miRNA World, Boston, MA 28-30 October 2014

Sponsors:
RESEARCHED & DEVELOPED BY:
Discover the Pre-clinical, Diagnostic &
Therapeutic Application of miRNAs
EXPERT SPEAKERS INCLUDE:
Frank Slack
Professor, Molecular,
Cellular &
Developmental Biology
Yale School of Medicine
Carl Novina
Associate Professor,
Microbiology &
Immunology
Dana-Farber Cancer
Institute
David Rodman
Executive Vice
President, Research &
Development
miRagen Therapeutics
Sinil Kim
Chief Medical Officer,
Vice President, Oncology
miRNA Therapeutics
Roel Schaapveld
Chief Executive Officer
InteRNA Technologies
Pavan Kumar
Senior Scientist
Eisai
George Calin
Professor, Department
of Experimental
Therapeutics
MD Anderson Cancer
Center
Hungyun Lin
Principal Scientist
Pfizer
Anders Näär
Professor, Cell Biology
Harvard Medical School
Tel: +1 212 537 5898 | Email: info@hansonwade.com
www.micro-rna-world.com
October 28th-30th 2014
BOSTON, MA

miRNA World 2014 Boston | October 28th-30th
WELCOME TO miRNA World 2014:
Crucial Topics – The topics addressed and discussed at miRNA World
are generated by and for the miRNA community to reflect the most pressing
challenges and opportunities in this dynamic space. Tackling these topics in-
depth will allow you to maximize the potential of your miRNA research.
Expert Individuals – miRNA World 2014 brings together the key opinion
leaders across academia, pharma and biotech who are revolutionizing the
field of miRNA research. Learn from and network with these individuals and
discover what make this field tick.
Interactive Formats – Utilizing an array of dynamic interactive formats,
miRNA World cultivates in-depth discussions which will yield tangible results.
Take advantage as we break down the boundaries between institutions
and debate best practice in the key topics areas which are yet to settle on
definitive answers.
WHAT MAKES miRNA WORLD 2014
SPECIAL? CONTENTS
Speakers: 3
Conference Day One: 4-5
Conference Day Two: 6-7
Review the Workshop: 8
Sponsors: 8
The Venue
Pricing Options: 9
Harness the Clinical Potential of miRNAs
miRNA World 2014 will enable you to take advantage of the burgeoning
potential and applications of miRNAs by showcasing the leading
advancements in the field.
Research and development of the non-coding genome is rapidly
maturing and miRNAs are the leading light in this dynamic field. miRNA
World 2014 will bring you together with the experts who are shaping
this landscape and driving us into this new realm of discovery.
Unearth the therapeutic potential of miRNA Mimics and Anti-miR’s
by gaining first hand knowledge from those who are conducting
the leading pre-clinical and clinical trials with these compounds.
Realize the biomarker and diagnostic applications of miRNAs across a
spectrum of disease areas and witness how these are providing precise
tools in order to deliver more personalized patient care.
Replicate success from the leaders in the field as we:
1. Overcome the key therapeutic delivery challenges by blueprinting
your success from those who are breaking the delivery bottleneck
2. Accelerate your development of miRNA diagnostic tests and assays
through the application of exosomal and cellular miRNAs
3. Develop a comprehensive pharmacology strategy for miRNA
therapeutics through optimizing drug efficacy and safety
4. Construct a complete toolkit to maximize your research in the
burgeoning field of miRNA biomarkers
5. Bridge the knowledge gap between miRNA biogenesis and its effect
on clinical research and drug discovery
Leave miRNA World 2014 brimming with new ideas, tools and
connections to maximize the potential of your miRNA research.

SPEAKER FACULTY
It exceeded my expectations. The talks
were high quality and the interaction
among participants was excellent.
Genentech
SEARCH GROUPS FOR:
miRNA World 2014
to join the online community.
David Rodman
Executive Vice President,
Research Development
Pavan Kumar
Senior Scientist
Usha Singh
Senior Scientist Scientific
Lead
George Calin
Professor, Department of
Experimental Therapeutics
Hungyun Lin
Principal Scientist
Lorenzo Sempere
Assistant Professor, Center for
Translational Medicine
Quan Lu
Environmental Genetics
Pathophysiology
Anders Näär
Professor, Cell Biology
Gyongyi Szabo
Professor Vice Chair for
Research
Andrei Thomas-Tikhonenko
Professor Division Chief
Mark Feinberg
Medicine
Carl Novina
Microbiology and Immunology
Frank Slack
Professor, Molecular, Cellular
Developmental Biology
Sinil Kim
Chief Medical Officer, Vice
President, Oncology
Andrea Kasinski
Assistant Professor,
Biological Sciences
Robert Lee
Professor, Pharmaceutics
Elisa Romeo
Senior Director, Research
Development
Roel Schaapveld
Chief Executive Officer

CONFERENCE DAY ONE
8.00 Registration and Breakfast
9.00 Chair’s Opening Remarks
Andrea Kasinski, Assistant Professor,
Biological Sciences, PurdueUniversity
9.10 Conversations that Matter
Close your laptops, put down your cell phones and eavesdrop on exclusive one-on-
one interviews played out on stage with key opinion leaders in the field of miRNA
research. Through interactive dialogue, this will be your ONLY opportunity be part
of an exclusive conversation between those revolutionizing the miRNA field as they
discuss:
• What are the opportunities with miRNA’s as biomarkers?
• Where are we heading with miRNA’s as diagnostics?
• miRNA Anti-miR’s: What promise do these therapeutics hold?
• miRNA Mimics: What promise do these therapeutics hold?
• How much fundamental biology is left to uncover?
• What does the future of miRNA research look like?
10.00 Panel Discussion and Open QA:
What is the clinical potential of mirna’s in research and development and what
hurdles must be overcome to maximize this potential?
Frank Slack, Professor, Molecular,
Cellular Developmental Biology,
Yale School of Medicine
George Calin, Professor, Department of
Experimental Therapeutics,
MD Anderson Cancer Center
David Rodman, Executive Vice President,
Research Development, miRagen
Therapeutics
10.20 Enhanced miRNA Activity is a Novel Oncogenic Function
of eIF4F
• eIF4E, a component of eIF4F, is thought to be increased in cancers to sustain
increased growth.
• We show that increased eIF4E has oncogenic functions through enforcement of
microRNA activity.
Carl Novina, Associate Professor,
Microbiology Immunology,
Dana-Farber Cancer Institute
10.50 Speed Networking Morning Refreshments
miRNA Target Validation Clinical Biomarkers
11.50 miRNAs in Airway Smooth Muscle Proliferation
and Asthma
• Expression profiling of microRNAs in human airway smooth muscle (ASM).
• miR-10a controls ASM proliferation via targeting of PI3 kinase pathway.
• miR-10a as a therapeutic target to reduce ASM proliferation in asthma.
Quan Lu, Associate Professor,
Environmental Genetics
Pathophysiology, Harvard School of
Public Health
12.20 Circulating miRNAs as Potential Non-Invasive
Biomarkers in Patients of T Helper-Mediated Diseases
• Implications for the utilization of circulating miRNA profiles as clinical biomarkers
for T-helper associated associated disease detection and patient stratification.
• Considerations of miRNA profiling platform selections, sample preparation in
clinical specimens, and computational support of data analysis.
Hungyun Lin, Principal Scientist, Pfizer
29th OCTOBER 2014 Keynote Session Interactive Session Networking Session

12.50 Detection and Applications of miRNAs in Drug
Development
• Describe optimal conditions and automated detection method for the detection of
miRNAs in tissues.
• Advantages and limitations of implementing miRNAs as a biomarker in drug
development.
Usha Singh, Senior Scientist, Scientific
Lead, Merck
1.20 Poster Session Networking Lunch
miRNA-Based Diagnostics
2.20 Regulation of IGF1R Expression and Signaling by
Polymorphisms in 3’UTR in Neuroblastoma: Implications
for Therapy
• IGF1R expression levels correlate with 3’UTR SNPs, which map to putative miRNA
binding sites.
• Certain 3’UTR haplotypes correlate with more robust PI3K signaling in the face of
therapy, potentially leading to chemoresistance.
Andrei Thomas-Tikhonenko, Professor
Division Chief, Children’s Hospital of
Philadelphia
2.50 miRNAs: Biomarkers, Diagnostics and Beyond
• Discovery and clinical validation of miRNAs as biomarkers in the blood.
• Clinical laboratory product development and analytical validation.
Elisa Romeo, Senior Director, Research
Development, Gensignia
3.05 Circulating miRNAs for Alzheimer’s Disease: This
Promise and the Challenges
• Discovery and validation of circulating 7-miRNA signature for AD.
• Protocol optimization and longitudinal effect on signature miRNAs.
Pavan Kumar, Senior Scientist, Eisai
3.35 miRNA Tissue-Based Diagnostics: Where are we
Heading?
• in situ co-detection of microRNAs and proteins for high-content marker analysis at
single cell resolution.
• Implementation of fully automated pipeline from tissue slide to image analysis in
translational and clinical settings.
• Prognostic application of miR-21 expression in tumor stroma in triple negative
breast cancer and miR-34 expression in tumor epithelial in bladder cancer.
Lorenzo Sempere, Assistant Professor,
Center for Translational Medicine, Van
Andel Institute
4.05 Afternoon Refreshments
micro-Discussions
4.35 The miRNA World speaker faculty is second to none but there is just as much
knowledge in the audience as there is onstage. Discover multiple perspectives on
the key issues during roundtable discussions, specifically to facilitate peer-to-peer
learning.
• miRNA Normalization Goals Approaches – What approaches are out there and
what is generating the most success?
• Polymorphism of 3’UTR – What clinical potential does this region hold?
• Fundamental Understanding of miRNA Biology – Where are the gaps in our
understanding and what does this mean from a therapeutic stand-point?
• Exosomal miRNAs as Biomarkers - How important are these non-invasive
biomarkers and what are their clinical applications.
Moderators
Philadelphia
5.35 Close of Day 1

CONFERENCE DAY Two
9.00 Chair’s Opening Remarks
miRNA Therapeutic Delivery Methods
9.10 Development of the First microRNA Mimic in
Clinic, MRX34
• Screening and selection of miRNA-mimic delivery systems.
• Preclinical studies leading to first-in-human phase I study.
• Design of phase I study and the interim results of phase I study.
Sinil Kim, Chief Medical Officer,
Vice President, Oncology, MiRNA
Therapeutics
9.40 Delivery of a miRNA Mimic for Acute and Chronic
Inflammatory Disease
• Understand emerging miRNA mimic delivery strategies for targeting tissues and
cell types relevant in acute and chronic inflammatory disease states.
• Gain mechanistic insights into how systemic miRNA mimic delivery can alter
disease targets in a cell-specific manner.
Mark Feinberg, Associate Professor,
Medicine, Brigham and Women’s
Hospital
10.10 Interactive Solution Finding: Your Chance to Discuss
How to Master miRNA Mimic Delivery
Use this interactive session to make your voice heard on the challenges and
opportunities engulfing miRNA replacement delivery and crowd-source solutions to
the problems you are facing.
• How are we going to master the Mimic specific delivery challenges?
• How much is the delivery bottleneck holding back miRNA mimics and what
advantages do certain delivery methods have over others?
Sinil Kim, Chief Medical Officer,
Vice President, Oncology, MiRNA
Therapeutics
Mark Feinberg, Associate Professor,
Medicine, Brigham and Women’s
Hospital
10.40 Morning Refreshments
11.10 Targets and Approaches for miRNA-Based Therapies in
Liver Diseases
• The microRNA signature of liver damage and inflammation is different depending
on the type of liver disease.
• Kupffer cell activation, a central component of steatoheptitis, is regulated by
miRN-155 which is a feasible target for therapeutic approaches.
Gyongyi Szabo, Professor Vice
Chair for Research, University of
Massachusetts Medical School
11.40 A Functional Screening Approach to Identify miRNA
Drug Candidates
• Lentiviral-based miRNA expression library for high-throughput screening in cell-
based assays.
• Platform for identification miRNA biology.
• Chemical modification and systemic delivery of miRNA mimics.
Roel Schaapveld, Chief Executive Officer,
30th OCTOBER 2014 Keynote Session Interactive Session Networking Session

12.10 Interactive Solution Finding: Your Opportunity to Shape
How the Field is Going to Master miRNA Anti-miR
Delivery
This session will provide the platform for the field to break the Anti-miR delivery
bottleneck. Make your voice heard as we discuss with the experts:
• How are we going to master the miRNA-Anti-miR specific delivery challenges?
• What advantage do certain delivery methods hold over others?
• How much is this delivery bottleneck holding back the therapeutic potential of
Anti-miR’s?
• What does the future look like for viral vectors?
Gyongyi Szabo, Professor and Vice
Chair for Research, University of
Massachusetts Medical School
Roel Schaapveld, Chief Executive Officer,
12.40 Networking Lunch
Developing miRNA Therapeutics: Efficacy and Safety
1.40 MicroRNAs as Therapeutic Targets in Cardiometabolic
Diseases
• Identification and characterization of microRNAs contributing to cholesterol/lipid-
related diseases.
• Evaluation of efficacy and safety of microRNA-targeting LNA antisense
therapeutics in mice and non-human primates.
Anders Näär, Professor, Cell Biology,
2.10 Interactive Solution Finding: Your Chance to Debate
Whether There is an Optimal miRNA Therapeutic
Pharmacology Strategy
This interactive segment will debate what a desired pharmacological strategy for
miRNA therapeutics looks like and what progress is being made on this front.
Cultivate new ideas and innovative thoughts as to how your strategy can be optimized
to deliver effective miRNA therapeutics.
• Different chemistry platforms: Is this a one size fits all approach?
• How much are off-target effects affecting the clinical utility of miRNA
therapeutics?
• How can we maximize efficacy in a cost-effective manner?
Anders Näär, Professor, Cell Biology,
2.40 Afternoon Refreshments Networking
Delivering on the Promise of miRNA Therapeutics
3.10 micro-Discussion Intelligence and Open QA
Missed a roundtable you wished you were at? Couldn’t write down all the high level
discussion and opinion sharing? Fear not as roundtable moderators present the
industry intelligence around the most pressing issues in the miRNA therapeutics.
• What are the next moves with miRNA Therapeutics?
• What needs to be done next on the maximize the potential of this field?
• How can we implement key solutions to therapeutic challenges and what are the
key outcomes to take forward?
• What do we want to be talking about at miRNA World 2015?
Philadelphia
4.10 Close of Congress

CONFERENCE WORKSHOP
SPONSORS
miRNA World 2014 is proud to partner with:
Gensignia
Gensignia Life Sciences, Inc. is the US subsidiary of Gensignia Ltd, a privately held microRNA company specializing in microRNA
signatures as diagnostic tools with offices in London, operations in San Diego, and research in Milan.
Gensignia is developing proprietary molecular diagnostics designed to provide more accurate detection of cancer. In 2015, Gensignia
intends to introduce a lung cancer diagnostic test in the US through its clinical laboratory (CLIA).
www.gensignia.com
Currently, one of the biggest challenge facing miRNA therapeutics is delivery.
With a variety of delivery methods under development, the industry is still trying to adapt to
what is on offer and keep up with the latest advancements in this space. Both anti-miRs and
miRNA mimics possess their own set of challenges, but the delivery vesicles are applicable
to both. Just another reason why there is so much invested interest in this field.
This workshop will provide extensive levels of knowledge and detail on the current methods
available for miRNA therapeutic delivery. Using first-hand case studies, you will learn
what approaches work best for certain scenarios, what has already been learnt from
pre-clinical and clinical studies and what approaches are going to lead the field over
the next few years.
Leave this workshop with:
• A comprehensive understanding of the delivery methods that are showing
success in pre-clinical and clinical studies
• Extensive knowledge of how best to incorporate delivery methods into
research strategies
• The scientific and strategic foundations when considering what delivery
vehicles to apply in your research
Robert J. Lee, Ph.D., Professor of Pharmaceutics, is a member of the OSU Comprehensive
Cancer Center, the OSU Nanotechnology Center NSF-NSEC and a faculty at OSU College
of Pharmacy since 1997 (tenured since 2002). He is an expert on targeted nanoparticle
and liposomal drug delivery systems. He obtained his Ph.D. in Biochemistry at Purdue
University, worked as a Senior Scientist at GeneMedicine Inc. in 1995-1996 and then served
as Vice President of RD at Endocyte, Inc., where he was involved in the successful effort in
taking 111In-DTPA-folate into a Phase I/II clinical trial.
Workshop leader
Robert Lee
Professor of Pharmaceutics
Ohio State University
Mastering miRNA Therapeutic Delivery
Date: 28th October 2014 | Time: 2.00pm – 5.00pm

Code:4528
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Venue: The Westin Boston Waterfront Hotel
is a AAA Four Diamond award-winner
and is less than three miles from Logan
International Airport, as well as a short
cab, bus, water taxi, or T train ride away
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miRNA World, Boston, MA 28-30 October 2014

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miRNA World, Boston, MA 28-30 October 2014