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A Dual Model System Approach to Study microRNAs and Cancer Progression
Aurora Esquela Kerscher, Assistant Professor, Eastern Virginia Medical School, United States of America

MicroRNAs have emerged as promising diagnostic and therapeutic targets for cancer. The Esquela-Kerscher laboratory uses a combination of C. elegans and mammalian models to functionally characterize miRNAs and determine their role in controlling processes related to cellular growth and differentiation.

microRNA Regulation of Cell Viability and Drug Sensitivity in lung Cancer
Alexander Pertsemlidis, Associate Professor, The University of Texas Health Science Center at San Antonio, United States of America

High-throughput screening of libraries of microRNA mimics and inhibitors identifies microRNAs and target genes and pathways that modulate cell viability, either alone or selectively in the context of chemotherapeutic agents.

MiR-149 Targets Caspase-2 to Promote Glioma Cell Growth in vitro & in vivo
Kan Ding, Professor, The National Center for Drug Screening, China

MiR-149 binds to the 3'UTR of caspase-2 to down-regulate caspase-2 expression. Caspase-2 may impair glioma cells growth. Overexpressed miR-149 in U87 glioma cells xenografted in nude mice resulted in significant tumor growth by targeting caspase-2.

Coffee Break and Networking in Exhibition Hall

Transcriptome Sequencing Identifies Oncogenic microRNA Signatures that Interact with NF-kappaB, TP53 and Notch Signaling in Head and Neck Squamous Cell Carcinoma
Anthony Saleh, IRTA scientist, NIDCD/NIH, United States of America

Functional comparison of microRNA expression signatures of head and neck squamous cell carcinoma (HNSCC) cell lines and tumor specimens has been used to identify oncogene driven microRNA expression patterns that repress the p53 and Notch signaling pathways.

Translating miRNA Discovery in Biofluids into Robust Biomarkers for Disease, Toxicology or Injury Studies – The Case of Minimally Invasive Colorectal Cancer Detection
Peter Mouritzen, Vice President, Exiqon A/S, Denmark

Using LNA™ based Universal RT-PCR system, we have profiled microRNA in thousands of biofluid samples including blood plasma/serum and urine. Implementation of extensive sample and data QC procedures will support development of robust biomarkers in disease, toxicology and injury studies.

Lunch Break and Networking in Exhibition Hall

Poster Viewing Session

Viability Screens with Pooled shRNA Libraries Identify Novel and Synthetically Lethal Anti-Cancer Targets
Alex Chenchik, President/CSO, Cellecta Inc, United States of America

We identified and confirmed hundreds of genes modulating proliferation and survival in oncogenic cells using pooled lentiviral shRNA libraries. Also, we adapted and executed an approach to combinatorially screen shRNA to discover additive and synergistic combinations that are synthetically lethal.

Surface-Enhanced Polymer Nanoparticles for Inhibition of Oncogenic MicroRNAs
Christopher Cheng, Postdoctoral Fellow, Yale University, United States of America

Cancers can become addicted to oncogenic microRNAs. I will detail the development of a nanotherapeutic system that elicits microRNA inhibition in solid tumors. Blocking miR-155 in a model of miR-155-addicted lymphoma inhibited tumor growth; introducing a new platform for microRNA therapeutics.

Coffee Break and Networking in Exhibition Hall

Asymmetric, Hydrophobically Modified RNAi Compounds: From Mechanism of Action to Clinical Development
Karen Bulock, Associate Director/Vice President, RXi Pharmaceuticals, United States of America

This presentation will provide an overview of our recent work to develop sd-rxRNA®, a class of improved RNAi compounds that are readily taken up by cells without any delivery vehicle or formulation.  Data supporting the potential therapeutic uses of sd-rxRNA will be presented.

Enabling RNAi Therapeutics Development through Modified siRNA
Xianbin Yang, Director, AM Biotechnologies LLC, United States of America

Chemically modified siRNA duplexes with enhanced delivery and improved pharmacokinetics properties are required for therapeutic applications. This presentation, will describe (1) RNA monomers with dual modification chemistries for synthesizing modified siRNAs; (2) the pharmaceutical and biological properties of modified siRNA, and (3) the high potency modified-siRNA gene silencing in vitro and in vivo.

Therapeutic Antagonism of microRNAs with LNA: A Translational Research Modality
Anja Hoeg, Associate Director of Discovery, Santaris Pharma, Denmark

LNA oligonucleotides are potent inhibitors of coding and non-coding RNA. SPC3649 is the first micro-RNA inhibitor in man and has shown safe and significant reduction in HCV viral load in patients. It will be illustrated in the talk how the clinical and pre-clinical data closely interrelate and how this has formed the basis for the fast and coherent development path of the drug. The SPC3649 case story suggests that LNA oligonucleotides targeting disease related microRNAs may enable translational research from bench to clinic.

MicroRNA Market Analysis: Life Science Research, Diagnostics, and Therapeutics
Enal Razvi, Managing Director, Select Biosciences Inc, United States of America

This presentation describes the latest market trends in the microRNA field.  Based on our continuous primary research, Select Biosciences describes the quantitative market metrics for microRNA research, as well as the utility of microRNAs as diagnostics as well as for therapeutic targeting and the current microRNAs that are being focused upon as well as the current programs across the industry where they are being targeted.

Drinks Reception

The Many Roles of Argonaute Proteins in microRNA Biogenesis and RNA Interference
Sven Diederichs, Group Leader, University Of Heidelberg, Germany

Argonaute proteins had been discovered as the effector proteins of the microRNA pathway. However, I would like to shed some new light on the many roles that Argonaute proteins can play in microRNA processing, stabilization, strand selection and RNA interference.

Life Without MicroRNAs
Michael McManus, Assistant Professor, University of California San Francisco, United States of America

The importance of miRNAs during the development and disease processes is well established. However, most studies have been done in cell lines or with patient tissues, and therefore the physiological roles of miRNAs are not well understood. We address these limitations with the use of knockout mouse models.

Coffee Break and Networking in Exhibition Hall

microRNA Activity and Cytoplasmic Stress Responses are Regulated by Poly(ADP-ribose)
Anthony Leung, Assistant Professor, Johns Hopkins Bloomberg School of Public Health, United States of America

Poly(ADP-ribose) is a polynucleotide of 2-200 ADP-ribose subunits covalently attached to a target protein. Since discovery in 1963, our understanding of this polynucleotide has been focussed on its roles in the nucleus, including DNA repair, transcription and chromatin structure maintenance. Recently, we discovered two novel roles of poly(ADP-ribose) in the cytoplasm – regulating microRNA activity and stress responses.

Improved Design of Anti-miRNA Oligonucleotides
Mark Behlke, Vice President/Chief Scientific Officer, Integrated DNA Technologies Inc, United States of America

Anti-miRNA Oligonucleotides (AMOs) are a version of antisense oligos that inhibit miRNA function by a steric blocking mechanism of action. IDT has developed a novel chemical modifying group which improves nuclease stability and increases potency while maintaining high specificity of 2’OMe AMOs.

Lunch Break and Networking in Exhibition Hall

Poster Viewing Session

Gene Signatures for Evaluating RNAi Screening Data and Performance: A Novel Paradigm for Output Convergence
Bhavneet Bhinder, Bioinformatics Engineer, Memorial Sloan Kettering Cancer Center, United States of America

RNAi has offered the premise to perform knockdowns to discover genes. To date, it is faced with challenges as confirmatory studies were scarce with some refuting data. A strategy to evaluate RNAi data through context-dependent signature is proposed and its merits will be discussed.

Nanoparticle Mediated Delivery of siRNA In Vivo; What Will It Take To Move Into Clinic?
Andrew Miller, Professor, Kings College London, United Kingdom

Nanoparticle technologies are a potentially potent way to solve the problem of functional delivery of RNAi effectors in vivo. This lecture is concerned with current proof of therapy experiments and will explore what needs to be achieved for meaningful clinical trials going forward.

Coffee Break and Networking in Exhibition Hall

Gagomers: Novel Safe Particle Clusters for Systemic Delivery of RNAi Payloads
Dan Peer, Head, Tel Aviv University, Israel

Gagomers are Lipid-based nanoparticle clusters coated with the glycosaminoglycan Hyaluronan (HA). The gagomers have a unique ability to entrap large amounts of RNAi payloads and deliver them selectively into tumors expressing HA receptors in a highly selective manner. In my talk, I will detail the physicochemical and structural properties of this novel strategy and will show biodistribution and specificity data combined with efficacy data and safety profile.

Systemic Delivery of a Tumor-Targeted Polymeric Nanoparticle Achieves Durable RNAi-Mediated Silencing in Breast Orthotopic Xenograft
Scott Eliasof, Vice President, Cerulean Pharma Inc, United States of America

We will present durable in vivo RNAi silencing in an orthotopic breast xenograft model with minimal toxicity, using a novel nucleic acid carrier designed and developed with our tumor-targeted polymeric nanoparticle technology.

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