短期课程

3月12日(二)  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.

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.

SC2: RNA Editing: Applications & Insights

RNA editing is a post-transcriptional RNA processing that is observed in different types of RNA moieties. There are different types of RNA editing observed which involves addition, deletion, or substitution of nucleotide bases. Programmable RNA editing can be used to correct disease-causing mutations and induce transient modulation of protein function, particularly for conditions where permanent genomic alterations are not desired. This course will offer an understanding into the cellular processes involved in RNA editing and offer insights into some of the challenges involved in using it for therapeutic development.

Instructors:

Tod Woolf, PhD, Executive Director of Technology Ventures, Beth Israel Deaconess Medical Center; Co-Founder, ETAGEN Pharma

Ronald Emeson, PhD, Joel G Hardman Chair in Pharmacology, Professor of Biochemistry, Molecular Physiology & Biophysics and Psychiatry & Behavioral Sciences, Vanderbilt University

Shanhu Hu, PhD, Director, Platform Biology, Korro Bio Inc.


Topics to be discussed include:

  • Introduction to different types of RNA editing
  • Understanding adenosine deaminase acting on RNA (ADAR) and other editing events
  • Tools for designing, delivering and enabling RNA editing
  • High throughput screening to study the impact of RNA editing in cells
  • Assays exploring the functional consequences of RNA editing for therapeutic development
  • Comparing technologies for assessing editing and off-target effects
  • Examples of how RNA editing has been used successfully

INSTRUCTOR BIOGRAPHIES:

Tod Woolf, PhD, Executive Director of Technology Ventures, Beth Israel Deaconess Medical Center; Co-Founder, ETAGEN Pharma

Tod Woolf leads the Technology Ventures Office at Beth Israel Deaconess Medical Center where he is a Lecturer at Harvard Medical School and co-founded ETAGEN in 2014. His team demonstrated the first BASE Editing (PNAS 1995). He was co-founder and CEO of Sequitur in 1996 where they developed NextGen Antisense, STEALTH RNAi, and MOD mRNA Therapeutics and vaccines in 1997. His team at ETAGEN developed Donor-Guides for CRISPR and single oligo genome editing technology that does not require programmable nucleases.

Ronald Emeson, PhD, Joel G Hardman Chair in Pharmacology, Professor of Biochemistry, Molecular Physiology & Biophysics and Psychiatry & Behavioral Sciences, Vanderbilt University

Ronald Emeson, PhD, serves as the Joel G. Hardman and Mary K. Parr Professor of Pharmacology at the Vanderbilt University School of Medicine. For the last three decades, his research group has focused upon the functional role(s) of adenosine-to-inosine (A-to-I) RNA editing events in transcripts encoding proteins critical for nervous system function including the GluA-2 subunit of the AMPA-subtype of ionotropic glutamate receptor, the alpha3-subunit of the GABA-A receptor, the Kv1.1-subtype of voltage-gated potassium channels, the 2C-subtype of serotonin receptor (5HT2C), calcium-dependent activator protein for secretion 1 (CAPS1), and ADAR2, one of two enzymes responsible for catalyzing A-to-I editing in vertebrates. While his work has significantly advanced our understanding of how RNA editing alters neurotransmitter receptor function, more recent studies in his laboratory have begun to examine the utility of using A-to-I editing as a therapeutic strategy to “repair” genetic mutations at the RNA level which underlie a variety of neurodegenerative and neurodevelopmental disorders.

Shanhu Hu, PhD, Director, Platform Biology, Korro Bio Inc.

Shanhu Hu, PhD is a Director, Head of High-Throughput Screening team at Korro Bio. Korro Bio focuses on the discovery and development of oligo-directed precision medicine that has the promise to address a myriad of previously undruggable targets. Korro’s platform OPERA leverages endogenous ADAR (Adenosine Deaminase Acting on RNA) to make targeted edits to a single RNA base, leading to reversible functional protein changes for targeted treatment of genetic diseases. Shanhu leads the HTS team to contribute to platform development and generate lead candidates for pipeline projects. Prior to Korro, Shanhu spent most of her professional time at Syros Pharmaceuticals where she led the discovery programs yielded CDK7 inhibitor SY-5609 and CDK12 inhibitor SY-12882. SY-5609 is currently in clinical evaluation for solid tumor. Shanhu Hu earned her PhD in pharmacology and toxicology from Dartmouth College.

* 活动内容有可能不事先告知作更动及调整。

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