Vaccines, Blood & Biologics

21st US-Japan Cellular and Gene Therapy Conference

Neurodegenerative Diseases: Biology, Cellular and Gene Therapy

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

Thursday, March 1, 2018
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 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. Neurodegenerative diseases are incurable and debilitating conditions that result in progressive loss of structure or function of neurons, including death of neurons. Seven speakers from Japan and five from the US will discuss the growing challenges posed by these devastating diseases, therapeutic advances and barriers to progress in the research area. The FDA speaker will present US regulatory consideration for clinical development of cellular and gene therapies.

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
ModeratorS. 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
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, University of Tokyo, Tokyo    
 Morning Session
ModeratorsHideyuki Okano, Professor, Department of Physiology; and Dean of School of Medicine, Keio University School of Medicine, Tokyo
Steven Goldman, Chief, Division of Cell and Gene Therapies; Co-Director, Center for Translational Neuromedicine, University of Rochester Medical Center, Rochester, New York
9:10 a.m. – 9:50 a.m.Challenge toward iPSCs Based Cell Therapy and Modeling of CNS Disorders and Current Status of Regenerative Medicine in Japan
Hideyuki Okano, Professor, Department of Physiology; and Dean of School of Medicine, Keio University School of Medicine, Japan
9:50 a.m. – 10:20 a.m.Humanized Patient-Specific Glial Chimeric Mice for Modeling Neurodegenerative Disease
Steven Goldman, Chief, Division of Cell and Gene Therapies; Co-Director, Center for Translational Neuromedicine, University of Rochester Medical Center, Rochester, NY
10:20 a.m. – 10:40 a.m.Coffee Break
10:40 a.m. – 11:10 a.m.Neuroprotective Gene Therapy for Parkinson’s Disease
Hideki Mochizuki, Professor, Department of Neurology, Division of Medicine, Osaka University Graduate School of Medicine, Osaka
11:10 a.m. – 11:40 a.m.Enhancing Delivery of Cellular and Gene Therapy for Neurodegenerative Diseases with Focused Ultrasound
Paul Fishman, Professor of Neurology, University of Maryland School of Medicine, Baltimore, Maryland
11:40 a.m. – 12:10 p.m. Gene Therapy for Neurodegenerative Diseases
Shin-ichi Muramatsu, Professor, Division of Neurology, Jichi Medical University, Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo, Tokyo
12:10 p.m. – 1:00 p.m. LUNCH
 Afternoon Session
ModeratorsRaj K. Puri, Director, Division of Cellular and Gene Therapies, CBER, US Food and Drug Administration, Silver Spring, Maryland
Tadashi Isa, Professor, Department of Neuroscience, Graduate School of Medicine, Kyoto University, Kyoto
1:00 p.m. – 1:30 p.m.Clinical Development of Gene and Cellular Therapies
Mike Singer, Medical Officer, General Medicine Branch 2, Division of Clinical Evaluation and Pharmacology/Toxicology, CBER, US Food and Drug Administration, Silver Spring, Maryland
1:30 p.m. – 2:00 p.m.Multi-hierarchical Mechanism of Recovery from Partial Spinal Cord Injury
Tadashi Isa, Professor, Department of Neuroscience, Graduate School of Medicine, Kyoto University, Kyoto
2:00 p.m. – 2:30 p.m.Giant Axonal Neuropathy: From the Disease to Outcome Measures to First-in-Human Intrathecal Gene Transfer
Carsten Bonnemann, Senior Investigator and Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section, Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD
2:30 p.m. – 2:50 p.m.Coffee Break
2:50 p.m. – 3:20 p.m.New Molecularly Targeted Therapeutics Deduced from Unbiased Approaches
Hitoshi Okazawa, Professor, Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, Tokyo
3:20 p.m. – 3:50 p.m.GSK3 -b/a - Synuclein Interactions in the Genesis of Parkinson’s disease Anita Sidhu, Professor, Department of Biochemistry and Molecular & Cellular Biologydisclaimer icon, Georgetown University Medical Center, Washington DC
3:50 p.m. – 4:20 p.m.Ectopic Neurogenesis Induced by Prenatal Antiepileptic Drug Exposure Increases Seizure Susceptibility in Adult Mice
Kinichi Nakashima,  Professor, Department of Stem Cell biology and Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka
4:20 p.m. – 4:50 p.m.Aging and Anti-Aging of Cognitive Functions
Tatsuhiro Hisatsune, Associate Professor, Department of Integrated Biosciences, Graduate School of Frontier Sciences, The University of Tokyo, Tokyo
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

Cover Image and source: A representation of a build-up of tau protein, which clumps together in nerve cells to form tangles in neurodegenerative diseases (Medical News Todaydisclaimer icon)

 

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