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RNA Interference: From Tools to Therapies

 

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Thursday, February 4

 

SCREENING AND VALIDATING DRUG TARGETS
USING RNAi SCREENS

8:25 AM Chairperson’s Remarks

Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co.

8:30 Cancer Target Identification and Validation by siRNA Library Screening

Xiaoyu Lin, Ph.D., Associate Research Investigator, siRNA Therapeutics, Abbott Laboratories

We have been performing large-scale siRNA library screens to identify novel cancer targets. One of the critical aspects of screen data analysis is to discard false positive hits due to siRNA off-target effect. We will discuss several different approaches to confirm on-target effect of siRNA library hits. Using several targets identified in the library screen as examples, we will update on the progress of how RNAi-based technologies have helped target discovery and validation in the oncology area.

9:00 Hitting Cancer Where it Hurts Most: Large Scale RNAi Screens for Cancer Cell Vulnerability

Roderick L. Beijersbergen, Ph.D., Group Leader, Division of Molecular Carcinogenesis, The Netherlands Cancer Institute

Large scale RNAi screens for cancer cell vulnerability RNA interference based technologies allow for the interrogation of the role and phenotype of all individual genes in the human genome. Using these techniques we aim to identify those genes that upon functional inactivation have a causal effect on tumor cell behavior and survival representing novel drug targets.

9:30 Leveraging RNAi and Chemogenomic Screens for Target Identification and Validation

Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co.

Libraries of RNAi reagents are being widely used for screening cellular assays for target identification.  We are using RNAi libraries in combination with libraries of small molecule tool compounds.  Using both types of libraries adds confidence to hits identified from these screens and provides genetic and chemical tools for hit follow up.  Examples will be presented to illustrate this approach in target identification and validation.

10:00 Sponsored Presentation (Sponsorship Opportunity Available)

10:30 Poster Competition Refreshment Break & Raffles in the Exhibit Hall

11:30 Panel: Do’s and Don’ts in RNAi Screening

Panelists:

Christophe J. Echeverri, Ph.D., CEO and CSO, Cenix BioScience GmbH

Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co.

Hakim Djaballah, Ph.D., Director, HTS Core Facility, Memorial Sloan Kettering Cancer Center

Paul Kassner, Ph.D., Scientific Director, Lead Discovery, Amgen, Inc.

12:30 PM Luncheon Presentation Sponsored by
RNAi and KinaseSwitch Technology Platforms
Christine L. Olsson, Ph.D., Commercialization Scientific Director, Taconic
Novel in vivo technology platforms have recently been developed that will enable investigators to gain greater insights into drug and target-related disease mechanisms. TaconicArtemis RNAi and KinaseSwitch mouse models are the newest commercially available additions to these technologies. Inducible/reversible RNAi technology enables gene knockdown in all tissues of the body and can be induced and reversed, providing an optimal surrogate for therapeutic drug action. More recently, KinaseSwitch technology, an invaluable tool during key stages of drug development, allows investigators to identify biological roles of a specific kinase and possible side effects that result from its inhibition.
 

1:45 Ice Cream Refreshment Break in the Exhibit Hall

@ ekSp@

 

PLENARY KEYNOTE SESSION

2:15 Plenary Keynote Introduction

2:25 Chips, Clones and Living Beyond 100

Paul J.H. Schoemaker, Ph.D., M.B.A., Chairman and Chief Executive Officer, Decision Strategies International, Inc.; Research Director, Mack Center for Technological Innovation, The Wharton School; Adjunct Professor of Marketing, The Wharton School Adjunct Professor, Wharton School of Business

As information technologies and life sciences continue to converge, new business opportunities and challenges will arise for the field of diagnostics and beyond. This keynote reviews the deeper forces shaping the future of the biosciences, from social and economic to technological and political, including the stresses they will introduce for existing business models and healthcare. Not only will bioconvergence introduce new products, services and competitors, it may create entirely new industries on a scale larger than the computer revolution has to date. Several broad scenarios will be painted for the state of the biosciences in 2025 and the forces that might take us there, summarizing a multi-year strategy study conducted and supervised by the speaker at the Wharton school. 

3:05 Refreshment Break in the Exhibit Hall

 

CLINICAL CHALLENGES WITH RNAi THERAPEUTICS

3:45 Chairperson’s Remarks

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

3:50 LNA Antimirs – Pioneering microRNA Therapeutics

Henrik Orum, M.Sc., Ph.D., VP and CSO, Santaris Pharma

Short, single stranded LNA oligonucleotides delivered systemically as naked molecules are able to potently and safely inhibit therapeutically attractive miRNAs in a range of tissues in experimental animals. The presentation will provide an update on the unique features of LNA oligonucleotides in miRNA therapeutics with particular emphasis on the pre-clinical and clinical development of SPC3649, an LNA AntimiR targeting miRNA-122.

4:20 Pre-clinical and Clinical Development of Atu027 (siRNA-lipoplex/AtuPLEX) for Oncology

Ansgar Santel, Ph.D., Senior Scientist, Silence Therapeutics plc

Atu027 refers to a liposomally formulated siRNA targeting PKN3 expression in the vascular endothelium. Pre-clinical studies in rodents and non-human primates demonstrated that intravenous administration is well tolerated and gives rise to RNAi-mediated suppression of PKN3 gene expression. Various proof-of-concept experiments on mouse tumor xenografts suggest profound inhibition of tumor progression and particularly of metastasis, which laid the foundation for therapeutic application in oncology. Atu027 is currently tested in a Phase-I clinical trial on subjects with advanced solid cancers. Pre-clinical data emphasizing pharmacological activity in mouse models and an update on the current Phase-I study will be discussed.

4:50 Talk Title to be Announced

Ian MacLachlan, Ph.D., CSO, Tekmira Pharmaceuticals

5:20 Talk Title to be Announced

Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals Corp.

5:50 Close of Day

 

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