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Informa
Nov 08
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09:00 - 09:10

Chairperson’s Opening Remarks

Showing of Streams
10:30 - 11:30

Morning Coffee and Networking

Showing of Streams
13:00 - 14:15

Lunch and Networking

Showing of Streams
15:45 - 16:15

Afternoon Coffee and Networking

Showing of Streams
17:15 - 17:20
End of Day 3

End of Day 3

09:00 - 09:10 10 mins
Chairperson’s Opening Remarks
09:10 - 09:50 40 mins
Opening Keynote Plenary Session
Critical Review of Oligonucleotide Therapeutics in the Clinic
more
  • Overview of oligonucleotide therapeutics in the clinic: Status and future projections
  • Number of oligos in different clinical phases
  • What compounds, disease indications and oligonucleotide product types are industry driving into the clinic?
  • Trends in clinical indications and patient population classes
  • What problems have been faced in the past for oligonucleotide clinical trials?
09:50 - 10:30 40 mins
Opening Keynote Plenary Session
Feedback from the FDA: Regulatory Applications of a Nonclinical Database for Oligonucleotide Therapeutics
10:30 - 11:30 60 mins
Morning Coffee and Networking
11:30 - 12:00 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Update on Safety Issues Emerging for Oligonucleotide Therapeutics
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·Update on safety issues emerging for oligonucleotide therapeutics
·Understanding the side effect of oligonucleotide drug classes: What are these, drug toxicity
·Strategies to optimise oligo safety in drug discovery and candidate selection

If you are interested in giving this presentation please contact catherine.marshall@knect365.com

11:30 - 12:00 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Impurity Qualification Strategy for Antisense Oligonucleotides
  • Yannick Fillon - Senior Scientist, Biogen
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Toxicology studies for antisense oligonucleotides are critical to establish control limits on drug substance impurities. This talk will describe how we evolved from separate impurity qualification toxicology studies with several test articles, to using a single engineered batch containing sufficient levels of all relevant impurities for the IND enabling tox study itself, circumventing the need for additional studies later in the program.

12:00 - 12:30 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
In Vitro And In Vivo Assessment Of Human Specific Oligonucleotides For Duchenne Muscular Dystrophy
  • Pietro Spitali - Assistant Professor, Leiden University Medical Center
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Duchenne muscular dystrophy is caused by protein truncating mutations, often out-of-frame deletions, which can often be corrected by exon skipping. I will present how we assess human specific oligonucleotides in vitro and in vivo and how the use of humanized mouse models can support and bridge the gap between preclinical and clinical development.

12:00 - 12:30 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Control and Qualification of Impurities for Oligonucleotides
  • Nadim Akhtar, PhD - Principal Scientist, AstraZeneca
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12:30 - 13:00 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
LNA, Antisense, Pre-Clinical Updates
  • Troels Koch, Ph.D., M.Sc. - Vice President and Head of Research, RNA Therapeutics, Roche pRED, Roche Innovation Center Copenhagen
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RNA therapeutics is a rapidly expanding field. The reasons are many, not least a particular focus on targets and tissues which are well validated for oligonucleotide drugs. Advances have also been made by introducing new oligonucleotide designs and chemistries. Targeting ligands conjugated to oligonucleotides for delivery purposes are now widely used, and with the development of sophisticated pharmacologically relevant assays, subtle variations in nucleoside and phosphorothioate chemical composition can result in significant drug improvements. The presentation will illustrate how disrupted discovery concepts, molecular modelling and bespoke tailoring of LNA oligonucleotide designs contribute to RNA therapeutic advancements.

12:30 - 13:00 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Analytical Techniques Applied for Quality Control in Oligonucleotide Manufacturing
  • A Representative from Avecia - -, Avecia
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13:00 - 14:15 75 mins
Lunch and Networking
14:15 - 14:45 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Preclinical and Clinical Updates on Oligonucleotides
  • Bill Marshall - CEO, MiRagen Therapeutics, USA
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14:15 - 14:45 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Analytical Method Development Strategies for Oligonucleotides
  • A Representative from Quality Assistance - -, Quality Assistance
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14:45 - 15:15 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
RNA Interference for Mass Markets: Groundbreaking Therapy for Cardiovascular Disease Brings Manufacturing Challenges
  • Clive Meanwell - Chief Executive Officer, The Medicines Company
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Short interfering RNA is rapidly emerging as a platform for new drugs. Most siRNA development compounds are aimed at rare diseases. One exception is inclisiran, an siRNA PCSK9 synthesis inhibitor in phase III trials to lower potentially harmful LDL-cholesterol. Phase II studies showed impressive effects with infrequent dosing and apposite safety data. Inclisiran aims at global markets in which tens of millions of patients need better protection against heart attacks and strokes. The opportunity to delay first heart attack or stroke by 30 years and to reduce recurrence by 30% with just one or two “flu shots” per year is ahead.

14:45 - 15:15 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Oligonucleotide Characterisation: Application of Mass Spectrometry
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If you are interested in giving this presentation please contact catherine.marshall@knect365.com

15:15 - 15:45 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Asymmetric siRNA Targeting Fibrotic and Ocular Disorders
  • Dong-Ki Lee, PhD - CEO, OliX Pharmaceuticals
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OLX10010, an anti-fibrotic 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 OLX10010 in anti-skin scar will be presented. In addition to skin scar, OLX10010 has a potential to be developed as therapeutics targeting various fibrotic disorders. We will present preclinical study data of OLX10010 in other fibrotic diseases in lung and eye, such as idiopathic pulmonary fibrosis (IPF) and subretinal fibrosis.

15:15 - 15:45 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Development and Application of New Analytical Methods and Technologies for Oligonucleotides
more
15:45 - 16:15 30 mins
Afternoon Coffee and Networking
16:15 - 16:45 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Spherical Nucleic Acids and Their Use in Treatment of Disease
  • David Giljohann - CEO, Exicure
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Spherical Nucleic Acids, or SNAs, are 3-dimensional confirmations of DNA or RNA wherein the architecture unlocks the potential of therapeutic oligonucleotides in a wide range of cells and tissues. SNA constructs overcome one of the most difficult obstacles to nucleic acid therapeutics: safe and effective delivery into cells and tissues. SNA constructs exhibit unparalleled transfection efficiency into numerous cell and tissue types including the skin without carriers or transfection agents. Moreover, SNAs can be used as potent immunotherapeutic agents for the treatment of cancer or infectious disease. Updates on their application in clinical will be presented.

16:15 - 16:45 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Optimising Analytical Method Development, Specification, Validation and Transfer for Oligonucleotides Therapeutics
  • Bianca Matthee - Head of Pharmaceutical Development, ProQR
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16:45 - 17:15 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Preclinical and Clinical Updates on Oligonucleotides
more

If you are interested in giving this presentation please contact catherine.marshall@knect365.com

16:45 - 17:15 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Future State of Collaborations for Oligonucleotides
  • Nadim Akhtar, PhD - Principal Scientist, AstraZeneca
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17:15 - 17:20 5 mins
End of Day 3