Institute for International Research

Keynotes for IBC's TIDES Oligonucleotide and Peptide® Technology and Product Development Conference

Human Genetics and Oligonucleotides: Essential Partners in Pharma's Future
The revolution in human genetics is providing an unprecedented number of disease-associated genes. Oligonucleotide drugs could unlock the value of these potential targets, many of which are otherwise undruggable. Realizing the full potential of oligonucleotides for target validation and therapeutics requires an appreciation for unintended effects, especially immune activation, which will be a focus of this presentation.
Arthur M. Krieg, M.D., Chief Scientific Officer, Research Technology Center, Pfizer Inc

Delivering Innovation with RNAi Therapeutics
The pharma industry is looking to innovation as a way to control the market-based forces driving constraints on drug pricing and reimbursement, and as a way to fill their diminishing pipelines. Alnylam is focused on the discovery, development, and commercialization of RNAi therapeutics, a promising approach for a whole new class of innovative medicines.
John Maraganore, Ph.D., Chief Executive Officer, Alnylam Pharmaceuticals, Inc.

Peptide Therapeutics: Trends in Global Clinical Development and Approval
Peptide therapeutics are undergoing a rapid rise in the rate of development, and nearly 50 products are approved for marketing. To determine development and approval trends for peptide candidates, we collected data for over 320 therapeutic peptides that entered clinical study sponsored by commercial firms. The analysis to be presented has revealed trends in the type and mechanism of action of candidates, as well as the probabilities of approval success.
Janice M. Reichert, Ph.D., Senior Research Fellow, Tufts Center for the Study of Drug Development; Editor-in-Chief, mAbs, Landes Bioscience

Taking Advantage of Peptide Aggregation
Peptide aggregation can be problematic in peptide synthesis, purification and formulation. Understanding the physical factors that contribute to aggregation can help attenuate these problems. However, actively promoting molecular self-assembly by manipulating these same factors can afford novel materials whose nanostructural architecture and function can be controlled. Peptide-based self-assembled materials are finding use in diverse applications such as tissue regenerative therapies and drug delivery.
Joel P. Schneider, Ph.D., Associate Professor, Department of Chemistry and Biochemistry, Department of Materials Science and Engineering, University of Delaware

Keynote for IBC's Nucleic Acids Technologies for Diagnostics Conference

Reagent Solutions to Challenges in Oligonucleotide Diagnostics
Instrumental and cell-based technologies have advanced dramatically in human diagnostics that target nucleic acids. Lagging behind, by comparison, are novel reagents that deliver novel capabilities to diagnostics architectures that target DNA and RNA. This talk will describe advances in small molecule and protein reagents that permit highly multiplexed PCR, orthogonal capture, and highly selective priming, and their combinations in architectures that support oligonucleotide analysis, from detection of very few targets in circulating blood to personalized genome resequencing.
Steven A. Benner, Ph.D., Distinguished Fellow, Foundation for Applied Molecular Evolution Inc.

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2008 Event Wrap Up

Approximately 750 attendees, speakers and exhibitors networked and learned at TIDES® 2008. The event was hailed as a great success, excellent learning experience, superb forum for making new contacts and a very enjoyable time by all involved.

Participants heard new data in nearly 30 scientific sessions with over 100 presentations, including over 15 case studies, and learned of new products and services offered by 75 providers in the exhibit hall. Learning about cutting-edge science included the new data presented in over 25 posters.

Poster Winners

The advisory committee was split in its choices of poster winners, so two poster presenters shared the $250 cash poster award sponsored by BioProcess International. The two winning posters are:

• Modifications to MerMade 192 High Throughput Synthesizers for Efficient Aptamer Synthesis presented by Dipak Goyal, Archemix Corporation
• A Novel Therapeutic Approach for Fibrosis- Site Specific Delivery of siRNA against HSP47 in Novel Carriers presented by Yi Jin and co-workers, Nitto Denko Technical Corporation (Registered under Kinovate Life Sciences, Inc.) and Sapporo Medical University

Regarding the Archemix technology, the advisors said: usually high-throughput synthesis rhymes with plain-vanilla oligos, with simple synthesis. This development shows that it is possible to make high-throughput synthesis of heavily modified oligos. This new development appears to be an enabling technology for the fast screening of aptamers.

The data from Nitto Denko/Kinovate demonstrated that siRNA against the collagen specific molecular chaperone, HSP47, can be delivered specifically to stellate cells, and the treatment approach can inhibit collagen synthesis and reverse fibrosis in liver, lung and pancreatic models. Liposomes conjugated with vitamin A served as vehicles for the targeted delivery of antifibrotic siRNA.

Event Highlights

The keynote presentation by Dr. Blair A. Fraser, of CDER, FDA on current expectations on the CMC of Peptide- and Oligonucleotide-based Therapeutics was particularly appreciated by attendees. Participants also gained insights from the lively standing-room-only discussion session on CMC Regulatory Expectations where panelists included Dr. Fraser, Dr. René Thürmer of BfArm, Germany's regulatory agency, and industry regulatory experts.

The latest information on big pharma's perspective on the potential for peptide and oligo-based therapeutics were given in additional keynotes and plenary session presentations: delivered by Alan Sachs, M.D., Ph.D., Vice President, RNA Therapeutics, Merck Research Laboratories and by Waleed Danho, Ph.D., Discovery Chemistry, Hoffmann-LaRoche Inc., and on new understanding of miRNA by experts from The University of Texas M.D. Anderson Cancer Center and Rosetta Inpharmatics LLC, Merck & Co., Inc.

Content of new data disclosed was rated the highest ever in the tracks on peptides and oligos in preclinical and clinical development, the Process and Analytical Development session, and the full day on routes of administration in development.

The conference on Nucleic Acids Technologies for Diagnostics drew record attendance with highlights of the keynote by Dr. Steven Gutman of CDRH, US FDA, and Dr. Ichiro Hirao of RIKEN Genomic Sciences Center and TagCyx Biotechnologies, Japan.