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

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.

Examples of Clinical and Anatomic Toxicopathology Findings Observed as Class-Effects Following Systemic and Intrathecal Administration of Oligonucleotides in Preclinical Safety Studies
Kuldeep Singh, PhD, Senior Director & Head Pathology, Wave Life Sciences

ADME and PK/PD Considerations for Preclinical Development of Antisense Oligonucleotides (ASOs)
Xiao Shelley Hu, PhD, Vice President, Head of DMPK and Clinical Pharmacology, Wave Life Sciences
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.

INSTRUCTOR BIOGRAPHIES:

Xiao Shelley Hu, PhD, Vice President, Head of DMPK and Clinical Pharmacology, Wave Life Sciences

Dr. Xiao Shelley Hu, PhD, is a Vice President at Wave Life Sciences in Boston, currently the head of the DMPK and Clinical Pharmacology department. Her group is responsible for ADME, PK/PD, and Pharmacometric studies in preclinical and clinical stages in neuromuscular, CNS, and hepatic diseases. Prior to joining Wave Life Sciences, she was a Director at Akebia Therapeutics in Boston, leading the Clinical Pharmacology, Pharmacometrics and Bioanalytical function. Shelley joined Akebia from Biogen, where her responsibility evolved from pre-clinical DMPK to Clinical Pharmacology, and later to Pharmacometrics for both small molecules and large molecules from discovery to post-market in Neurology, Rheumatology, Oncology, and Immunology. Shelley received her PhD in Pharmaceutical Sciences from the Ohio State University, MS in Pharmaceutical Sciences from Ohio State University, MS in Environmental Chemistry from Chinese Academy of Sciences, and BS in Pharmaceutical Sciences from Peking University Health Science Center. She has fourteen publications in peer-reviewed journals and one book chapter and serves as reviewers for various journals.

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.

Kuldeep Singh, PhD, Senior Director & Head Pathology, Wave Life Sciences

Kuldeep Singh is currently the Head of Pathology at Wave Life Sciences. He obtained his BVSc (Bachelor of Veterinary Sciences) from G.B. Pant University of Agriculture and Technology in India, MS from Utrecht University, The Netherlands, and PhD and residency in anatomic pathology from Oklahoma State University. He joined the American College of Veterinary Pathologists as a diplomate in 2009. Prior to joining Wave Life Sciences, he worked as an Investigative Pathologist at Sanofi and as a Director of Toxicologic Pathology at WuXiApptech. He served the University of Illinois as Head of Anatomic Pathology and Clinical Associate Professor in the College of Veterinary Medicine, Urbana-Champaign. His professional interests include oligonucleotides, toxicology, biomarker identification, safety assessment, animal model characterization, and investigative and digital pathology. Kuldeep has authored or co-authored more than 55 peer-reviewed articles, book chapters, and more than 75 abstracts and posters. He is currently an ad hoc reviewer for 14 journals. Kuldeep has presented at multiple national and international conferences as an invited speaker. He is a primary or co-investigator on 2 patents (application filed) based on oligonucleotides.

SC2: Successful Regulatory Submission for a Complex Oligonucleotide

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。

Choose your language
English

早期割引き
最大 $400 OFF
12月31日までのお申込み
Present a Poster
Oligo Discovery & Delivery
Oligo CMC & Regulatory
mRNA Design & Delivery
Emerging Oligo Modalities

会議の詳細はこちらをご参照ください