Pre-Conference, In-Person Dinner Short Course

Tuesday, March 12, 2024  5:30 - 8:00 pm

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. In addition, numerous mRNA and CRISPR therapeutic programs have entered clinical stages. Despite the common “nucleic acid” component, the mechanisms-of-action and of non-specific effects differ for each of these drug types.
Xiao Shelley Hu, PhD, Vice President, Head of DMPK and Clinical Pharmacology, Wave Life Sciences
Sarah Lamore, PhD, DABT, Senior Director, Toxicology, PepGen, Inc.
Kuldeep Singh, PhD, Senior Director & Head Pathology, Wave Life Sciences Inc.

Instructors:

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

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

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


Topics to be discussed include:

  • Different types of nucleic acid-based drugs
  • Mechanisms-of-action and non-specific effects
  • Current approaches to address non-specific and potentially toxic effects


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


  • Introduce and explain the differences among 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 acid drugs​

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, Inc.

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 Inc.

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.





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