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Conference Documentation: RNA Therapeutics

Conference Documentation: RNA Therapeutics

Selective inhibition of gene expression by RNA interference (RNAi) and wider oligonucleotide methods hold the potential to address significant unmet medical needs with importance across major disease areas such as cancer, genetic disorders, cardiovascular disease and autoimmune disorders. Recent advances in small interfering RNA delivery and target selection provide unprecedented opportunities for clinical translation and positioning in the market place.

This conference offers the opportunity to engage with strategies for advancing RNAi–based therapies for rare and major diseases through devlopment and clinical trials to market, case study updates on approaches to optimising RNAi formulation and delivery and hear lessons learned through successful submissions from regulators.


Day 1
8:30
Registration & Coffee
9:00
Chairman's Opening Remarks
Claude Paul Malvy, Professor, Universite Paris Sud
9:10
Atu027: a novel RNAi therapeutic in oncology
Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics
9:40
RNAi therapeutics for eye conditions
Covadonga Paneda, R&D Manager, Sylentis
10:10
Morning Coffee
10:40
From Bench to Bedside – The evolution of mirror-image oligonucleotides as new therapeutic modalities
Sven Klußmann, Co-Founder & Chief Technology Officer, Noxxon Pharma
11:10
RNA activation in therapy
Nagy Habib, Chairman and Co-Founder, MiNA Therapeutics
11:40
CRISPR-Cas as a “can opener” for genome editing?! First insights into early translational work for human therapeutics
Rodger Novak, CEO, CRISPR Therapeutics
12:10
Exon skipping in Duchenne Muscular Dystrophy: a tale of two diseases
Giles Campion, Chief Medical Officer and SVP R&D, Prosensa Therapeutics
12:40
Networking Lunch
13:40
Hepatic targeting and avoidance of oligonucleotide drugs: What is the real story?
Bård Smedsrod, Chief Scientific Officer, D'Liver
14:10
The use of LNA/UNA-aptamers for anticancer and delivery purposes
Jesper Wengel, Professor, BioNEC Center Director, University of Southern Denmark
14:40
Transfection: Viral and synthetic techniques converge
Steffen Panzner, Managing Director, Lipocalyx
15:00
Delivering macromolecules to the right address - many hands make light work
Steven Hood, Director, Scinovo, GSK
15:40
Afternoon Tea
16:10
Protecting biotech inventions, how much for how little?
James Legg, Patent Attorney , Boult Wade Tennant
16:40
Biochemically modified messenger RNA encoding nucleases for in vivo gene correction of severe inherited lung diseases
Michael Kormann, Group Leader, Pediatric Infectiology and Immunology, Eberhard-Karls-University Tuebingen
17:10
Round Table Discussion –Developing RNA therapeutics for rare diseases
Giles Campion, Chief Medical Officer and SVP R&D, Prosensa Therapeutics
17:40
Chairman’s Closing Remarks and Close of Day One
Claude Paul Malvy, Professor, Universite Paris Sud
Day 2
8:30
Registration & Coffee
9:00
Chairman's Opening Remarks
Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics
9:10
RNA Therapeutics: The 3rd major engine of pharmaceutical value creation?
Dirk Haussecker, Consultant, RNA therapeutics
9:40
RNA Therapeutics: Unlocking the potential with locked nucleic acids
Bo Rode Hansen, VP, Drug Discovery & Alliance, Roche
10:10
Networking towards clinical application of antisense mediated exon skipping
Annemieke Aartsma-Rus, Associate Professor , Leiden University Medical Center
10:40
Morning Coffee
11:10
Tackling Intractable targets in oncology – progress towards viable therapeutic oligonucleotides platforms
David Blakey, Chief Scientist, AstraZeneca
11:40
Panel Debate - Collaborative working from academia to big pharma
Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics
12:20
Networking Lunch
13:30
In vivo RNAi and functional genomics
Victor Kotelianski, Director of the Centre for Research, Education and Innovation, Skolkovo Institute of Science and Technology
14:00
From RNA splicing to Eteplirsen trials: a drug for Duchenne muscular dystrophy (DMD)
Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics
14:30
Fusion-oncogene junctions in cancers: Delivery and specific targets for siRNAs
Claude Paul Malvy, Professor, Universite Paris Sud
15:00
Afternoon Tea
15:30
Taking siRNA beyond the liver: Multifunctional nano-complexes for targeted siRNA delivery to brain, lung and tumours
Stephen Hart, Professor in Molecular Genetics , UCL
16:00
Oligonucleotide therapies for neuromuscular disease
Matthew Wood, Professor of Neuroscience and Assistant Head of Research , University of Oxford
16:30
Chairman’s Closing Remarks and Close of Day Two
Joerg Kaufmann, Chief Scientific Officer, Silence Therapeutics

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