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Harnessing the Potential of Genome Editing for Drug Discovery: Translational Frontiers of in vitro and in vivo Applications

Harnessing the Potential of Genome Editing for Drug Discovery: Translational Frontiers of in vitro and in vivo Applications

Tuesday, February 24, 2015

The New York Academy of Sciences

Presented By


Until recently, the generation of precise alterations in mammalian genomes has been a challenging and cumbersome process. A rapid succession of technologies in the past several years has brought about a revolution in molecular biology: zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN) and then clustered regularly interspaced short palindromic repeats (CRISPR). Each successive advancement has brought us closer to a routine, bench-level method to modify endogenous genetic elements. Now proof-of-concepts abound and encompass exciting frontiers such as rapid animal model development in addition to knockout and knock-in human and mouse cell lines. More recently, the framework of TALEN and CRISPR has been extended to a vast array of applications including epigenetic modulation, stimulation of gene expression, and targeted repression of gene transcription. What do these advances mean for drug discovery and translational research? This symposium will cover the latest techniques and methodologies in genome editing and the potential they hold for therapeutic development. In particular, the union of CRISPR and TALEN with stem and iPS cell technologies is an avenue that holds promise in improving the physiological relevance of disease models. However, the application of TALEN and CRIPSR to drug discovery is still in a nascent stage and innovation will be a core theme as we explore opportunities and current limitations.

Call for Poster and Presentation Abstracts

Abstract submissions are invited for a poster session, and several abstracts will be selected for short 'Late-breaking abstract' presentations. For complete submission instructions, please send an e-mail to with the words "Abstract Information" in the subject line. The deadline for abstract submission is February 3, 2015.

*Networking Reception to follow.

This event will also be broadcast as a webinar, for which you can register here.

Please note: Transmission of presentations via the webinar is subject to individual consent by the speakers. Therefore, we cannot guarantee that every speaker's presentation will be broadcast in full via the webinar. To access all speakers' presentations in full, we invite you to attend the live event in New York City when possible.

Registration and Webinar Pricing

Student / Postdoc Member$15
Nonmember (Academia)$65
Nonmember (Corporate)$85
Nonmember (Non-profit)$65
Nonmember (Student / Postdoc / Resident / Fellow)$45

The Biochemical Pharmacology Discussion Group is proudly supported by

  • Pfizer
  • Pfizer
  • Merck
  • Pfizer
  • WilmerHale

Mission Partner support for the Frontiers of Science program provided by   Pfizer


* Presentation titles and times are subject to change.

February 24, 2015

8:00 AM

Registration and Continental Breakfast

9:00 AM

Introductory Remarks
Sonya Dougal, PhD, New York Academy of Sciences
Samuel Hasson, PhD, Pfizer

9:15 AM

CRISPR Technologies for Genome Editing and Gene Regulation
Lei (Stanley) Qi, PhD, University of California San Francisco

9:55 AM

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
Randall Platt, MSc, Massachusetts Institute of Technology

10:35 AM

Networking Coffee Break

11:00 AM

Genome Editing in Assay Design: The Synergy of qHTS, Endogenous Reporters, and Phenotypic Drug Discovery
James Inglese, PhD, National Center for Advancing Translational Sciences, NIH

11:40 AM

A CRISPR Way for Novel Target Discovery
Yi Yang, PhD, Novartis Institutes for Biomedical Research

12:20 PM

Networking Lunch Break and Poster Session

1:40 PM

Genome Editing: From Modeling Disease to Novel Therapeutics
Chad Cowan, PhD, Harvard University

Technical Session

2:20 PM

Inducible in vivo Genome Editing with CRISPR/Cas9
Lukas Dow, PhD, Weill Cornell Medical College

2:40 PM

Repurposing Endogenous Type I CRISPR-Cas Systems for Programmable Gene Repression
Michelle Luo, MS, North Carolina State University

3:00 PM

Networking Coffee Break

3:30 PM

Comparison of Large Deletions and Targeted Integration in Mouse and Rat Embryos Mediated by CRISPR/Cas9 and ZFNs
Evguenia Kouranova, MS, Sage Labs

3:50 PM

Zinc-finger Nuclease- and CRISPR-mediated Genome Editing in Plasmodium Falciparum Malaria Parasites
David Fidock, PhD, Columbia University Medical Center

4:10 PM

Defining and Improving the Specificities of CRISPR-Cas Nucleases
J. Keith Joung, MD, PhD, Massachusetts General Hospital

4:50 PM

Closing Remarks
Samuel Hasson, PhD, Pfizer, Cambridge, MA

5:00 PM

Networking Reception

6:00 PM




Mercedes Beyna, MS


Mercedes Beyna is a scientist in the Neuroscience Research Unit at Pfizer. Her research focuses on target identification and assay development in the areas of psychiatric as well as neurodegenerative disorders. Captivated by neuroscience, she has worked in the field for over 10 years, in both academic and industrial laboratory settings. Before joining pharmaceutical R&D, Mercedes held lab manager and senior lab technician positions at New York University (NYU). Mercedes attended Binghamton University, earning her undergraduate degree in Biology, and subsequently received her Master's Degree in Biology from NYU. As the Pfizer lead in the Biochemical Pharmacology Discussion Group at the New York Academy of Sciences, she enjoys developing interesting and educational symposia.

Mi Cai, PhD


Dr. Cai is currently a senior scientist in the Neuroscience Research Unit at Pfizer Inc.. Her research interests include identifying and validating novel drug targets in neurodegenerative diseases with functional genomics and genome editing technologies. Prior to joining Pfizer, Dr. Cai was a postdoctoral fellow at Novartis Institutes for Biomedical Research Inc., where her research focused on the development of genome editing technologies (ZFN, TALEN and CRISPR) and applied them to target validation and disease modeling. Specifically, Dr. Cai and her colleagues developed an integrated approach for identification and validation of drug targets by combining haploid screening, next-generation exon sequencing and CRISPR-Cas9 gene editing technologies. Dr. Cai obtained her Ph.D. degree in Molecular Cell Biology in 2012 from Washington University in St. Louis. During her PhD training in the laboratory of Dr. Kenneth M. Murphy, she used both in vitro ES cell differentiation and in vivo mouse models to dissect the transcriptional basis of cardiovascular and hematopoietic development. Dr. Cai is originally from Hubei, China and went Peking University for undergraduate study.

Sandra J. Engle, PhD


Sandra Engle received her Bachelor’s of Art Degree in Biology with an emphasis in human genetics and a minor in chemistry from Ball State University in Muncie, Indiana, in 1987. She obtained her PhD in 1995 in Medical and Molecular Genetics from Indiana University School of Medicine in Indianapolis, Indiana, and held post-doctoral fellowships at the University Of Cincinnati College Of Medicine, Cincinnati, Ohio. In 2001, Sandra moved to pharmaceutical research with a position in the Genetically Engineered Mouse Models Group in Aventis (now Sanofi) where she applied her skills to generating in vivo mouse models and in vitro mouse stem cell derived models. In 2004, she joined the Genetically Modified Models Research Center of Emphasis with Pfizer where she continued to generate in vivo and in vitro models. Currently, Sandra is an Associate Research Fellow at Pfizer where she leads the Pluripotent Stem Cell Biology, Genomic Editing and Molecular Biology Laboratory which focuses on the generation of human induced pluripotent stem cells, in vitro differentiation of stem cells to terminally differentiated cell types including neurons, cardiomyocytes, macrophage, and endothelial cells and the genetic modification of human stem cells.

Samuel Hasson, PhD


Sam Hasson is currently heading a lab focused on discovery technology and target exploration in neurodegenerative disease. The main efforts center on uniting microglial and mitochondrial biology, human data, and pharmacology with the frontiers of CRISPR/Cas9-mediated genome editing. He is utilizing his background in high throughput chemical genetics, assay design, and functional genomics to develop target space for CNS disorders. Prior to joining Pfizer, Sam was a Pharmacology Research Associate Fellow at the NIH with co-mentors Richard Youle (NINDS) and Jim Inglese (NCATS). His postdoctoral research focused on translating the emerging biology around PINK1/Parkin mediated mitochondrial quality control into large-scale drug discovery and functional genomics.

Sonya Dougal, PhD

The New York Academy of Sciences


Chad Cowan, PhD

Harvard University

Chad Cowan received his BA and BS, with honors, from the University of Kansas. He received his PhD, from the University of Texas Southwestern at Dallas, garnering the Nominata award for most outstanding thesis. He subsequently completed a Damon Runyon postdoctoral fellowship with Professor Douglas Melton at Harvard University. He was named a Stowers Medical Investigator in 2006 and in 2008, he became an Assistant Professor at Harvard University. He is currently an Associate Professor at Harvard University in the Department of Stem Cell and Regenerative Biology and Massachusetts General Hospital, with appointments in the Center for Regenerative Medicine, Cardiovascular Research Center and Center for Human Genetics Research. He is an Associate Member of the Broad Institute and a Principal Faculty member of the Harvard Stem Cell Institute where he directs the Diabetes Disease Program and the iPS Cell Core Facility. Professor Cowan has led or been a member of several large efforts to utilize stem cells to better understand disease, including the NHLBI’s Next Gen iPS Cell Project and the Progenitor Cell Biology Consortium. In 2013, Professor Cowan received a Transformative Research Award from the NIH to create isogenic human pluripotent stem cell-based models of human disease mutations. More recently, Professor Cowan has focused on using genome-editing tools as therapeutics and as a co-founder of CRISPR Therapeutics hopes to see these discoveries translated into treatments or cures.

James Inglese, PhD

National Center for Advancing Translational Sciences, NIH

Dr. Inglese directs the laboratory of Assay Development and Screening Technology within the National Center for Advancing Translational Sciences (NCATS) and is an Adjunct Investigator of the National Human Genome Research Institute (NHGRI). His post-doctoral training program has been recognized by disease foundations as an effective collaborative bridge to NCATS expertise and resources enabling a champion-driven effort in early stage translation. Prior to the formation of NCATS he co-founded the NIH Chemical Genomics Center (NCGC) acting as its Deputy Director. Dr. Inglese received his PhD in Organic Chemistry from the Pennsylvania State University and completed post-doctoral training in the laboratory of Prof. Robert J. Lefkowitz at Duke University Medical Center. Before coming to the NIH, Dr. Inglese led research teams at the Princeton-based biotech Pharmacopeia and Merck Research Laboratories. Dr. Inglese has contributed to over 150 publications and patents; his efforts on the early drug discovery process focus on efficient and enabling high throughput screening paradigms. Dr. Inglese is the Founding Editor of the journal, ASSAY and Drug Development Technologiesserving as Editor-in-Chief from 2002-2014. Dr. Inglese participates on the scientific advisory boards of several NIH-funded chemistry and screening centers and international chemical biology consortia.

J. Keith Joung, MD, PhD

Massachusetts General Hospital

J. Keith Joung is a leading innovator in the field of genome editing. He currently serves as associate chief of pathology for research and the Jim and Ann Orr Research Scholar at Massachusetts General Hospital (MGH) and is an associate professor of pathology at Harvard Medical School. He is also a member of the Center for Cancer Research and the Center for Computational and Integrative Biology at MGH. Dr. Joung has been a pioneer in the development of important technologies for targeted genome editing and epigenome editing of human cells. He has received numerous awards including an NIH Director’s Pioneer Award, an NIH Director’s Transformative Research Project R01 Award, the Jim and Ann Orr MGH Research Scholar Award, and election into the American Association of University Pathologists. He is a scientific co-founder of Editas Medicine, a company dedicated to the translation of genome editing technologies for therapy of human diseases.

Dr. Joung holds a PhD in genetics from Harvard University, an MD from Harvard Medical School and an AB in biochemical sciences from Harvard College.

Randall Platt, MSc

Massachusetts Institute of Technology

Randall Platt is a graduate student in Biological Engineering at MIT and the Broad Institute. Platt works with advisor Feng Zhangto develop and apply genome editing technologies (TALENs and CRISPR) towards elucidating the genetic and molecular basis of disease. His research empowers genome editing applications directly in vivo, which enables the rapid creation of versatile disease models and provides a novel platform for discovering new disease-associated genes and testing therapeutics. At MIT he is an NSF,McGovern, and Afeyan Fellow. He obtained a bachelor's degree from the University of Utah in biomedical engineering and chemistry, and as a Whitaker Fellow he obtained a master's degree from the Imperial College London in material science.

Lei (Stanley) Qi, PhD

University of California San Francisco

Dr. Lei Stanley Qi is an Assistant Professor of Bioengineering at Stanford University. He obtained his PhD in Bioengineering from the University of California, Berkeley, and performed independent research as a Systems Biology Fellow in the University of California, San Francisco. He has developed the CRISPR/Cas technology for targeted genome engineering in mammalian cells. His work has lead to a series of technologies for gene regulation and imaging, including CRISPR interference (CRISPRi) and genome-scale screening. He also worked in the field of Synthetic Biology, and has developed methods to generate synthetic noncoding RNA regulators of transcription, translation, and as molecular sensor for chemicals and intracellular proteins.

Yi Yang, PhD

Novartis Institutes for Biomedical Research

After graduating from Beijing Medical University (nowBeijing University Medical School), I came to the United States and studied the apolipoprotein B (apo B)mRNA editing machinery with Dr. Harold Smith at the University of Rochester where I received my Ph.D. in Biochemistry in 1997. I did my postdoctoral training in mousegenetics with Dr. Brain Seed at Harvard University/Massachusetts GeneralHospital. I joined Novartis Institutes for BioMedical Research in 2003 where I have spent over 11 years on developing genetic tools for modeling human diseases and identifying novel therapeutic targets.


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The Biochemical Pharmacology Discussion Group is proudly supported by

  • Pfizer
  • Pfizer
  • Merck
  • Pfizer
  • WilmerHale

Mission Partner support for the Frontiers of Science program provided by   Pfizer

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