Conference Documentation: RNAi, miRNA & siRNA

Published by: SMI Publishing, Ltd

Published: Jun. 28, 2011

Table of Contents

DAY 1

8.30 Registration & Coffee

9.00 First Chair's Opening Remarks

Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals.

9.10 Single stranded antisense - today’s situation and the future’s potential

Overview of the RNAi industry

Current applications of siRNA, miRNA and oligonucleotides

Progress to make

Troels Koch, VP, Research Division, Santaris Pharma A/S.

9.45 RNAi: Developing a New Therapeutic Platform

Unique new modality of RNAi therapeutics

Challenges remaining in unlocking this unique potential

The need for clear demonstration of efficacy in well-controlled human clinical study

Translating the therapeutic potential of RNAi into clinical reality

Rachel Meyers, Vice President, Research and RLD , Alnylam Pharmaceuticals.

10.20 Opportunities and Challenges in siRNA Therapeutics Development

siRNA lead discovery and optimization

Lipid nanoparticle siRNA lead optimization

Biophysical and biochemical tools

Pharmacokinetics and pharmacodynamic modeling

Species differences in LNP performance

Mitigation of delivery vehicle toxicities

Laura Sepp-Lorenzino, Senior Director, Merck & Co.

10.55 Morning Coffee

11.15 PANEL DISCUSSION - The effect of pharma cuts on the further development and commercialisation of RNAi technologies

Pharma in RNAi and Oligonucleotides: Market overview

The immediate and lasting effect of 2009 cuts and 2010 cost repositioning by pharma

Internal v.s external development for pharma

Pharma expectations and Biotech’s capabilities

Progress for and potentials of alternative/parallel technologies and financing opportunities

Gary Carter, Market Research Director, Aligent Technologies.

Emma Hickman, Research Investigator, Novartis.

Troels Koch, VP, Research Division, Santaris Pharma A/S.

Rachel Meyers, Vice President, Research and RLD , Alnylam Pharmaceuticals.

Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals.

Laura Sepp-Lorenzino, Senior Director, Merck & Co.

12.00 Guanidino modified siRNAs and EPR for structure determination

Cationic siRNAs for stability and uptake

siRNA design rules

Hepatitis B virus inhibition

Spin labeled RNA for structure studies

RNA structure and RNase H accessibility

Joachim Engels, Professor, University of Frankfurt.

12.35 Overcoming a key challenge of RNAi Therapeutics: Effective Systemic Delivery of siRNA in Humans

Flexible and Scalable Manufacturing System

Highly Attractive Safety Profile

Flexibility Across Organ Systems

Expanding the Platform

Christopher Anzalone, CEO, Calando Pharmaceuticals.

1.10 Networking Lunch

2.10 Second Chair's Opening Remarks

Attila Seyhan, Head, RNAi and Compound Screens, Pfizer.

2.20 sd-rxRNA™ – New class of RNAi compounds: Chemistry, in vivo efficacy and preclinical development

Addressing the key challenge of RNAi therapeutic development: in vivo delivery

sd-rxRNA combine beneficial properties of RNAi and conventional antisense technologies, which enables robust cellular uptake and gene silencing

in vivo efficacy achieved using local administration of sd-rxRNA compounds

RXi’s dermal anti-scarring (anti-fibrotic) and ocular preclinical development programs

Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals.

2.55 Delivery and therapeutic potential of naked (non-formulated) siRNAs (local and systemic administrations)

Not all indications require specific siRNA formulations for effective delivery

Naked synthetic chemically stabilized siRNA can be efficiently delivered to the eye, inner ear, CNS, lung and kidney

Delivery of naked siRNA to these organs and tissues results in target gene knockdown and alleviation of disease

Elena Feinstein, Chief Scientific Officer, Quark Pharmaceuticals.

3.30 Nanoparticle Dicer Substrate RNA (DsiRNA) Delivery System to Achieve Target Knockdown in Orthotopic and Subcutaneous Tumor Models

Novel Dicer Substrate RNA-based therapeutics (DsiRNA) utilize an earlier entry step in the gene silencing process, namely the engagement of the enzyme Dicer

Compared to other RNAi approaches, this next generation RNAi strategy demonstrates greater potency and longer duration of action

DsiRNA payloads formulated in lipid nanoparticle delivery systems achieve >80% knockdown in mouse liver and >50% knockdown in Hep3B liver tumor models

Sujit Basu, Senior Director, Formulation, Dicerna Pharmaceuticals.

4.05 Afternoon Tea

4.25 Pulmonary delivery of siRNA - Prospects for therapeutic intervention in respiratory disease

Targeting epithelial expression in the lung with naked siRNA

Tailoring of the dosing strategy and the effect on siRNA distribution

Immune considerations in the lung

Challenges and future prospects

Emma Hickman, Research Investigator, Novartis.

5.00 Endosomolytic PepFect peptides for nucleic acid delivery

pH titratable PF6 peptide for ubiquitous siRNA transfections of primary cells in vitro and for systemic in vivo delivery.

A nuclear targeting peptide, PF14, for efficient delivery of splice switching oligonucleotides in disease models caused by aberrant alternative splicing

Solid formulations of PepFect peptides with nucleic acids

Samir EL-Andaloussi, Post Doc Researcher, Karolinska Institute.

5.35 Manufacture and control of oligonucleotides – a new challenge for CMC

An overview of the current oligonucleotide manufacturing landscape

Specific challenges for analytical control of oligonucleotides

The challenges for regulators and originators of oligonucleotide CMC filings

Prospects for the future

Mike Webb, Head API Chemistry & Analysis UK, GSK.

6.10 Second Chair’s Closing Remarks and Close of Day One

Attila Seyhan, Head, RNAi and Compound Screens, Pfizer.

DAY 2

8.30 Registration & Coffee

9.00 Chair's Opening Remarks

Sterghios Moschos, Principal Scientist, Pfizer.

9.10 Small RNAs as triggers for epigenetic control of gene expression

Small RNAs and regulation of gene expression in yeast and plants

Transcriptional gene silencing triggered by small RNAs in mammals

Transcriptional gene activation triggered by small RNAs in mammals

Possible mechanisms and roles for argonaute proteins

John Rossi, Dean, Graduate School of Biological Sciences , Beckman Research Institute City of Hope.

9.30 PANNEL DISCUSSION: Epigenetics in RNAi

What can epigenetics bring to RNAi study and therapeutic development?

Unexplored areas of fundamental biochemistry

Techniques and essential controls

Sterghios Moschos, Principal Scientist, Pfizer.

John Rossi, Dean, Graduate School of Biological Sciences , Beckman Research Institute City of Hope.

Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics.

10.10 Strategies for inhibiting the miRNA pathway

miRNA pathway introduction

Natural silencing of miRNAs - stress and oocytes

Inhibition by eliminating protein components of the pathway

Protein inhibitors

Inhibition by blocking miRNAs with modified oligonucleotides

Small-compound inhibitors

Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics.

10.45 Morning Coffee

11.05 A performing approach to identify biological active miRNAs

Lentiviral-based miRNA expression library in 2D matrix

Determination of miRNA function using high content screens

miRNA expression data base in human tissues

Gregoire Prevost, Chief Development Officer, InteRNA Technologies.

11.25 Role of miRNAs in angiogenesis - from mechansims to therapeutic apporaches

miRNA target identification in cardiovascular disease

Highthroughput identification of functional miRNAs in cardiovascular disease

Therapeutic manipulation of miRNAs in CV disease

Jan Fiedler , Institute of Molecular and Translational Therapeutic Strategies , Hannover Medical School.

12.00 Role of miRNAs and long intergenic non-coding RNAs (lincRNAs) in respiratory and inflammatory disease

miRNAs and the regulation of the innate immune response

miRNAs and asthma

Long non-coding RNAs and non-small cell lung cancer.

Eleni Tsitsiou, Research Associate, University of Manchester.

12.35 Networking Lunch

1.35 IP and Oligonucleotides

Patent species: microRNA, anti-miR, delivery, use, process

Construing patent claims

Breakthroughs versus increments

Long term issues with combination products

IP strategy - to win, you have to be in the game

Lorna Brazell, Partner, Bird & Bird.

2.10 Is silence still golden? Mapping the RNAi patent landscape

Despite recent turbulence within the RNAi field, the last couple of years have witnessed increasing patenting activity

A few corporate players dominate RNAi-related patenting but academia generates a large proportion of the IP within RNAi

Although crucial for many therapeutic applications, delivery only accounts for a small share of the IP

Key events have historically boosted RNAi patenting growth, making the outlook for the future interesting

Per Lundin, IP Attorney, Albihns Zacco AB.

2.50 TGF-ß gene silencing: experiences with competent authorities/regulatory bodies and clinical trials

Beginning clinical trials with gene silencing compounds.

Interactions with EMA, FDA and PMDA for starting clinical trials.

TGF-ß2 gene silencing in glioma, pancreatic carcinoma and malignant melanoma.

Local versus intravenous application of gene silencing compounds

Karl-Hermann Schlingensiepen, Chief Executive Officer, Antisense Pharma G Mb H.

3.25 Afternoon Tea

3.45 Computational support of potential RNAi therapeutics

Minimise siRNA off-target signalling

Network analysis of paired mRNA-miRNA datasets

Functional annotation of human miRNA

Paul Wilson, Bioinformatics Discovery and Analysis, GlaxoSmithKline.

4.20 RNA interference as a research and therapeutic tool…Has its time come?

A genome-wide RNAi screen identifies novel targets for neratinib sensitivity leading to neratinib and paclitaxel combination drug treatments

RNAi, principles, technological aspects, and applications

RNAi libraries for targeting HCV and SFV viruses

Multiplexed RNAi (optional)

Genome-wide RNAi screening for the Identification drug sensitizer genes and novel drugs for breast cancer

Attila Seyhan, Head, RNAi and Compound Screens, Pfizer.

4.55 Chair’s Closing Remarks and Close of Day Two

Sterghios Moschos, Principal Scientist, Pfizer.

Abstract

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