Meeting Agenda

8th Annual Meeting of the Oligonucleotide Therapeutics Society
Boston, Massachusetts

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Schedule subject to change

DAY ONE
Date: Sunday, October 28

10:00am-12:45pm    Registration and Poster Set Up
12:45pm-1:00pm      Welcome and Opening
………………………..       Gunther Hartmann, MD, PhD, OTS President
1:00pm-3:30pm        Keynote Session

Keynote Address:

1:00-1:45pm
RNA Biology and Therapeutic Innovations
Phillip A. Sharp, PhD, Massachusetts Institute of Technology

Plenary Lectures:

1:45pm-2:30pm
RNAi Therapeutics: State of the State
John Maraganore, PhD, Alnylam Pharmaceuticals

2:30pm-3:30pm
Antisense Technology: Past, Present, Future
Stanley T. Crooke, MD, PhD, Isis Pharmaceuticals

3:30pm-4:00pm       Refreshment Break
4:00pm-5:30pm       Session I: “Hot Topics, Late Breaking News”
……………………………    Chair: Masad Damha, PhD, McGill University
…………………………….   Speakers selected from submitted abstracts

4:00-4:15pm
Tumor-targeted nanoparticles that exploit oncomiR addiction for cancer therapy
Christopher Cheng, PhD, Yale University

4:15-4:30pm
Long Noncoding RNAs Link Transcriptional Regulation of Inflammatory Pathway Genes
Bethany Janowski, PhD, UT Southwestern Medical Center

4:30-4:45pm
Highly efficient siRNAs with very low off-target effects
Gunter Meister, PhD, University of Regensburg

4:45-5:00pm
Lariat and Mini-lariat RNA: Synthesis and Debranching-Dependent RNA Interference
Subha Das, PhD, Carnegie Mellon University

5:00-5:15pm
siRNA dynamics – What happens inside a cell? About uptake, release, degradation and stability
Markus Hirsch, PhD Candidate, University of Mainz

5:15-5:30pm
Improving the Pharmacological Effects of Oligonucleotides by Enhancing Endosomal Escape
Rudy Juliano, PhD, University of North Carolina


5:30pm-7:00pm       Welcome Reception with Exhibitors and Informal Poster Viewing

                                              Dinner on your own

DAY TWO
Date: Monday, October 29

8:00am-10:00am      Session II: Chemical Modifications and Structural Biology I
……………………………    Chair: Fritz Eckstein, PhD, Max-Planck-Institute for Experimental Medicine

8:00-8:30am
Structural and Mechanism of Human Argonaute-2
Ian MacRae, PhD, The Scripps Research Institute

8:30-9:00am
Chemically Modified Nucleosides for RNAi:  A Platform Approach
Gabor Butora, PhD, Merck

9:00-9:30am
2’-O-Me-4’-thioRNA as a Potential Use for Oligonucleotide Therapeutics
Akira Matsuda, PhD, Hokkaido University

9:30-10:00am
microRNA Quantification in Tissue Sections and Plasma from Human
Tom Tuschl, PhD, Rockefeller University

………………………………
10:00am-10:30am     Refreshment Break

10:30am-12:30pm  Session III: Chemical Modifications and Structural Biology II
………………………………  Chair: Tom Tuschl, PhD, Rockefeller University

10:30-11:00am
Finding RNA Structure in Influenza and Using Isoenergetic Microarrays to Reveal Oligonucleotide Binding Sites
Douglas Turner, PhD, University of Rochester

11:00-11:30am
Why Induced Fit Matters
Andy Ellington, PhD, University of Texas

11:30am – 12:00pm
Stereocontrolled Synthesis and Properties of P-chiral Oligonucleotides
Takeshi Wada, PhD, University of Tokyo

12:00-12:30pm
Structure-Activity Relationships of RNase H Oligonucleotides
Punit Seth, PhD, Isis Pharmaceuticals
12:30pm-2:00pm…. Lunch and Informal Poster Viewing

2:00pm-4:00pm       Session IV: Delivery of Oligonucleotides
……………………………… Chair: Muthiah Manoharan, PhD, Alnylam Pharmaceuticals

2:00-2:30pm
Conjugation Strategies for In Vivo siRNA Delivery
Rajeev Kallanthottathil, PhD, Alnylam Pharmaceuticals

2:30-3:00pm
Optimization of Lipid Nanoparticles and siRNA Conjugates for Enablement of siRNA Therapeutics
Vasant Jadhav, PhD, Merck

3:00-3:30pm
Recent Advances in Lipid Nanoparticle-Mediated Delivery of RNAi Therapeutics
Martin Maier, PhD, Alnylam Pharmaceuticals

3:30-4:00pm
Dynamic PolyConjugate (DPC) Technology for Targeted Delivery of siRNA
David Lewis, PhD, Arrowhead Research Corporation

4:00pm-4:30pm       Refreshment Break

4:30pm-6:30pm       Poster Session I: Attended Poster Session
…………………………..   .Odd numbered posters

6:30pm-8:00pm       Meet the Experts
……………………………  . An offsite networking opportunity with “no host” dinner

DAY THREE
Date: Tuesday, October 30

8:00am-10:00am      Session V: MicroRNAs and Non-Coding RNAs
…………………………  …  Chair: Art Krieg, MD, RaNA Therapeutics

8:o0-8:30am
microRNAs and their Regulatory Effects
David Bartel, PhD, Massachusetts Institute of Technology

8:30-9:00am
Protection of the hepatitis C viral RNA genome and modulation of polyadenylation site using Insig1 mRNA by liver-specific pre- and mature microRNA 122
Peter Sarnow, PhD, Stanford University

9:00-9:30am
microRNA Therapeutics- Issues and Answers
Art Levin, PhD, miRagen Therapeutics

9:30-10:00am
miR-33: A Therapeutic Target for Cardiometabolic Diseases
Kathryn Moore, PhD, New York University Langone Medical Center

10:00am-10:30am     Refreshment Break
10:30am-12:30pm     Session VI: RNAi/ASO Preclinical Studies
……………………………. …Chair: Bob D. Brown, PhD, Dicerna Pharmaceuticals

10:30-11:00am
Targeting alpha1-antitrypsin for the Treatment of A1AT Liver Disease
Shuling Guo, PhD, Isis Pharmaceuticals

11:00-11:30am
RNAi-Mediated Inhibition of a Natural Anticoagulant for the Treatment of Hemophilia
Akin Akinc, PhD, Alnylam Pharmaceuticals

11:30am – 12:00pm
Spherical Nucleic Acids (SNAs): Novel Topical Agents for the Treatment of Skin Disease and Brain Cancer  Chad A. Mirkin, PhD, Northwestern University

12:00-12:30pm
MicroRNAs as Targeted Therapeutics and Therapeutic Targets in Cancer
Frank Slack, PhD, Yale University

12:30pm-2:00pm     Lunch and Informal Poster Viewing

2:00pm-4:00pm       Session VII: Alternate Mechanisms Preclinical Studies
…………………………….. Chair: Ryszard Kole, PhD, Sarepta Therapeutics

2:00-2:15pm
AAV6-mediated Systemic Delivery of Therapeutic RNAi Hairpins as a Treatment for Dominant Muscular Dystrophy Joel Chamberlain, PhD, University of Washington

2:15-2:30pm
Potentiating immunological memory in mice using aptamer targeted siRNA delivery to inhibit mTORC1 function in CD8+ cytotoxic T lymphocytes
Eli Gilboa, PhD, School of Medicine, University of Miami

2:30-3:00pm
Treatment of Spinal Muscular Atrophy with Antisense Morpholinos that Alter Splicing of SMN2 Good Distribution in Neonates and Adult Animals
Arthur Burghes, PhD, Ohio State University

3:00-3:30pm
Correction of myotonic dystrophy in mouse models by antisense targeting of nuclear-retained RNA
Charles Thornton, MD, University of Rochester Medical Center

3:30-4:00pm
Knocking Down HIV Transmission Using CD4 Aptamer-siRNAs
Judy Lieberman, MD, PhD, Children’s Hospital Boston and Harvard Medical School

4:00pm-4:30pm       Refreshment Break

4:30pm-6:30pm       Poster Session II: Attended Poster Session
…………………………….. Even numbered posters

6:30pm-9:30pm       Banquet Dinner
                               Tickets: $50.00 per personLimited space available and RSVP required.
…………………………….  The banquet dinner will include networking opportunities, live music by
…………………………….  Takeshi Wada, and a talk by Dr. Paul Schimmel from the Scripps
…………………………….  Research Institute.

DAY FOUR
Date: Wednesday, October 31

8:00am-10:00am      Session VIII:
…………………………….   Advances in Oligonucleotide Clinical Development I
…………………………….   Chair: Anastasia Khvorova, PhD

8:00-8:30am
Development of Miravirsen an Oligonucleotide Directed Against miR-122 : Clinical Study Results
Michael Hodges, MD, Santaris Pharma a/s

8:30-9:00am
Exon Skipping in Duchenne Muscular Dystrophy
Giles Campion, MD, PhD, Prosensa

9:00-9:30am
RNAi: From Concept to Human Proof-of-Concept
Akshay Vaishnaw, MD, PhD, Alnylam Pharmaceuticals

9:30-10:00am
Development of Antisense Drugs for Cancer
Brett Monia PhD, Isis Pharmaceuticals

10:00am-10:30am     Refreshment Break
10:30am-12:30pm     Session IX:
………………………………  Advances in Oligonucleotide Clinical Development II
……………………………..  Chair: Giles Campion, PhD, Prosensa

10:30-11:00am
Efficacy of CYT003-QbG10 – an A-type CpG Delivered in Virus-like Particles – in Patients with Allergic Asthma          Philipp Müller, MD, Cytos Biotechnology

11:00-11:30am
Results of the Eteplirsen Phase 2b and Phase 2b Extension Study in Duchenne Muscular Dystrophy
Ed Kaye, MD, Sarepta Therapeutics

11:30am-12:00pm
Bioanalytical Tools for Optimization of siRNA Chemistry and Improved In Vivo      Functionality
Ingo Röhl, AxoLabs
12:00-12:30pm

Development of a 2’-MOE Antisense Oligonucleotide as a Novel Approach to Treat Skin Scarring & Fibrosis    Jeff Jensen, BS, Pfizer

12:30pm-1:30pm      Lunch

1:30pm-3:30pm       Session X: Oligonucleotide Safety, PK, and Toxicity
……………………………… Chair: Art Levin, PhD, miRagen Therapeutics

1:30-2:00pm
Pharmacokinetic and Pharmacodynamic Properties of Antisense Oligonucleotides: Bridging Nonclinical to Clinical
John Grundy, PhD, Isis Pharmaceuticals

2:00-2:30pm
Renal Tolerability of 2’-MOE Antisense Oligonucleotides (ASOs) and The Role of Drug Accumulation
Husam Younis, PharmD, PhD, Isis Pharmaceuticals

2:30-3:00pm
Hepatic Safety Across Species
Yann Tessier, DVM, PhD, Santaris Pharma a/s

3:00-3:30pm
PFRED – An Open Source Oligonucleotide Design and Analysis Tool
Robert Stanton PhD, Pfizer

Closing Remarks
End of Conference