Vaccines, Blood & Biologics

The 20th US-Japan Cellular and Gene Therapy Conference

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 syed.husain@fda.hhs.gov, (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

http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241748.htm.

Program TimeProgram Topic
8:30 a.m. - 9:00 a.m.

Registration
Coffee and Breakfast

Moderator
S. Rafat Husain, Sr. Staff Scientist, Division of Cellular and Gene Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland

9:00 a.m. 9:05 a.m.

Opening Remarks
Wilson Bryan, Director, Office of Tissues and Advanced Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland

9:05 a.m. 9:10 a.m.

Yoshikazu Ohya, Professor, Department of Integrated Biosciences, Graduate School of Frontier Sciences, University of Tokyo, Tokyo

Morning Session

9:10 a.m. 9:40 a.m.

Moderators

Raj K. Puri, Director, Division of Cellular and Gene Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland
Yumiko Saga, Professor, Genetics Strains Research Center, National Institute of Genetics, Mishima

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
Denis Gavin, Chief, Gene Therapy Branch, Division of Cellular and Gene Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland

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
Satoru Takahashi, Professor, Department of Anatomy and Embryology and Laboratory Animal Resource Center, Faculty of Medicine, University of Tsukuba, Tsukuba

11:00 a.m. 11:30 a.m.

Gene Editing and Modern Genetics in Zebrafish to Understand Human Biology and Disease
Shawn Burgess, Senior Investigator, Genome Technology Branch, National Human Genome Research Institute, NIH, Bethesda, Maryland

10:30 a.m. 12:00 p.m.

Germ Cell-specific Conditional KO via Cas9-mediated Chimera Analyses
Yumika Saga
, Professor, Genetics Strains Research Center, National Institute of Genetics, Mishima

12:00 p.m. 1:00 p.m.LUNCH

Afternoon Session

1:00 p.m. 1:30 p.m.

Moderators

Masahito Ikawa, Professor, Department of Experimental Genome Research, Research Institute for Microbial Diseases, Osaka University, Osaka

Cynthia 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 Editing

Cynthia 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
Tsukasa Ohmori, Associate Professor, Department of Biochemistry, Jichi Medical University School of Medicine, Shimotsuke

2:00 p.m. 2:30 p.m.

Specific Targeting at a Genomic Mutation to Eradicate Leukemic (stem) Cells: Fantasy to Feasibility?
Linzhao Cheng, Professor of Medicine and Oncology, Associate Director for Basic Research, Division of Hematology in Department of Medicine; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore

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
Tetsushi Sakuma, Assistant Professor, Department of Mathematical and   Life Sciences, Graduate School of Science, Hiroshima University, Hiroshima

3:30 p.m. 4:00 p.m.

Optimizing Methodologies for Genome Editing in Livestock
Bhanu Telugu, Assistant Professor, Department of Animal and Avian Sciences, University of Maryland, College Park

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
syed.husain@fda.hhs.gov
raj.puri@fda.hhs.gov

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
Japan
ohya@k.u-tokyo.ac.jp

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.

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