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 Don’t miss essential insight on oligonucleotide discovery and development from this two-part webinar series.


 Recent years have seen a huge surge of interest in oligonucleotide therapeutics, fueled by major advances in our understanding of how to make such molecules stable, selective, and efficient as drug modalities. The field is now diverse in terms of therapeutic areas being addressed, biological processes being manipulated and delivery technologies employed.

This two-part webinar series explores recent developments in oligonucleotide chemistry, biology, DMPK, toxicological assessment, manufacture, and formulation. For more information on each session, click the links below:


Available OnDemand

Oligonucleotide Drug Discovery

  • Examine the synthesis and modification of oligonucleotides
  • Explore methods for determining pharmacokinetic parameters
  • Hear about state of the art discovery strategies

In this first session we will have presentations from experts involved in providing state of the art technical support to the discovery stages of an oligonucleotide therapeutic programme. We will discuss the synthesis and modification of oligonucleotides, design and execution of suitable in vitro and in vivo assays, and methods for determining pharmacokinetic parameters. We will also hear about state of the art discovery strategies with a case history from an exciting young biotech company employing oligonucleotides to create impactful therapeutics. Finally our expert panel will be available for a question and answer session.


Speakers

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Mark Edbrooke

Independent consultant

"Intro / Industry overview"


Mark Edbrooke is an Independent Scientific Consultant specialising in oligonucleotide therapeutics and macromolecule delivery. He worked for 20 years in GlaxoSmithKline (GSK) before joining AstraZeneca (AZ), working on therapeutic antisense, RNA interference, microRNA, and modified RNA. He now consults for numerous UK-based universities, US- and UK-based investment companies, UK- and non-UK biotech companies, and was involved in the early days of the Nucleic Acid Therapy Accelerator (NATA), a government-funded accelerator for nucleic acid therapeutics.

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Qiong Zhou

Executive Director, Discovery Biology, WuXi AppTec

"An integrated biology platform to support discovery of oligonucleotide drugs"


Dr. Zhou led the team to have developed an end-to-end biology platform for support of the discovery of oligonucleotides with capabilities to include design of sequences, assessment of in vitro and in vivo activity and delivery, evaluation of immunotox and other early toxicity in vitro assays, as well as analysis of off-target effect of oligonucleotides. The team collaborates with many clients and offers significant experiences in the discovery of oligonucleotide drugs. Dr. Qiong Zhou is also responsible for the HBV, viral vector and viral immunology platforms in WuXi DBU.

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Jun Zhou

Senior Principal Scientist, Associate Director, WuXi AppTec

"Oligonucleotide chemical modification and conjugation"


Dr. Jun Zhou leads the development of WuXi AppTec’s versatile oligonucleotide discovery chemistry platform, and has a proven track record in delivering an extensive collection of chemical modification and conjugation chemistries to enable discovery of advanced oligonucleotide therapeutics. Prior to joining WuXi AppTec, he conducted his postdoc research at Georgia State University and co-op training in medicinal chemistry at GlaxoSmithKline in RTP, North Carolina. Dr. Jun Zhou received his PhD in organic chemistry from East China Normal University in 2014.

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Nan Zhao

Principle scientist III, DMPK service department, WuXi AppTec

"Drug metabolism and pharmacokinetic strategies for oligonucleotide therapeutics"


Dr. Nan Zhao obtained a Ph.D. in Physics from Georgia State University. She previously worked for Covance and Stoke therapeutics, Inc, and has many years of experience in GLP bioanalysis and oligonucleotide therapeutics. Dr. Nan Zhao joined DMPK service department at WuXi AppTec in 2020, and she is responsible for the construction of multiple bioanalysis platforms for oligonucleotide and promotion of new capabilities.

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Rob Goodnow

VP Medicinal Chemistry, Stoke Therapeutics

"Optimization Strategies for ASOs Applied to Stoke’s TANGO Therapeutic Platform"


Robert Goodnow is vice president of Medicinal Chemistry at Stoke Therapeutics. In this role, Robert leads efforts to oligonucleotides that can be developed as potential new medicines for patients. Prior to joining Stoke, Robert was Vice President of Innovation Chemistry at Pharmaron. Robert’s research has focused on the implementation of several novel chemistry platform technologies, including the use of new DNA backbones as antisense agents and small molecule-targeted delivery of siRNA, for therapeutic applications in oncology, inflammation, and metabolic diseases. He has authored or co-authored more than 80 scientific papers, book chapters, and patent applications. Robert holds a B.S. in chemistry from Georgetown University and has a Ph.D. in natural products organic/bioorganic chemistry from Columbia University

Available OnDemand

Oligonucleotide Drug Development (CMC)

  • Explore synthesis strategies and analytical methodologies
  • Compare the advantages and disadvantages of different formulation strategies 
  • Review the latest CMC strategies and regulatory framework


In this second session, we will have presentations from experts involved in the manufacture and formulation of oligonucleotide therapeutics. We will discuss available synthesis strategies, analytical methodologies, and the advantages and disadvantages of different formulation strategies for delivering this class of molecule. We will also discuss CMC strategies and the regulatory framework with a presentation from a consultant supporting multiple biotech and pharma clients in this area. Finally, as in Part One, our expert panel will be available for a question and answer session.


Speakers

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Mark Edbrooke

Independent consultant

"Intro / Industry overview"


Mark Edbrooke is an Independent Scientific Consultant specialising in oligonucleotide therapeutics and macromolecule delivery. He worked for 20 years in GlaxoSmithKline (GSK) before joining AstraZeneca (AZ), working on therapeutic antisense, RNA interference, microRNA, and modified RNA. He now consults for numerous UK-based universities, US- and UK-based investment companies, UK- and non-UK biotech companies, and was involved in the early days of the Nucleic Acid Therapy Accelerator (NATA), a government-funded accelerator for nucleic acid therapeutics.

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William Fang

Vice President, WuXi STA, a WuXi AppTec Company

"WuXi STA’s Oligonucleotide CRDMO Platform"


William Fang, Vice President, Head of Oligo/Peptide Business at STA Pharmaceutical, a WuXi AppTec Company (WuXi STA). He has held various roles for over seven years with WuXi AppTec, accumulating experience in peptide R and D and manufacturing. Previously he worked at Zhejiang Runxin Pharmtech and Polypeptide labs. William received his bachelor’s degree from Beijing Medical University, Master’s degree at Creighton University and his MBA at UCLA.

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Lu Tian

Director, CMC Project Management, WuXi STA, a WuXi AppTec Company

"Oligo drug delivery and LNP platform at STA"


Dr. Lu Tian is currently the lead of LNP platform development and director of CMC project management at STA Pharmaceutical Co Ltd, a WuXi AppTec Company. Lu has been working on the drug delivery and oligo formulation development over 18 years at MIT, Abbott, Merck, and his own startup Innovform Therapeutics. To date, he has authored more than 10 scientific journal publications, 6 issued patents, and over 16 patent applications.

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Mike Webb

Independent consultant

"Challenges in CMC Development of Therapeutic Oligonucleotides"


Mike Webb obtained his BSc in Chemistry from the University of Essex, an MSc from Kingston University and PhD from Imperial College. He spent over 30 years with GSK and its legacy companies becoming VP for Development Chemistry and Analysis in the UK and setting up GSK’s first oligonucleotide chemistry team. Since leaving GSK 6 years ago, Mike has been consulting with Pharma and Biotech companies around the world on oligonucleotide CMC matters.

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