Leaders in Pharmaceutical Business Intelligence (LPBI) Group

Gene editing, particularly using the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system, is now being extensively used as a research and functional screening tool in drug discovery. Cambridge Healthtech Institute’s second annual conference on Translational Gene Editing will bring together experts from all aspects of preclinical research, from early target discovery to drug delivery, to talk about the progress being made in gene editing and how it’s being applied. Learn about ways in which CRISPR/Cas9 is being used to identify targets, create relevant cell lines and in vivo disease models, set up functional screens, and for targeted drug delivery. What can you do to overcome some of the inherent challenges with design, delivery and off-target effects associated with CRISPR/Cas9? Hear from experts in pharma/biotech, academic and government labs who will share their experiences leveraging the utility of gene editing for diverse applications, particularly in oncology and immunotherapy.

PRELIMINARY AGENDA

Gene Editing for Screening Pathways and Drug Targets

CRISPR-Cas9 Whole Genome Screening: Going Where No Screen Has Gone Before
Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer 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

Cross-Species Synthetic Lethal Screens and Applications to Drug Discovery

Norbert Perrimon, Ph.D., Professor, Department of Genetics, Harvard Medical School and Investigator, Howard Hughes Medical Institute

Scouring the Non-Coding Genome by Saturating Edits

Daniel E Bauer M.D., Ph.D., Assistant Professor of Pediatrics, Harvard Medical School and Staff Physician in Pediatric Hematology/Oncology, Boston Children’s Hospital and Dana-Farber Cancer Institute, Principal Faculty, Harvard Stem Cell Institute

Building the CRISPR Toolbox

Optimized sgRNA Libraries for Genetic Screens With CRISPR-Cas9

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

Beyond Cas9: Discovering Single Effector CRISPR Tools

Jonathan Gootenberg, Member, Laboratories of Dr. Aviv Regev and Dr. Feng Zhang, Department of Systems Biology, Harvard Medical School, and Broad Institute of Harvard and MIT

CRISPR-Cas9 Genome Editing Improves Sub-cellular Localization Studies

Netanya Y. Spencer, M.D., Ph.D., Research Fellow in Medicine, Joslin Diabetes Center, Harvard Medical School

Improving Precision and Delivery

Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

Daniel G. Anderson, Ph.D., Professor, Department of Chemical Engineering, Institute for Medical Engineering & Science, Harvard-MIT Division of Health Sciences & Technology and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

Understanding and Translating In vivo Findings

Application of Genome Editing Tools to Model Human Genetics Findings in Drug Discovery

Myung Shin, Ph.D., Principal Scientist, Biology-Discovery, Genetics and Pharmacogenomics, Merck Research Laboratories

Translating CRISPR/Cas9 Into Novel Medicines

Alexandra Glucksmann., Ph.D., COO, Editas Medicine

Recommended Short Course*

SC8: A Primer to Gene Editing: Tools and Application – Learn More

Instructors:

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

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

-Stephanie Mohr, Ph.D., Lecturer, Genetics & Director of the Drosophila RNAi Screening Center, Harvard Medical School

– Claire Yanhui Hu, Ph.D, Senior Bioinformatician, Drosophila RNAi Screening Center, Department of Genetics, Harvard Medical School

The course will help the novice understand the basics of how gene editing works, what tools are available for use and how those tools differ from each other. For the expert, this course will offer details on the CRISPR technology, how to set up CRISPR-based screens and complement it with existing RNAi-based screens using proper analysis and follow-up studies. The instructors will also cover the use of gene editing in drug discovery and disease modeling and best practices for design and workflows when working with other model systems, besides mammalian cells.

*Separate Registration Required

For questions or suggestions about the meeting, please contact:

Tanuja Koppal, Ph.D.

Conference Director

Cambridge Healthtech Institute

T: (+1) 973-525-4667

E: tkoppal@healthtech.com

For sponsorship and exhibit sales information including sponsored podium presentations, contact:

Joseph Vacca

Associate Director, Business Development

Cambridge Healthtech Institute

T: (+1) 781-972-5431

E: jvacca@healthtech.com

For more information visit

WorldPreclinicalCongress.com/Gene-Editing