Vaccines, Blood & Biologics

22nd US-Japan Cellular and Gene Therapy Conference

Adeno-associated Virus-Mediated Gene Therapy

FDA White Oak Campus, Building 31, Great Room, 1503 A
Silver Spring, Maryland 20993

Thursday, March 7, 2019

8:30 am – 5:00 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 Japan and the US. 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.

Gene therapy is one of the frontiers of modern biomedicine. Adeno-associated virus (AAV)-mediated gene therapy is becoming a promising approach to treat a variety of human diseases. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector. The speakers from Japan and the US will discuss the most recent advances, key achievements and emerging issues in the AAV-mediated gene therapy. The FDA speaker will present US regulatory consideration for clinical development of AAV-based vectors.

No prior registration is required. Attendance is free and open to the public. For more information, please contact S. Rafat Husain at syed.husain@fda.hhs.gov or (240) 402-9598.

The event can be viewed live via Adobe Connect at https://collaboration.fda.gov/us-japan/. Click the link and enter as a guest. Driving directions and visitors parking information is available at
https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241748.htm

  PROGRAM
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, Office of Tissues and Advanced Therapies (OTAT), CBER, US Food and Drug Administration, Silver Spring, Maryland
9:00 a.m. – 9:05 a.m. Opening Remarks
Peter Marks
, Director, Center for Biologics Evaluation and Research (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, The University of Tokyo, Tokyo
  Morning Session
Moderators Shin-ichi Muramatsu, Professor, Division of Neurology, Center for Gene Therapy Research, Jichi Medical University, Shimotsuke & Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo,
Charles Venditti, Senior Investigator, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland
9:10 a.m. – 9:40 a.m. AAV Vectors for CNS and Liver Gene Therapy
Shin-ichi Muramatsu, Professor, Division of Neurology, Center for Gene Therapy Research, Jichi Medical University, Shimotsuke & Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo
9:40 a.m. – 10:10 a.m. Gene Therapy for Methylmalonic Acidemia (MMA) - Lessons from Patients and Mice
Charles Venditti, Senior Investigator, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, Maryland
10:10 a.m. – 10:40 a.m. Coffee Break
10:40 a.m. – 11:10 a.m. Regulatory Considerations for the Development of AAV-based Gene Therapy Vectors
Zenobia Taraporewala, Microbiologist, Gene Therapy Branch, Division of Cellular and Gene Therapies, OTAT, CBER, FDA, Silver Spring, Maryland
11:10 a.m. – 11:40 a.m. CRISPR-mediated Genome Engineering and its Application in Animal Models
Tomoji Mashimo, Professor, Genome Editing Research and Development Center, Institute of Experimental Animal Sciences, Graduate School of Medicine, Osaka University, Osaka
11:40 a.m. – 12:10 p.m. Role of Glycan Binding in AAV Transduction and Identification of AAVs with Terminal Glucose Binding Activities
John (Jay) Chiorini, Senior Investigator, Adeno-Associated Virus Biology Section, Molecular Physiology and Therapeutics Branch, National Institute of Dental and Craniofacial Research, NIH, Bethesda, Maryland
12:10 p.m. – 1:00 p.m. LUNCH
  Afternoon Session
Moderators Raj K. Puri, Director, Division of Cellular and Gene Therapies, OTAT, CBER, US Food and Drug Administration, Silver Spring, Maryland
Masahisa Katsuno, Professor, Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya
1:00 p.m. – 1:30 p.m. Structure and Engineering of the CRISPR-Cas9 Genome Editor Nucleases
Hiroshi Nishimasu, Associate Professor, Department of Biological Sciences, Graduate School of Science, The University of Tokyo, Tokyo
1:30 p.m. – 2:00 p.m. Ocular AAV-mediated Gene Transfer for Sustained Delivery of Therapeutic Proteins
Peter Campochiaro, Departments of Ophthalmology and Neuroscience, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland
2:00 p.m. – 2:30 p.m. TDP-43 Regulates Early-Phase Insulin Secretion from Pancreatic Beta Cells
Masahisa Katsuno, Professor, Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya
2:30 p.m. – 2:50 p.m. Coffee Break
2:50 p.m. – 3:20 p.m. AAV1 Mediated Gene Therapy for Cystic Fibrosis
Liudmila Cebotaru, Associate Professor, Departments of Medicine and Physiology, Johns Hopkins University, Baltimore, Maryland
3:20 p.m. – 3:50 p.m. miRNA–mediated Therapeutic Approaches for Neurodegenerative Diseases
Yu Miyazaki, Assistant professor, Department of RNA Biology and Neuroscience, Graduate School of Medicine, Osaka University, Osaka
3:50 p.m. – 4:20 p.m. Targeted Gene Expression Using AAV: Gene Therapy for Cardiovascular Disease
Prasad Konkalmatt, Assistant Professor, Department of Medicine, George Washington University, Washington DC
Thank you for your participation!
Organizers: 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

 

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