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Nov 08
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09:00 - 09:10

Chairperson’s Opening Remarks

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Showing of Streams
10:30 - 11:30

Morning Coffee and Networking

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Showing of Streams
13:00 - 14:15

Lunch and Networking

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Showing of Streams
15:45 - 16:15

Afternoon Coffee and Networking

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

End of Day 3

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09:00 - 09:10 10 mins
Chairperson’s Opening Remarks
09:10 - 09:50 40 mins
Info
Opening Keynote Plenary Session
Critical Review of Oligonucleotide Therapeutics in the Clinic
  • 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
Info
Opening Keynote Plenary Session
RNA Interference for Mass Markets: Groundbreaking Therapy for Cardiovascular Disease Brings Manufacturing Challenges
  • Clive Meanwell - Chief Executive Officer, The Medicines Company

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.

10:30 - 11:30 60 mins
Morning Coffee and Networking
11:30 - 12:00 30 mins
Info
TRACK 1: Non-Clinical, Preclinical and Clinical Development
The Dose Makes All the Difference: Impact of Targeted Delivery on Appearance and Therapeutic Margins for the Typical 2’-MOE ASO Class Effects.
  • Scott Henry, PhD, D.A.B. - Vice President of Nonclinical Development, Ionis Pharmaceuticals, Inc.



11:30 - 12:00 30 mins
Info
TRACK 2: CMC and Analytical Method Development Strategies
Impurity qualification strategy for antisense oligonucleotides
  • Yannick Fillon - Senior Scientist, Biogen

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
Info
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

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 - Principal Scientist, AstraZeneca
12:30 - 13:00 30 mins
Info
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

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
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
14:15 - 14:45 30 mins
Info
TRACK 2: CMC and Analytical Method Development Strategies
Oligonucleotides: absolute quantification and P=O/P=S ratio in a single ICP-MS/MS run
  • Philippe De Raeve - Scientific Director, Quality Assistance

Oligonucleotide quantification by UV on basis of a calculated absorption coefficient lacks accuracy due to inaccuracy of the predicted coefficient and potential bias from other UV absorbing molecules.

A sensitive method was developed to determine the phosphorus content by ICP-MS, after microwave digestion of the sample: accurate quantification of oligonucleotide can then be obtained, knowing the fixed stoichiometry of phosphorus in the sample.

Sulfur may also be measured in the same run, allowing the simultaneous accurate determination of the P=O/P=S ratio, a very important parameter for the characterization of therapeutic phosphorothioates, that can only be measured by  31P-NMR, a technique not readily available in pharmaceutical labs.

14:45 - 15:15 30 mins
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Childhood Blindness due to a Photoreceptor Cilium Defect Treated with an Intravitreal Antisense Oligonucleotide (QR-110)
  • Pete Adamson - Sr VP Ophthalmology, ProQR Therapeutics
14:45 - 15:15 30 mins
Info
TRACK 2: CMC and Analytical Method Development Strategies
Oligonucleotide Characterisation: Application of Mass Spectrometry

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

15:15 - 15:45 30 mins
Info
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Asymmetric siRNA Targeting Fibrotic and Ocular Disorders
  • Sun Woo Hong - Director, OliX Pharmaceuticals

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
  • A Representative from Intertek - -, Intertek
15:45 - 16:15 30 mins
Afternoon Coffee and Networking
16:15 - 16:45 30 mins
Info
TRACK 1: Non-Clinical, Preclinical and Clinical Development
Spherical Nucleic Acids and Their Use in Treatment of Disease
  • David Giljohann - CEO, Exicure

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
Info
TRACK 2: CMC and Analytical Method Development Strategies
Evaluating the impact of sterilization on oligonucleotide based drug products
  • Erik van der Hage, PhD - Director Analytical Development & Quality Control, ProQR Therapeutics

Erik van der Hage and Vera Brinks

Sterile medicinal products can be sterilized in two ways, either via terminal methods on the product in its final container or via aseptic processing.

Recent studies on oligonucleotide based drug products evaluated the effects that different sterilization methods have on product quality. Physical and chemical properties such as rheology, color formation, particle formation, and degradation of the active ingredient were studied. The latest results and insights will be presented.

16:45 - 17:15 30 mins
TRACK 2: CMC and Analytical Method Development Strategies
Future State of Collaborations for Oligonucleotides
  • Rachel Orr - Senior Scientist, GlaxoSmithKline R&D
17:15 - 17:20 5 mins
End of Day 3