Cambridge Healthtech Institute’s Second Annual

New Frontiers in Gene Editing

Striving for Better Design, Precision, and Efficiency

March 10-11, 2016 | Hilton San Francisco Union Square | San Francisco, CA
Part of the 23rd International Molecular Medicine Tri-Conference


Gene editing is rapidly progressing from being a research/screening tool to one that promises important applications downstream in drug development, cell therapy and bioprocessing. Cambridge Healthtech Institute’s second annual symposium on New Frontiers in Gene Editing will bring together experts from all aspects of basic science and clinical research to talk about the progress being made in gene editing and how it’s being applied. Knowing the strengths and limitations of the different tools, how does one decide when to use the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas system, as opposed to Transcription Activator-Like Effector Nucleases (TALENs), zinc finger nucleases (ZFNs) and other systems? What is being done to overcome some of the inherent challenges with design, delivery and off-target effects, associated with each of these techniques? Experts from pharma/biotech, academic and government labs will share their experiences leveraging the utility of gene editing for diverse applications.

Preliminary Agenda


Activities and Applications of Neisseria meningitidis Cas9

Erik Sontheimer, Ph.D., Professor, RNA Therapeutics Institute and the Program in Molecular Medicine, University of Massachusetts Medical School

Engineered Nucleases for Targeted Genome Integration

Pablo Perez-Pinera, M.D., Ph.D., Assistant Professor, Department of Bioengineering, University of Illinois at Urbana-Champaign

In vivo Genome Engineering Using S. aureus Cas9: Development and Applications

Winston Yan, Graduate Student, M.D.-Ph.D. Program, Laboratory of Dr. Feng Zhang, Broad Institute of MIT and Harvard

Presentation to be Announced


Therapeutic Genome Editing for Blood Diseases

Matthew Porteus, M.D., Ph.D., Associate Professor, Pediatrics, Stanford University School of Medicine

CRISPR Libraries for Functional Genomics: Optimizing On-Target Activity, Avoiding Off-Target Effects

John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT

Engineering Orthogonal Drug Switchable Precise Control for CRISPR Transcription Regulation

Xin (Cindy) Xiong, Ph.D., Research Scientist, Agenovir Corporation


Genome Edited Reporter Systems to Enable Cell-based HTS Assays for Chemical Biology and Drug Discovery

James Inglese, Ph.D., Head Assay Development & Screening Technologies, National Center for Advancing Translational Sciences and Adjunct Investigator National Human Genome Institute

Optimizing CRISPR-Cas9 System to Improve Genome-Wide Knockout Screening Performance

Haoquan Wu, Ph.D., Associate Professor, Department of Biomedical Sciences, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center

Parallel shRNA and CRISPR/Cas9 Screens Reveal Biology of Stress Pathways and Identify Novel Drug Targets

Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University

Strategies and Applications Using shRNA and CRISPR Technology for Identification of New Druggable Targets

Donald Apanovitch Ph.D., Director, Functional Genomics (Oncology), Pfizer Research

Recent Progress towards Efficient Targeted Gene Modification in Primary Human Hematopoietic Cells

David Rawlings, M.D., Director, Center for Immunity and Immunotherapies Seattle Children's Research Institute; Professor of Pediatrics and Immunology University of Washington School of Medicine


Precise Genome Engineering in Human iPS Cells to Model and Treat Disease

Bruce R. Conklin, M.D., Investigator, Roddenberry Center for Stem Cell Biology and Medicine, Gladstone Institutes and Professor, Division of Genomic Medicine University of California, San Francisco

Engineering Human Stem Cells by CRISPR

Su-Chun Zhang, M.D., Ph.D., Steenbock Professor in Behavioral and Neural Sciences and Professor of Neuroscience and Neurology Waisman Center, University of Wisconsin


Thursday, March 10, 6:30-9:00 pm

 SC26:  A Primer to Gene Editing: Tools and Applications

Course Instructors:
John Doench, Ph.D., Research Scientist, Broad Institute of Harvard and MIT

Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University

Premier Sponsors:  

Bina Technologies




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 Precision for Medicine 


Silicon Biosystems

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