- March 8, 2017
CRISPR/Cas9 Gene Editing In Vivo
FDA White Oak Campus, Building 2, Room 2047 (East-West)
Silver Spring, Maryland 20993
Thursday, March 9, 2017
8:30 am – 4:30 pm
The goal of the annual US-Japan cellular and gene therapy conference is to exchange ideas on cutting edge and diverse areas of biomedical research, and enhance opportunities for collaborations among scientists from the US and Japan. The conference is jointly supported by the Center for Biologics Evaluation and Research (CBER), US Food and Drug Administration (FDA); and the Ministry of Education, Culture, Sports, Science and Technology, Japan under the US-Japan Cooperative Research Program. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat)/Cas9 (CRISPR associated protein-9) is currently most versatile and precise method of gene manipulation. Six speakers from the US and five from Japan will discuss the advances and potential applications of CRISPR/Cas9-based genome editing system. The FDA speaker will present US regulatory perspective of gene editing technologies.
No prior registration is required. Attendance is free and open to the public. Visitors must enter through Building 1 where they will go through a security check for building entry. After entry visitors are requested to wait to be escorted to the meeting room. For more information, please contact S. Rafat Husain at firstname.lastname@example.org, (240) 402-9598 or (301) 335-6757 (BB).
The event can be viewed live via Adobe Connect at https://collaboration.fda.gov/japan/. Click the link and enter as a guest. Driving directions and parking information is available at
|Program Time||Program Topic|
|8:30 a.m. - 9:00 a.m.||
|9:00 a.m. 9:05 a.m.||
|9:05 a.m. 9:10 a.m.||
Yoshikazu Ohya, Professor, Department of Integrated Biosciences, Graduate School of Frontier Sciences, University of Tokyo, Tokyo
|9:10 a.m. 9:40 a.m.||
Raj K. Puri, Director, Division of Cellular and Gene Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland
CRISPR/Cas9 Mediated Genome Editing and its Application for the Study of Reproduction
Masahito Ikawa, Professor, Department of Experimental Genome Research, Research Institute for Microbial Diseases, Osaka University, Osaka
|9:40 a.m. 10:10 a.m.||
Regulatory Considerations for Gene Therapy Products involving Gene Editing Technologies
|10:10 a.m. 10:30 a.m.||Coffee Break|
|10:30 a.m. 11:00 a.m.||
Generation of Knock-in Mice by CRISPR/Cas9 R Transfer into Fertilized Eggs
|11:00 a.m. 11:30 a.m.||
Gene Editing and Modern Genetics in Zebrafish to Understand Human Biology and Disease
|10:30 a.m. 12:00 p.m.||
Germ Cell-specific Conditional KO via Cas9-mediated Chimera Analyses
|12:00 p.m. 1:00 p.m.||LUNCH|
|1:00 p.m. 1:30 p.m.||
Masahito Ikawa, Professor, Department of Experimental Genome Research, Research Institute for Microbial Diseases, Osaka University, OsakaCynthia Dunbar, Head, Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung and Blood Institute, NIH
Use of Non-human Primate Models to Optimize the Safety and Efficacy of Hematopoietic Stem Cell Gene EditingCynthia Dunbar, Head, Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung and Blood Institute, NIH
|1:30 p.m. 2:00 p.m.||
CRISPR/Cas9-Mediated Genome Editing using an AAV8 Vector Improves Hemostasis in a Mouse Model of Hemophilia B
|2:00 p.m. 2:30 p.m.||
Specific Targeting at a Genomic Mutation to Eradicate Leukemic (stem) Cells: Fantasy to Feasibility?
|2:30 p.m. 3:00 p.m.||Coffee Break|
|3:00 p.m. 3:30 p.m||
Recent Development and Application of Genome Editing Tools and Methods
|3:30 p.m. 4:00 p.m.||
Optimizing Methodologies for Genome Editing in Livestock
|4:30 p.m. 5:00 p.m.||
Use of High Content Screening Approaches to Identify Neuroprotective Compounds for the Treatment of Glaucoma and Retinal Degeneration
Donald Zack, Professor of Ophthalmology, Co-director, Johns Hopkins Center for Stem Cells and Ocular Regenerative Medicine, Johns Hopkins University School of Medicine, Baltimore
Thank you for your participation!
S. Rafat Husain, Ph.D.
Raj K Puri, M.D., Ph.D.
Tumor Vaccines and Biotechnology Branch
Division of Cellular and Gene Therapies
WO52/72 Rm 3123, 10903 New Hampshire Av.
Silver Spring, MD 20993-0002
Ph. 240-402-9598; Fax 301-595-1093
Yoshikazu Ohya, Ph.D.
Professor, Division of Biosciences
Department of Integrated Biosciences
Graduate School of Frontier Sciences
University of Tokyo
Bldg. FSB-101, 5-1-5 Kashiwanoha
Kashiwa, Chiba Prefecture 277-8562
Acknowledgments: We appreciate the kind help of Dr. Akiko Suzuki and Dr. Rukmini Bhardwaj, Tumor Vaccines and Technology Branch, Division of Cellular and Gene Therapies, CBER in organizing the conference.