Eldorado Hotel & Spa Floorplan

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This meeting took place in 2012



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Nucleic Acid Therapeutics: From Base Pairs to Bedsides (A2)


Organizer(s) Alan M. Gewirtz (in memoriam), Anastasia Khvorova and Mark A. Kay
January 10—15, 2012
Eldorado Hotel & Spa • Santa Fe, New Mexico USA
Abstract Deadline: Sep 15, 2011
Late Abstract Deadline: Oct 13, 2011
Scholarship Deadline: Sep 15, 2011
Early Registration Deadline: Nov 10, 2011

Sponsored by Isis Pharmaceuticals, Inc. and Regulus Therapeutics Inc.

Summary of Meeting:
Small molecules and antibodies have revolutionized the treatment of cancer and appear promising for the treatment of many other diseases as well. Nonetheless, there are many candidate therapeutic targets that are not amenable to attack by the current generation of targeted therapies and, in a small but growing number of patients, resistance to initially successful targeted treatments evolves. This symposium will focus on the medicinal promise of pre- and post- transcriptional gene silencing (PPTGS) using oligodeoxynucleotides, small interfering RNA, microRNA and other nucleic acid based molecules. The meeting is motivated by the hypothesis that these molecules can be developed into effective drugs for the treatment of many common diseases that are collectively responsible for a great deal of human suffering. As this symposium will highlight, there is still much to do, but many in the field now hope that the time has finally arrived when “antisense” therapies will finally come of age and fulfill their promise as the magic bullets of the 21st century.

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No registration fees are used to fund entertainment or alcohol at this conference

Conference Program    Print  |   View meeting in 12 hr (am/pm) time


TUESDAY, JANUARY 10

15:00—19:30
Registration

Zia Concourse
18:15—19:15
Refreshments

Zia Concourse
19:15—20:00
Welcome and Introductory Remarks
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom

WEDNESDAY, JANUARY 11

07:00—08:00
Breakfast

Eldorado Court
08:00—11:00
Basic Mechanisms Underlining Oligonucleotide Therapeutic Development
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Jennifer A. Doudna, HHMI/University of California, Berkeley, USA

Bruce A. Sullenger, Duke University, USA
Controlling Thrombosis with Aptamers and Inflammation with Their Antidotes

Robert J. Crouch, NICHD, National Institutes of Health, USA
Ribonucleases H: Function in Hydrolysis of RNA/DNA Hybrids

John L. Rinn, University of Colorado, Boulder, USA
Large Intergenic Non-Coding RNAs: From Discovery to Mechanism

Jin Billy Li, Stanford University, USA
Generating an Atlas of Mammalian A-to-I RNA Editing

Dongbo Yu, University of Texas Southwestern Medical Center, USA
Short Talk: Allele-Selective Inhibition of Huntingtin Expression by Single-Stranded RNAs that Act through the RNAi Pathway

09:20—09:40
Coffee Break

Zia Concourse
11:15—13:00
Poster Setup

Sunset Room/Eldorado Court
13:00—22:00
Poster Viewing

Sunset Room/Eldorado Court
 
On Own for Lunch and Recreation

16:30—17:00
Coffee Available

Zia Concourse
17:00—19:00
Preclinical Developments
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Bruce A. Sullenger, Duke University, USA

Anastasia Khvorova, University of Massachusetts Medical School, USA
Asymmetric, Hydrophobically Modified RNAi Compounds: From Mechanism of Action to Clinical Development

Ryszard Kole, Ercole Biotech Consulting, USA
Eteplirsen, an Exon-Skipping Drug Candidate for Treatment of DMD

Rasmus O. Bak, University of Aarhus, Denmark
Short Talk: Variable Potency and Transfer Efficiency of Lentivirus-Encoded miRNA Inhibitors

Darren Wong, Medivation, Inc., USA
Short Talk: Antitumor Efficacy with Dicer Substrate siRNAs in the Absence of Immunostimulatory Effects

Anita G. Seto, miRagen Therapeutics, USA
Short Talk: Discovery and Development of Novel Cardiovascular Drug Candidates Based on microRNA Targeting

19:00—20:00
Social Hour with Lite Bites

Eldorado Court/Sunset Room
19:30—22:00
Poster Session 1

Sunset Room/Eldorado Court

THURSDAY, JANUARY 12

07:00—08:00
Breakfast

Eldorado Court
07:30—08:00
Poster Setup

Sunset Room/Eldorado Court
08:00—17:00
Poster Viewing

Sunset Room/Eldorado Court
08:00—11:15
Clinical Applications I
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Larry Gold, SomaLogic Inc., USA

Giles Campion, Prosensa Therapeutics, Netherlands
Systemic Antisense Treatment for Duchenne Muscular Dystrophy

Richard S. Geary, Ionis Pharmaceuticals, Inc., USA
Role of Mipomersen in Potential Treatment of Severe Familial Hypercholesterolemia

Ian MacLachlan, Tekmira Pharmaceuticals Corp., Canada
Integrating Preclinical and Clinical Experience in the Development of siRNA Products

Eli Gilboa, University of Miami, Miller School of Medicine, USA
Aptamer-Targeted Modulation of Tumor Immunity

Elena Feinstein, Quark Pharmaceuticals Inc., Israel
Short Talk: siRNA Therapeutics for Eye Diseases

09:20—09:40
Coffee Break

Zia Concourse
11:15—12:00
Lunch

Eldorado Court/Sunset Room
12:00—14:30
Poster Session 2

Sunset Room/Eldorado Court
16:30—17:00
Coffee Available

Zia Concourse
17:00—17:50
Keynote Address
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Mark A. Kay, Stanford University, USA

Craig C. Mello, University of Massachusetts Medical School, USA
RNA World and Therapeutic Applications

17:50—19:00
Structural Insights: Form Follows Function
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
Jennifer A. Doudna, HHMI/University of California, Berkeley, USA
Dicing and Beyond: Regulatory RNA in Humans and Bacteria

Remo Rohs, University of Southern California, USA
DNA Shape is a Source of Protein-DNA Binding Specificity

19:00
On Own for Dinner


FRIDAY, JANUARY 13

07:00—08:00
Breakfast

Eldorado Court
08:00—11:00
Novel Technologies in Oligonucleotide Target Discovery
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, USA

Peter Sarnow, Stanford University School of Medicine, USA
Masking of the Hepatitis C Viral Genome by miR-122

Jonathan S. Weissman, University of California, San Francisco, USA
Monitoring Protein Synthesis One Codon at a Time through Ribosome Profiling

Stephen B. Baylin, Johns Hopkins University School of Medicine, USA
Methylation Mapping

Larry Gold, SomaLogic Inc., USA
SOMAmers for Diagnostics and Therapeutics

Shuo Gu, Stanford University, USA
Short Talk: Slicing Independent RISC Activation Requires the Argonaute Paz Domain

09:20—09:40
Coffee Break

Zia Concourse
 
On Own for Lunch and Recreation

16:30—17:00
Coffee Available

Zia Concourse
17:00—19:00
Clinical Applications II
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Peter Sarnow, Stanford University School of Medicine, USA

Dinah Sah, Voyager Therapeutics, USA
RNAi Therapeutics in Liver and Neurologic Diseases

Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, USA
MicroRNA Therapeutics in Cancer

Arthur A. Levin, Avidity Biosciences, USA
Targeting miR-122 for Treatment of Chronic Hepatitis C Infection with Miravirisen a 15- mer LNA-Modified AntimiR Oligonucleotide: From Bench to Bedside

19:00—20:00
Social Hour with Lite Bites

Eldorado Court/Sunset Room

SATURDAY, JANUARY 14

07:00—08:00
Breakfast

Eldorado Court
08:00—11:15
Endogenous Small RNAs vs Exogenous Oligos
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Arthur M. Krieg, Checkmate Pharmaceuticals, USA

Mark A. Kay, Stanford University, USA
RISC Loading of Duplex RNAs: Unwinding the Prospects for Antiviral Therapeutics

Christine Esau, Arcturus Therapeutics, USA
Targeting miR-33 for Atherosclerosis

Muthiah Manoharan, Alnylam Pharmaceuticals, Inc., USA
“The End of the Beginning:” Evolution of siRNA Conjugates as the Next Generation Systemic Delivery Approach for RNAi Therapeutics

Joshua T. Mendell, HHMI/University of Texas Southwestern Medical Center, USA
MicroRNAs in Cancer: Biology and Therapeutic Applications

Ruth Broering, University Hospital of Essen, Germany
Short Talk: In vivo Application of LNP01-Formulated siRNAs Activates the Hepatic Immune System which can be Controlled by Chemical Ribose Modifications

Christopher J. Cheng, Yale University, USA
Short Talk: Cell-Penetrating Nanoparticles for Tumor-Targeted OncomiR Inhibition

09:00—09:20
Coffee Break

Zia Concourse
 
On Own for Lunch and Recreation

16:30—17:00
Coffee Available

Zia Concourse
17:00—19:15
Novel Chemistries, Enhancing Enzymology and Animal Models in Oligonucleotides Therapeutics Development
Meeting has ended...abstracts no longer viewable online.

Anasazi Ballroom
* Ian MacLachlan, Tekmira Pharmaceuticals Corp., Canada

Brett P. Monia, Ionis Pharmaceuticals, Inc., USA
Broadening RNaseH-Based Antisense Pharmacology through Medicinal Chemistry

Allen W. Nicholson, Temple University, USA
Alternative Mechanisms of Antisense Silencing by 2’-Fluoroarabinose Oligonucleotides

Arthur M. Krieg, Checkmate Pharmaceuticals, USA

Thazha P. Prakash, Ionis Pharmaceuticals, Inc., USA
Short Talk: Chemical Approaches for Achieving Activation of RNA Interference with Single Stranded Oligonucleotides in Animals

Marcin T. Kortylewski, Beckman Research Institute, City of Hope, USA
Short Talk: Two-Pronged Approach to Human Cancer Immunotherapy using CpG-STAT3 siRNA Conjugates

19:15—20:15
Social Hour with Lite Bites

Eldorado Court/Sunset Room
20:00—23:00
Entertainment

Eldorado Court/Sunset Room

SUNDAY, JANUARY 15

 
Departure


*Session Chair †Invited, not yet responded.



We gratefully acknowledge support for this conference from:


Directors' Fund


These generous unrestricted gifts allow our Directors to schedule meetings in a wide variety of important areas, many of which are in the early stages of research.

Click here to view all of the donors who support the Directors' Fund.



Keystone Symposia thanks our Sponsors for generously supporting this meeting:

Isis Pharmaceuticals, Inc. Regulus Therapeutics Inc.

We gratefully acknowledge additional support for this conference from:

Integrated DNA Technologies Sigma Life Science

We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:



Isis Pharmaceuticals, Inc.


Prosensa Therapeutics


Regulus Therapeutics Inc.


RXi Pharmaceuticals


Tekmira


We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:


Click here to view more of these organizations


Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:


Click here to view more of these organizations


If your organization is interested in joining these entities in support of Keystone Symposia, please contact: Sarah Lavicka, Director of Development, Email: sarahl@keystonesymposia.org,
Phone:+1 970-262-2690

Click here for more information on Industry Support and Recognition Opportunities.

If you are interested in becoming an advertising/marketing in-kind partner, please contact:
Yvonne Psaila, Director, Marketing and Communications, Email: yvonnep@keystonesymposia.org,
Phone:+1 970-262-2676