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TIDES: Oligonucleotide and Peptide Therapeutics from Research through Commercialization

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Access the hottest fields in therapeutics by attending TIDES - the only event with full coverage of discovery, development and manufacturing

May 12-15, 2014
Rhode Island Convention Center
Providence, RI

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  • Japanese
  • Korean
  • Taiwanese
  • Chinese

Oligonucleotide Therapeutics Discovery

Oligonucleotide Therapeutics Discovery

Oligonucleotide Therapeutics Discovery

Tuesday, May 13, 2014 · Keynote & Plenary Sessions

Tuesday keynote and plenary sessions are shared across all tracks. Please click on the respective links below for the full Tuesday agenda.

7:30
Registration and Coffee

12:05
Lunch on your own

5:30
Boston Tea Party
Celebrate Like It's 1773!
Networking, Wine and Cheese Reception in Poster and Exhibit Hall
Relax with fellow attendees in the poster & exhibit hall over complimentary drinks & snacks, as we celebrate the 240th anniversary of the famous Boston Tea Party held in 1773.
Sponsored by

Wednesday, May 14, 2014 · Main Conference

8:00
Registration and Coffee

8:15
Chairman's Remarks
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark

Medicinal Chemistry and Chemical Modifications to Improve Oligonucleotide Properties

8:30
Small Molecules That Enhance the Pharmacological Effects of Oligonucleotides
The attainment of strong pharmacological effects with oligonucleotides is hampered by inefficient delivery of these molecules to their sites of action in the cytosol or nucleus. We have developed a novel approach to delivery involving a non-toxic small molecule. We demonstrate that the compound Retro-1 can substantially enhance the effectiveness of antisense, siRNA and splice switching oligonucleotides in cell culture. This effect occurs at the level of intracellular trafficking and correlates with increased oligonucleotide accumulation in the nucleus. We also show that Retro-1 can alter the effectiveness of oligonucleotides in the in vivo setting. These observations indicate that it is possible to enhance the pharmacological actions of oligonucleotides using non-toxic small molecule adjuncts.
Rudy Juliano, Ph.D., Professor, School of Pharmacy, University of North Carolina

9:00
New Structure Activity Determinants for LNA Oligonucleotides
Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark

9:30
RNAi Therapeutics Using GalNAc-siRNA Conjugate Delivery Platform
Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.

10:00
Carba-LNA Modified Oligos (siRNA & Antisense) for Cellular mRNA Targeting
Incorporation of lipophilic or hydrophobic substituents on the Carba-LNAs (cLNA) lead to significant modulation of the antisense and siRNA properties, such as target RNA affinity, nuclease resistance and RNase H or the ago protein elicitation [JOC, 76, 440 (2011)]. For example: the cLNA modified siRNAs targeting HIV-1 TAR region downregulates HIV-1 replication successfully better than LNA counterparts [Med. Chem. Commun., 2, 1110 (2011); ibid 2, 206 (2011)]. Similarly, Allele-Selective Inhibition of single mutant Huntingtin expression can be effected with cLNA modified antisense oligonucleotides targeting the expanded CAG Repeat [Biochemistry, 9(47), 10166 (2010)].
Jyoti Chattopaohyaya, Ph.D., Professor, Chemical Biology Program, Department of Cell & Molecular Biology, University of Uppsala, Sweden

10:30
Networking Refreshment Break in Poster and Exhibit Hall

Medicinal Chemistry and Chemical Modifications to Improve Oligonucleotide Properties

11:15
DNA Strand Invading Locked Nucleic Acid Oligonucleotides Downregulate Huntingtin Gene Expression
We have developed locked nucleic acid-based phosphorothioate oligonucleotides directed against the Huntingtin (HTT) gene. These oligonucleotides have the capacity to invade into double-stranded DNA and downregulate the expression of HTT by more than 70%. Downregulation was obtained in fibroblast cell lines from patients with Huntington Disease. Different design features and modes of delivery will be discussed.
C. I. Edvard Smith, M.D., Ph.D., Professor, Laboratory Medicine Karolinska Institutet, Sweden

11:45
Self-Delivering Bioreversible, PhosphoTriester RiboNucleic Neutral (siRNN) Prodrugs
In the world of RNAi therapeutics, siRNA delivery remains the technological problem to solve. We pioneered self-delivering monomeric RNAi triggers by synthesis of neutral bioreversible phosphotriester siRNN prodrugs that are converted by intracellular restricted enzymes into charged phosphodiester siRNAs that induce robust RNAi responses.
Steven Dowdy, Ph.D., Professor, Cellular and Molecular Medicine, UCSD School of Medicine

12:15
Role of Chemical Modification in Improving RNA Therapeutics
Results from structural (X-ray), in vitro, and in vivo studies will be reported to show that 2′-OMe-phosphorodithioate (MS2) monomers are remarkably useful as novel constituents of siRNA duplexes, anti-miRNAs, and aptamers. The introduction of MS2 modifications have improved the potency of unmodified siRNA duplexes by a factor of 6, increased the binding affinity of unmodified therapeutic aptamers by a factor of 1000 (KD from nM to pM), and significantly increased the ability of anti-miRNAs to inhibit miRNAs.
Xianbin Yang, Ph.D., Director, R&D, AM Biotechnologies

12:45
Networking Luncheon in Poster and Exhibit Hall
Enjoy roundtable discussion topics where you can have follow up discussions to the information presented earlier. Sponsored by

1:55
Chairman's Remarks
Steven Dowdy, Ph.D., Professor, Cellular and Molecular Medicine, UCSD School of Medicine

Discovery and Preclinical Strategies

2:00
Targeted Therapeutic Alternative Splicing
Antisense oligonucleotides can redirect pre-mRNA splicing patterns to bypass protein truncating mutations and slow the progression of the muscle wasting disease, Duchenne muscular dystrophy. Targeted splice switching will be relevant to many different genetic disorders, and is more dependent on the type of gene lesion than the gene itself.
Steve Wilton, Ph.D., Director, Foundation Chair in Molecular Therapeutics, Australian Neuromuscular Research Institute, Murdoch University, Australia

2:30
A Novel, Potent, and Safe RNAi Therapeutic Platform Utilizing LUNAR© Delivery Technology
Arcturus Therapeutics, Inc. owns an intellectual property (IP) portfolio of 32 patents relating to Unlocked Nucleic Acid (UNA) technology. Arcturus is focused on the development and commercialization of LUNAR© siRNA drug delivery systems for the orphan disease market. Utilizing a novel, potent, and safe LUNAR© Delivery Technology, Arcturus plans to nominate its first clinical candidate in 2014 and expand its pipeline, bringing safe and effective RNAi therapeutics to the clinic.
Pad Chivukula, Ph.D., Chief Operating Officer, Arcturus Therapeutics, Inc.

3:00
Novel Potent Antisense Oligonucleotides Targeting Transforming Growth Factor beta (TGF-β) Isoforms
The main outcomes of extensive R&D programs around identification and selection of highly potent and selective antisense oligonucleotides targeting the various TGF-β isoform mRNAs will be presented. The preclinical profile of the initial development candidates for oncology therapeutic intervention will be discussed.
Michel Janicot, Ph.D., Chief Scientific Officer, Isarna Therapeutics GmbH, Germany

3:30
Networking Refreshment Break in Poster and Exhibit Hall

Discovery and Preclinical Strategies

4:15
The Prognostic and Predictive Utility of Measuring Biomarkers in Serum and Other Bodily Fluids
The measurement of microRNAs in the serum provides an opportunity to gain both prognostic and predictive information. We are showing that distinct microRNA signatures can distinguish patients with multiple sclerosis from normal volunteers, patients with high risk of metastasis of colorectal cancer and other malignant disease. We are also developing a microRNA signature that may be both prognostic and predictive for patients with rheumatoid arthritis. The ability to develop such microRNA signatures from routine blood tests could have important implications in the management of patients with a wide range of disease states.
Nelson Chau, Ph.D., Director of Target Identification and Target Assay Group, Regulus Therapeutics

Audience Interactive Panel Discussion

4:45
What Constitutes a Good Target for Oligonucleotide Modalities?
  • Identifying targets by comparing expression profiles of disease vs. healthy tissues
  • Credentialing targets with relevant animal models and/or human systems
  • Coding or non-coding - what are the issues?
  • Target expression in organs
  • Targeted effects versus off-target effects?
Moderator:
Nelson Chau, Ph.D., Director of Target Identification and Target Assay Group, Regulus Therapeutics

5:30
Summer Soirée
Networking Cocktail Reception in Poster and Exhibit Hall
What better way to kick off Summer 2014 than to relax over cocktails and snacks with fellow attendees? Join us at this summer-themed reception in the poster and exhibit hall, where you can network with attendees and speakers in a relaxed, informal environment. Don't forget to participate in our booth crawl, where many exhibitors will be giving away complimentary food and prizes.

Thursday, May 15, 2014 · Main Conference

7:30
Coffee

8:25
Chairwoman's Remarks
Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel

PK/PD and Toxicity of Oligonucleotides

8:30
Understanding Delivery Barriers of siRNA Therapeutics by Monitoring Metabolic Trafficking
We have developed numerous methodologies to monitor and understand the detailed in vivo metabolic trafficking of siRNAs administered by various delivery modalities from injection to loading into the RNA-induced silencing complex. By following siRNA metabolic trafficking in various pre-clinical species we have been able to better understand key delivery barriers of various siRNA delivery modalities.
Mark Cancilla, Ph.D., Director, PPDM, Merck & Co., Inc.

9:00
Mechanisms of Vascular Toxicity in Monkeys with Antisense Oligonucleotides
Drug induced vascular injury (DIVI) has been noted in monkeys with a variety of antisense oligonucleotide therapies demonstrating proinflammatory potential. The pathophysiologic mechanisms of DIVI caused by these therapeutics (associated with complement activation) appear different than those of small molecules, with unique risk assessment. This session will explore these important topics.
Kendall S. Frazier, Ph.D., Director of Pathology, GlaxoSmithKline

Preclinical and Clinical Oligonucleotides

9:30
Update on Mirror-image Aptamers in Phase IIa Studies
Mirror-image oligonucleotides enjoy the properties of being extremely biostable and non-immunogenic in biological environments. Currently, three L-RNA oligonucleotide-based drugs inhibiting different targets are evaluated in Phase IIa clinical studies. An update on learnings from patient studies will be presented.
Sven Klussmann, Ph.D., Chief Scientific Officer, Noxxon Pharma AG, Germany

10:00
The Role of PMO Chemistry in Emerging Infections Diseases: Efficacy and Safety in Hemorrhagic Virus Therapeutics to Potential Treatment of Multi-drug Resistant Bacteria
Michael Wong, M.D., Senior Medical Director, Sarepta Therapeutics

10:30
Networking Refreshment Break in Poster and Exhibit Hall

11:15
Aptamers and Conjugates Exemplified by UNA- and LNA-based Constructs
Examples will be given on evolution and conjugation of aptamers containing LNA (locked nucleic acid) and UNA (unlocked nucleic acid) nucleotides. These will include variants of the known anti-cancer aptamer AS1411 as well as novel apatmer constructs.
Jesper Wengel, Ph.D., Chairman and CSO, RiboTask and Professor, Center Director BioNEC, University of Southern Denmark

11:45
Multivalent N-Acetylgalactosamine Conjugate Significantly Improves Potency of Antisense Oligonucleotides in Mice
Asialoglycoprotein receptor is a C-type lectin expressed on hepatocytes and it recognizes terminal N-acetylgalactosamine (GalNAc) glycan. We utilized multivalent GalNAc conjugates to improve the productive uptake of antisense oligonucleotide (ASO) in liver. Multivalent GalNAc conjugate improves potency of ASOs in liver 8-10 fold. The details of these results will be presented.
Thazha P. Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals

12:15
Use of RNAi for Treatment of Inner Ear Disorders
Disorders of the inner ear represent major unmet medical need and no specific pharmacological treatments are yet available. Our data indicate that siRNA can be efficiently delivered to various inner ear structures in both small and large animals. Some of our siRNA compounds conferred significant and substantial inner ear neuroprotection and attenuation of hearing loss in mouse genetic model of Meneire's disease. Another subset of our siRNA compounds appeared effective in promoting regeneration of inner ear sensory epithelia and recovery of balance and hearing function in animals in which these functions were previously impaired by ototoxic treatments.
Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel

12:45
Networking Luncheon in Poster and Exhibit Hall Sponsored by

1:55
Chairman's Remarks
David Brown, Ph.D., Director of Research, Mirna Therapeutics, Inc.

Preclinical and Clinical Oligonucleotides

2:00
Preclinical and Clinical Development of a miRNA-Based Cancer Therapy
Our lead therapeutic candidate, MRX34, is a liposome-formulated mimic of the tumor suppressor miRNA miR-34. The systemic delivery of MRX34 produces complete tumor regression in two orthotopic models of liver cancer and is currently being evaluated for safety, tolerability, and pharmacokinetics in a Phase I clinical trial. In parallel with the Phase I trial, we are using cell and animal studies to identify cancer indications in addition to primary liver cancer that are responsive to MRX34 alone or in combination with approved therapies.
David Brown, Ph.D., Director of Research, Mirna Therapeutics, Inc.

2:30
Nucleic Acid Ligands with Enhanced Chemical Diversity for Development of Novel Therapeutics
We have recently developed a new class of nucleic acid ligands with protein-like side chains, called slow off-rate modified aptamers (SOMAmers). Expanded range of accessible epitopes has allowed us to identify SOMAmers to over 1,100 proteins. Some of these proteins represent validated drug targets, creating the potential for development of SOMAmer-based therapeutics.
Nebojsa Janjic, Ph.D., Chief Science Officer, SomaLogic, Inc.

3:00
Networking Refreshment Break

3:30
RNA Therapeutics to Selectively Activate Protein Expression
Long non-coding RNAs have recently been ascribed many roles that control biological processes including gene transcription. lncRNAs appear to affect gene expression in response to different developmental or environmental signals. One of the mechanisms through which lncRNAs regulate gene expression is via recruitment of PRC2. On the X chromosome, PRC2 recruitment to the lncRNA Xist can prevent binding of PRC2 or other specific epigenetic factors, thereby interfering with X chromosome inactivation. We present evidence that this or a similar mechanism may operate generally across the genome to constitutively suppress gene transcription, and that oligonucleotide inhibitors of lncRNA interactions with epigenetic factors such as PRC2 may selectively de-repress gene expression in vitro and in vivo. Such selective activation of protein expression could have therapeutic benefits in multiple disease areas.
Romesh Subramanian, Ph.D., Senior Director, Biology, RaNA Therapeutics

4:00
Clinical Development of RXI-109 to Prevent Dermal Scarring
RXI-109 targets connective tissue growth factor (CTGF), a key regulator of fibrosis. In Phase 1 clinical trials, RXI-109 was safe and well tolerated and reduced CTGF mRNA and protein locally in a dose dependent manner following intradermal administration. In Phase 2, RXI-109 will be evaluated in patients undergoing scar revision surgery or keloid removal.
Karen G. Bulock, Ph.D., Vice President, Research, RXi Pharmaceuticals

4:30
Presentation TBA
Speaker TBA

5:00
Close of Conference

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