Conference
Event Title
22nd US-Japan Cellular and Gene Therapy Conference
March 7, 2019
- Date:
- March 7, 2019
- Time:
- 8:30 AM - 5:00 PM ET
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 [email protected] 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 [email protected] [email protected] |
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 [email protected] |