorship or exhibit package for 2016, contact:

Jon Stroup
Sr. Business Development Manager



2015 Tracks

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

2015 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