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Tuesday, June 29 • 2:00pm - 3:00pm
#243 SL: Epigenetic Drugs: Taking it to the Clinic- An Up-and-Coming Mode of Targeting Disease

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-21-579-L04-P; CME 1.00; IACET 1.00; RN 1.00

When we thinking about the genome, we think of the DNA double helix, and the 4 canonical nitrogenous bases. The epigenome encompasses chemical modifications of DNA bases, most commonly on cytosine, as well as chemical modifications of the histone proteins that compress nuclear DNA into chromatin. Understanding the functional significance of these modifications continues to be, an active area of research investigation, but broadly speaking, chemical modifications of the epigenome influence gene expression. As such, the ability to modify the epigenome is becoming an active area of therapeutic development. In this session will focus on a basic understanding of the epigenome, as well as two very different therapeutic strategies: one focusing on small molecule drugs, and the other using epigenome editing.

Learning Objectives

Describe the basic components of the epigenome, and functional consequences; Illustrate an example of the development of a drug to modulate the epigenome; Discuss strategies to adapt genome editing technology to modify the epigenome.


Philip (P.J.) Brooks, PhD


Epigenetic Drugs’ Challenging and Exciting Road to the Clinic
Sivakumar Gowrisankar, PhD

Epigenome Editing for Gene Therapy and Functional Genomics
Charles A. Gersbach, PhD

avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), NIH, United States
Philip John (P.J.) Brooks is a Program Director in the NCATS Office of Rare Diseases Research. He is also the Working Group Coordinator for the NIH Common Fund program on Somatic Cell Genome Editing. He earned his Ph.D. in neurobiology from the University of North Carolina at Chapel... Read More →
avatar for Sivakumar Gowrisankar

Sivakumar Gowrisankar

Director, Genomic Medicine, Parexel International, United States
Sivakumar Gowrisankar, PhD is a director in the translational medicine group at Parexel International. His work focusses on integrating genetics into drug discovery and development, including preclinical and all phases of clinical trials. He has a broad range of expertise in genetics... Read More →

Charles Gersbach

Professor of Biomedical Engineering, Duke University, United States
Dr. Charles A. Gersbach is the John W. Strohbehn Distinguished Professor of Biomedical Engineering at Duke University, the Director of the Duke Center for Biomolecular and Tissue Engineering, and the Director of the Duke Center for Advanced Genomic Technologies. His research interests... Read More →

Tuesday June 29, 2021 2:00pm - 3:00pm EDT
TBD Virtual Event Horsham, PA 19044
  06: PreClin Dev-EarlyPhaseCR, Session