ショートコース | 10th Annual OPT Congress Oligonucleotide & mRNA Therapeutics 2025 公式サイト

2025年コースのアジェンダ

2025年3月11日（火） 8:00 - 10:00 am

SC1: Safety & Toxicity of Nucleic Acids
SC1：核酸の安全性と毒性

Nucleic acid drugs continue to deliver on their promise to become a third therapeutic modality, in addition to small molecules and biologics. Several antisense oligonucleotide drugs have been on the market for some time, while the first RNAi approval was granted in 2018. Despite the common “nucleic acid” component, the mechanisms of action and of non-specific effects differ for each of these drug types.

Topics to be discussed include:

Different types of nucleic acid-based drugs
Mechanisms of actions and non-specific effects
Current approaches to address non-specific and potentially toxic effects
Findings secondary to class-effect of oligonucleotides

Aimed at both novice and advanced nucleic drug developers, the course will:

Introduce and explain the differences between various types of nucleic acid drugs
Summarize our current understanding of the origins of non-specific and potentially toxic effects
Provide direction on how to minimize the potential toxic effects of nucleic acids drugs
Provide an overview of DMPK considerations from a safety evaluation perspective

SHORT COURSE PRESENTATIONS:

Nonclinical Considerations for the Development of Oligonucleotide Therapeutics
Sarah Lamore, PhD, DABT, Senior Director, Toxicology, PepGen
This presentation will review some of the common toxicities associated with oligonucleotide administration, discuss the regulatory expectations for the nonclinical safety evaluation of oligonucleotide therapeutics in the context of the applicable guidance documents, and present case studies based on recently approved products.

Application of In Vitro Toxicity Assays in Preclinical Safety Assessment of Systemically and Intrathecally Administered Antisense Oligonucleotides (ASOs)
Fengjiao Zhang, PhD, Director, Toxicology, Preclinical & Clinical Discovery & Development Team, Wave Life Sciences

ADME and PK/PD Considerations for Preclinical Development of Antisense Oligonucleotides (ASOs)
Xiao Shelley Hu, PhD, President and Founder, Translational Consulting LLC
ASOs are an emerging class of medicines for unmet medical needs. The ADME and PK/PD entail specific considerations to support effective preclinical development. This talk will focus on factors relevant to preclinical development of ASOs.

Further Considerations for the Development of Complex Oligonucleotide-Based Therapeutics
Kristy Szretter, PhD, Scientific Director, Takeda Pharmaceutical
This presentation will build on the regulatory expectations for the nonclinical development of ASOs and GalNAc-siRNAs, and address the additional considerations for more complex oligonucleotide-based modalities (e.g., lipid nanoparticles, antibody-oligo conjugates, circular RNAs). Relevant guidance documents and case studies will be used to highlight the similarities and unique aspects of each nonclinical package.

INSTRUCTOR BIOGRAPHIES:

Xiao Shelley Hu, PhD, President and Founder, Translational Consulting LLC

Dr. Xiao Shelley Hu is a seasoned pharmaceutical scientist with over 20 years of experience driving innovation across multiple therapeutic areas. She earned her Ph.D. and M.S. in Pharmaceutics from The Ohio State University, an M.S. in Environmental Chemistry from the Chinese Academy of Sciences, and a B.S. in Pharmacy from Peking University. As the President and Founder of Translational Consulting LLC, Dr. Hu provides specialized expertise in DMPK, clinical pharmacology, pharmacometrics, and translational strategies to pharmaceutical and biotech companies. Her leadership experience includes serving as Vice President and Head of DMPK and Clinical Pharmacology at Wave Life Sciences, where she spearheaded regulatory filings and advanced global programs involving antisense oligonucleotides, siRNA, and A-to-I editors. Previously, at Akebia Therapeutics, she led Clinical Pharmacology and Bioanalytical Science Department, overseeing registrational studies leading to the approval for VAFSEO. During her tenure at Biogen, she played a pivotal role in the approval of PLEGRIDY and contributed to TECFIDERA world-wide approval. A published author and sought-after speaker, Dr. Hu is committed to leveraging her deep expertise to champion data-driven approaches and shape the future of personalized medicine.

Sarah Lamore, PhD, DABT, Senior Director, Toxicology, PepGen

Sarah was until recently Director of Toxicology and Head of Investigative Toxicology at Wave Life Sciences. Prior to joining Wave, Sarah was a Toxicologist at Biogen where she worked on several modalities including small molecules and antisense oligonucleotides. She did her postdoctoral training at AstraZeneca and then joined the company as a Discovery Safety Scientist. She holds a PhD in Pharmacology and Toxicology from University of Arizona and is a Diplomate of the American Board of Toxicology.

Kristy Szretter, PhD, Scientific Director, Takeda Pharmaceutical

Kristy Szretter is a Scientific Director in the Drug Safety Research and Evaluation department at Takeda. Prior to joining Takeda, she served as a nonclinical lead at a number of organizations in the biotech industry. With over a decade of experience in nonclinical development, she has supported early and clinical development of biologics, cell therapies, vaccines, and viral and non-viral gene therapies in oncology, autoimmunity, infectious disease, neurology, and rare disease indications. She holds a PhD in Immunology and Molecular Pathogenesis from Emory University and is a Diplomat of the American Board of Toxicology.

Fengjiao Zhang, PhD, DABT, Director, Toxicology, Preclinical & Clinical Discovery & Development Team, Wave Life Sciences

Fengjiao is Director of Toxicology in the Preclinical & Clinical Discovery & Development Team at Wave Life Sciences. Prior to joining Wave, Fengjiao served as Principal Investigator (PI) and Professor in Shenyang Pharmaceutical University with extensive Drug Discovery & Development experience. She did her postdoctoral training at University of Arizona. She holds a PhD in Pharmacology and Toxicology and is a Diplomate of the American Board of Toxicology.

SC2: Successful Regulatory Submission for a Complex Oligonucleotide
SC2：合成のオリゴヌクレオチドの承認申請を成功させるには

ICH guidelines have established clear expectations for the control strategy for synthetically manufactured medicines. Oligonucleotides fall into the synthetic category and yet their manufacture and control are very different compared to small molecules. In this short course we will look at the requirements for a control strategy combining starting material control, process understanding and final drug substance specifications and methods. With a common understanding in mind we will discuss how apply the control principles to therapeutic oligonucleotides from early development to registration.

Topics to be discussed include:

The principles of a control strategy from ICH guidance and what this means in practice
The challenges (and advantages) in applying the principles to oligonucleotides
Where to start in early development of oligonucleotides
How to get to a successful commercial control strategy for oligonucleotides

Benefits of attending include:

Understand the activities required to carry out and fund in order to achieve regulatory success for a therapeutic oligonucleotide
Understand the when to plan in relevant activities when initiating an oligonucleotide development project
Support decision-making from early development onwards
Support CDMO selection, development and financial planning
Understand the regulatory challenges and how to overcome them

INSTRUCTOR BIOGRAPHIES:

Mike Webb, PhD, Founder and CEO, Mike Webb Pharma; Former Vice President, API Chemistry & Analysis, GSK

Mike received his PhD from imperial college in London. He spent most of his career at GSK and its legacy companies primarily as an analytical scientist and then becoming the Vice President of Development Chemistry and Analysis for GSK in the UK. He was heavily involved in establishing initial GSK’s oligonucleotide CMC development efforts. Mike has edited 3 books on the analysis of pharmaceuticals, including one on the analysis of oligonucleotides. Since leaving GSK in 2016, he has been consulting with Big Pharma and Biotechnology companies in the development, analysis and manufacture of therapeutic oligonucleotides from pre-clinical to marketing submission.

* 不測の事態により、事前の予告なしにプログラムが変更される場合があります。

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