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Informa
8:00am - 8:10am

Registration and Coffee

Showing of Streams
10:15am - 10:55am

Networking Refreshment Break with Exhibit and Poster Viewing

Showing of Streams
Showing of Streams
4:45pm - 4:50pm
Close of Conference

Close of Conference

8:00am - 8:10am 10 mins
Registration and Coffee
8:10am - 8:15am 5 mins
Delivery and Targeting Strategies
Chairperson’s Remarks
  • Robert Gronke, PhD - Senior Principal Scientist, Technical Development, Biogen, Inc
more
8:15am - 8:45am 30 mins
Delivery and Targeting Strategies
R&D Trend of Oligonucleotide Therapeutics in Japan
  • Aya Kubota - Deputy Editor, Nikkei Business Publications, Inc.
more

In recent years, the R&D of Oligonucleotide Therapeutics has become very active in Japan. Key players from not only pharmaceutical companies but also venture companies and academic researchers moving towards practical applications have appeared. This presentation will discuss the trends, prospects and expectations for for R&D activity of oligonucleotide therapeutics from the viewpoint of journalist.

8:45am - 9:15am 30 mins
Delivery and Targeting Strategies
Developing mRNA Therapeutics with LNP
  • James Heyes, PhD - Vice President, Drug Delivery, Arbutus Biopharma, Corp.
more

Arbutus’ Lipid Nanoparticle (LNP) platform is enabling several early and late stage clinical trials. They are designed to deliver their nucleic acid payloads to sites of disease and have been used to target both viral and endogenous gene targets. Here we describe their application to mRNA payloads.

9:15am - 9:45am 30 mins
Delivery and Targeting Strategies
Arrowhead’s TRM™ Delivery System – Potent, Modular and Versatile for RNAi
  • Bruce Given, MD - Chief Operating Officer, Arrowhead Research Corporation
more

Arrowhead’s new TRM™ delivery system is designed for RNAi trigger delivery to a variety of tissues, not just the liver.  We expect to be in human trials with this new technology in 2018 as we look to expand the boundaries of current RNAi efforts.

9:45am - 10:15am 30 mins
Delivery and Targeting Strategies
Enhancing ASO Potency in Extra-Hepatic Tissues
  • Alfred Chappell, PhD - Assistant Director, Structural Biology, IONIS Pharmaceuticals
more

Oligonucleotide therapeutics represent the third distinct platform for drug discovery in the pharmaceutical industry. Recent advances in targeted delivery have greatly enhanced the potency of oligonucleotide therapeutics for suppressing gene expression in hepatocytes. We have explored strategies to enhance potency of oligonucleotide therapeutics in extra-hepatic tissues such as muscle, endocrine organs and in lymphocytes, which will be presented

10:15am - 10:55am 40 mins
Networking Refreshment Break with Exhibit and Poster Viewing
10:55am - 11:00am 5 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Chairperson’s Remarks
  • Zicai Liang - Chairman and CEO, Suzhou Ribo Life Science Co. Ltd.
more
10:55am - 11:00am 5 mins
Peptide Track: Peptide Development and Peptide Delivery
Chairperson’s Remarks
  • Bruce Morimoto, PhD - Vice President, Scientific Affairs, Celerion, Inc.
more
11:00am - 11:30am 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Advancing LNA Therapeutics
  • Troels Koch, PhD - Vice President and Head of Research, RNA Therapeutics, Roche pRED, Roche Innovation Center Copenhagen
more

Recently therapeutic oligonucleotide discovery has seen a shift from paradigms based primarily upon sequence diversity, towards exploiting both sequence and design diversity: Small structural alterations can greatly affect the pharmacological properties of oligonucleotides. More recently, it has been realized that the specific stereo definition of phosphorothioate internucleoside linkages (PS) in LNA oligonucleotides are also strong pharmacological determinants. It will be shown in the presentation that new opportunities for oligonucleotide drug discovery are enabled when collective sets of diversity parameters are exploited.

11:00am - 11:30am 30 mins
Peptide Track: Peptide Development and Peptide Delivery
I/O, CBP501 Combo Clinical trial: Rationale and Updates
  • Takumi Kawabe, MD, PhD - President and CEO, CanBas Co., Ltd.
more

Update on CBP501, a 12aa d-type peptide. It binds calmodulin, increases cytotoxicity of platinum, induces tumor immunogenic cell death, suppresses M2 macrophages, reduces cancer stem cells, and suppresses epithelial-to-mesenchymal transition. A Phase Ib study for the triple drug combination of CBP501, cisplatin and anti-PD1 antibody is ongoing.

11:30am - 12:00pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Development of Cancer Peptide Vaccines: Can Cancer Peptide Vaccines Survive?
  • Yasuhide Uejima, PhD - General Manager, CMC & QA, BrightPath Biotherapeutics, Co., Ltd.
more
12:00pm - 12:30pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Asymmetric siRNA Targeting Fibrotic Disorders
  • Dong-Ki Lee, PhD - Professor / CEO, Sungkyunkwan University School of Medicine / OliX Pharmaceuticals
more

OLX10010, a cell-penetrating asymmetric siRNA (cp-asiRNA) targeting connective tissue growth factor (CTGF), effectively reduces target gene expression as well as expression of fibrotic markers in animal model study. Preclinical as well as clinical study update of OLX101 in anti-skin scar will be presented. In addition to skin scar, OLX101 has a potential to be developed as therapeutics targeting various fibrotic disorders. We will present animal proof-of-concept study result of OLX101 in other fibrotic diseases in lung and eye, such as idiopathic pulmonary fibrosis (IPF) and subretinal fibrosis.

12:00pm - 12:30pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Peptides as Medical Devices
  • Naoki Yamamoto, PhD - Business Development, 3D Matrix
more

3D Matrix has developed the self-assembling peptide platform technology and we successfully obtained the CE marking certificate for one surgical hemostatic agent based on the technology of PuraMatrix(r). In this presentation we will introduce our recent progress from the viewpoint of CMC.

12:30pm - 1:40pm 70 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Networking Luncheon with Exhibit and Poster Viewing
12:30pm - 1:40pm 70 mins
Peptide Track: Peptide Development and Peptide Delivery
Networking Luncheon with Exhibit and Poster Viewing
1:40pm - 1:45pm 5 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Chairperson’s Remarks
  • Torsten Hoffmann, Ph.D. - Chief Operating Officer, Silence Therapeutics
more
1:40pm - 1:45pm 5 mins
Peptide Track: Peptide Development and Peptide Delivery
Chairperson’s Remarks
  • Bruce Morimoto, PhD - Vice President, Scientific Affairs, Celerion, Inc.
more
1:45pm - 2:15pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Pushing RNA Targeting Drug Development in China towards Clinical Stages
  • Zicai Liang - Chairman and CEO, Suzhou Ribo Life Science Co. Ltd.
more

Over the years we have established a comprehensive platform for siRNA drug development in Kunshan, China and through our intramural research and high end collaborations with world leaders in the sector such as Ionis and Quark, we have been able to not only add the antisense dimension to our drug pipeline, but also push the first siRNA drug into the clinical stage. This combinatoric strategy will allow as to advance 3-4 RNA targeting drugs into IND or clinical stages over the next 12-18 months.

1:45pm - 2:15pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Exploring Stapled Peptide Permeability Using p53 As a Model System
  • Anthony Partridge, PhD - Principal Scientist, Early Discovery Pharmacology, Merck, Sharp & Dohme
more

Stapled peptides can inhibit intracellular protein-protein interactions to address “yet-to-be-drugged” therapeutic targets. However, their pipeline advancement is hindered by suboptimal membrane permeability. Using cell-active sequences derived from p53, we have generated a library of several hundred stapled peptides to understand how peptide properties dictate membrane permeability. We will describe insights gained and novel screening tools.

2:15pm - 2:45pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Recent Advances in RNAi Therapeutics at Arrowhead Pharmaceutical
  • Zhen Li, PhD - Senior Vice President, Chemistry and Manufacturing, Arrowhead Pharmaceuticals
more

In the presentation, Arrowhead Pharmaceutical’s new hepatic delivery platform TRiM will be presented. Recent in vivo data in rodents and NHP for ARO-AAT and ARO-HBV will be discussed. ARO-AAT is for the treatment for a genetic disorder, alpha-1 antitrypsin deficiency (AATD), and ARO-HBV is for the treatment of hepatitis B. DMPK data in these two programs will also be disclosed.

2:15pm - 2:45pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Applications of Biodegradable Silica in Parenteral Delivery of Peptides
  • Lasse Leino, PhD - Chief Executive Officer, Adjunct Professor, DelSiTech Ltd.
more

Biodegradable silica polymer matrix provides a versatile tool for controlled release of parenteral therapeutics. Peptides and proteins are easily and effectively encapsulated in nanoporous silica using the sol gel process which preserves the biological activity of these molecules. Because the drug release is based on adjustable matrix surface erosion, very accurate zero-order release profiles can be obtained. Using this technology, it is possible to administer peptides in a controlled manner even for several months.

2:45pm - 3:15pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Antibody-Mediated Delivery of siRNAs to the Muscle
  • Tsukasa Sugo, PhD - Principal Scientist, Takeda Pharmaceutical Co. Ltd.
more

We explored the applicability of antibodies as a targeting ligand of siRNAs. In a mouse model of Peripheral Artery Disease (PAD), which is a kind of muscle wasting disease, the treatment with antibody conjugated myostatin-targeting siRNA by intramuscular injection resulted in significant silencing of myostatin and hypertrophy of the gastrocnemius without any changes in the body or heart weight, which was translated into the recovery of running performance. This is the first data that myostatin inhibition therapy caused an improvement in running performance in PAD mice.

2:45pm - 3:15pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Peptide Delivery: How Nanosystems Can Help Address Present and Future Challenges
  • Joel Richard, PhD - Senior Vice President, Peptides Development, Head of Pharm. Dev. Dreux Site, IPSEN
more

Peptides have become very attractive drugs in the last decades, due to their selectivity, their high bioactivity and low toxicity. These drugs have been successfully developed for the treatment of major diseases like type 2 diabetes and cardiovascular disorders, various types of cancer and multiple sclerosis. Due to their poor stability in extreme pH conditions, their enzymatic degradation and poor absorption across epithelial membranes, as well as their short plasma half-life, peptides remain difficult-to-administer drugs. At the present time, they are predominantly administered via injection, using sustained-release (SR) formulations mainly based on polymer matrices slowly releasing the peptide over months. These formulations have become the most successful injectable peptide formulations on the market. However, the use of alternative routes of administration, like the oral route or the transmucosal route, is likely to increase in the future, due to the pain and invasiveness of injections, as well as disposal issues associated with used needles and relatively complicated injection protocols. Low bioavailability due to limited permeability through the membranes remain a key challenge for these alternative delivery routes. In addition, new challenges have emerged recently, related to the need for intracellular delivery of peptides to new targets in cancer treatment and to the crossing of the blood-brain barrier (BBB) for peptide delivery to the brain. Then, in this context, nanodelivery systems (e.g. nanotubes, nanoparticles or nanocapsules) can provide appropriate solutions to address present and future challenges of peptide delivery, especially as regards SR formulations and delivery systems crossing cellular membranes (either intestinal epithelium or BBB) or entering cells to target intracellular receptors. This paper will present various successful nanosystems for peptide delivery that have entered the clinic or even progressed to the market, and discuss prospective approaches mainly focused on the crossing of membranes.

3:15pm - 3:45pm 30 mins
Oligonucleotide Track: Oligonucleotide Case Studies
Networking Refreshment Break
3:15pm - 3:45pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Networking Refreshment Break with Exhibit and Poster Viewing
3:45pm - 4:15pm 30 mins
Oligonucleotide Track: Oligonucleotide CMC Strategies
AJIPHASE® Process Development Focusing on Impurity Suppression
  • Taisuke Ichimaru - Researcher, Bio-functional Molecular Chemistry Group, Ajinomoto Co. Inc.
more

To address the rising demand of oligonucleotide drugs, we have been developing a unique and novel solution-phase synthesis, AJIPHASE®. The significant difference between AJIPHASE® technology and solid-phase synthesis is that our technology can show the reaction detail in each step by direct analysis. This presentation will highlight the elucidation of side reaction in AJIPHASE® and suppression of the impurities to obtain high purity oligonucleotides.

3:45pm - 4:15pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Update on Advances in Oral Delivery of Peptides
  • Christopher Rhodes, PhD - President and CEO, Drug Delivery Experts
more

The various technologies and strategies for oral delivery of peptides will be discussed.

In addition, data available from literature examples will be used to provide concrete examples for the pharmacokinetics profile achieved with each technology. Finally, new technologies that show some promise for oral delivery will be discussed.

4:15pm - 4:45pm 30 mins
Oligonucleotide Track: Oligonucleotide CMC Strategies
Case Story: What Does It Take to Integrate LNA Phosphoramidite Specialty Building Blocks into the Supply Chain?
  • Christoph Rosenbohm, PhD, MBA - Vice President and Head of Discovery Operations, Roche Innovation Center Copenhagen
more

The chemical complexity of oligonucleotides drug candidates has increased significantly over the past years. This puts a special focus on the quality of the novel starting materials that are chemically complex and are sources of impurities in the final API. In addition, all these factors put extra pressure on the design of a robust supply chain for the novel building blocks spanning from initiation to drug development.

4:15pm - 4:45pm 30 mins
Peptide Track: Peptide Development and Peptide Delivery
Functional Peptide-modified Exosomes for Intracellular Delivery of Therapeutic Molecules
  • Ikuhiko Nakase, PhD - Special Lecturer (Tenure Track Lecturer), Nanoscience and Nanotechnology Research Center, Osaka Prefecture University
more

Exosomes are naturally occurring nanomaterials and could potentially represent the next generation of biological tools for delivering therapeutic molecules. However, the low cellular targeting efficacy and insufficient release of exosomal contents inside the cytosol can hinder their application. I will be discussing novel techniques that were developed to effectively target receptors and enhance cytosolic release of exosomal contents via biofunctional peptides that were modified on exosomal membranes.

4:45pm - 4:50pm 5 mins
Close of Conference