쇼트코스

3월12일(화) 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.
Padma Narayanan, PhD, Head, Preclinical Safety, Wave Life Sciences

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.

Padma Narayanan, PhD, Head, Preclinical Safety, Wave Life Sciences


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.

Padma Narayanan, PhD, Head, Preclinical Safety, Wave Life Sciences

PadmaKumar "Padma" Narayanan, BVSc (DVM equivalent), MS, PhD is currently Vice President, Head of Preclinical Development at Wave Life Sciences, Boston, MA. Padma has spent 24 years in the Pharmaceutical Industry, with previous stints as Senior Director and Global Head of Immunology and Translational Toxicology in the Nonclinical Safety Department at Janssen Pharmaceuticals R&D, La Jolla, CA, Executive Director of Pathology and Toxicology at Ionis Pharmaceuticals, Carlsbad, CA, Director of Cell Signaling and Immunotoxicology Group of Discovery Toxicology, Amgen, Inc, Seattle, WA, and Senior Pathologist, SmithKline Beecham/GlaxoSmithKline, Philadelphia, PA. He obtained his BVSc degree from College of Veterinary & Animal Sciences, Kerala, India; a Master’s in Veterinary Surgery from Madras Veterinary College, Tamil Nadu, India, a Ph.D. in Immunopharmacology from School of Veterinary Medicine, Purdue University, Indiana, USA and completed his post-doctoral training at the Life Sciences Division, Los Alamos National Laboratories, Los Alamos, NM. Padma not only leads and contributes to the overall strategic direction of preclinical development programs supporting development of novel therapeutics to combat neurodegenerative diseases, but is also deeply interested in understanding immune-mediated adverse events to drugs in nonhuman primates and its relevance in a clinical setting. Padma has co-authored more than 50 peer-reviewed publications, reviews, book chapters and has given numerous invited seminars at both national and international scientific meetings. Padma has co-chaired multiple continuing education courses, symposia and workshops at the annual Applied Pharmaceutical Toxicology, International Society for Advancement of Cytometry, and American Association of Pharmaceutical Sciences meetings, and was a past Chair of the Applied Pharmaceutical Toxicology Society.

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.
Paloma Giangrande, PhD, Independent Consultant
Shanhu Hu, PhD, Director, Platform Biology, Korro Bio Inc.

Instructors:

Paloma Giangrande, PhD, Independent Consultant

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


Topics to be covered 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
  • Exploring the functional consequences of RNA editing for therapeutic development
  • Examples of how RNA editing has been used successfully

INSTRUCTOR BIOGRAPHIES:

Paloma Giangrande, PhD, Independent Consultant

Dr. Giangrande was until recently the Vice President of Biology at Wave Life Sciences in Cambridge, MA and an Adjunct Professor of Internal Medicine at the University of Iowa. She obtained her Bachelor of Science degree in Biochemistry at Wheaton College, Norton, MA in 1994 and her PhD in Pharmacology and Cancer Biology at Duke University, Durham, NC in 1999. Her career has been dedicated to the investigation and development of RNA-based therapeutic approaches (including RNA aptamers, siRNAs, mRNA, RNA editing) for cancer, cardiovascular disease, rare diseases, and critical illness. As a consequence, Dr. Giangrande developed a deep understanding of these technologies, diseases, and associated disease mechanisms. She is an internationally recognized expert in oligonucleotide therapeutics and delivery. Towards this end, she was the first to demonstrate that RNA ligands (aptamers) can be used to deliver therapeutic siRNAs to target cells. Her 70+ publications and 9+ patents are a testament to this body of work and her commitment to the development of novel RNA-based therapies for many diseases.

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.

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2024년 컨퍼런스
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Oligo Discovery & Delivery

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