Moderator: Dr. Hiroshi Kobayashi (The Jikei Univ. School of Medicine) Announcer:Dr. Saori Miwa (ditto)
09:00-09:15 Opening Remarks and Welcome Greetings Toya Ohashi (President of the 9th ICFHGTGD, The Jikei University School of Medicine) Katsunobu Kato (Chairman of the LDP General Council) 09:15-10:15 Genome Editing Session
Chairs:Yumi Kanegae (The Jikei Univ. School of Medicine) ＆ Masafumi Onodera (NCCHD)
1."Molecular mechanism of CRISPR and structure-based development of genome editing tool towards medical applications"
Osamu Nureki (The University of Tokyo)
2."Therapeutic potential of genome editing for genetic disorders"
Toru Uchiyama (NCCHD)
3."Regulatory Aspects of gene therapy by gene editing tool"
Teruhide Yamaguchi (Kanazawa Institute of Technology/ Nihon Pharmaceutical University)
10:15-11:45 Foreign Biotech Companies I
Chairs:Hiroshi Kobayashi (The Jikei Univ. School of Medicine) ＆ Motomichi Kosuga (NCCHD)
1."Gene silencing in Huntington's disease"
Sander van Deventer (uniQure)
2."Advances in ex vivo lentiviral gene therapy for Fabry disease"
Nerissa Kreher (Avrobio)
11:45-12:45 Corporate Seminar
Chair:Hikaru Takakura (Contract Service Business Development,TAKARA BIO INC.)
"Gene transduced cell manufacturing and cell processing facility"
Ikuei Nukaya (CDM Center Division 1,TAKARA BIO Inc.) 13:00-14:00 Novel Newborn Screening for Genetic Diseases
Chairs:Torayuki Okuyama (NCCHD) ＆ Akihiro Kume (Jichi Medical University)
1."Newborn screening for lysosomal storage diseases"
Torayuki Okuyama (NCCHD)
2."Newborn screening for X-linked adrenoleukodystrophy"
Ryuichi Mashima (NCCHD)
3."Newborn screening for spinal muscular atrophy"
Hisahide Nishio (Faculty of Rehabilitation Kobe Gakuin University)
14:00-15:00 Foreign Biotech Companies II
Chair:Keiya Ozawa (Jichi Medical University)
1."Pfizer perspectives on gene therapy ~ Now and future to drive transformation for healthier world ~"
Yuko Hoshino (Pfizer R&D Japan)
2."Preclinical assessment of valoctogene roxaparvovec (BMN270)"
Gordon Vehar (BioMarin) 15:00-15:30 Invited Lecture I
Chair:Takanori Yamagata (Jichi Medical University)
"Regulation of neural crest fate specification for regenerative medicine"
Ronald J. Parchem (Baylor College of Medicine)
15:30-15:40 Coffee Break I
15:40-16:40 Foreign Biotech Companies III
Chair:Shin-ichi Muramatsu (Jichi Medical University)
1."AVXS-101 gene replacement therapy in spinal muscular atrophy"
Douglas M. Sproule (Avexis)
2."Shire’s liver-targeted AAV gene therapy platform to tackle monogenic rare genetic diseases"
Friedrich Scheiflinger (Shire) 16:40-17:30 Invited Lecture II
Chair:Toya Ohashi (President of the 9th ICFHGTGD2019, The Jikei University School of Medicine)
"Advancing gene therapy strategies for genetic diseases: ex vivo gene editing of hematopoietic stem cells and liver-directed gene transfer"
Luigi Naldini (San Raffaele Telethon Institute for Gene Therapy)
17:30-17:40 Coffee Break II
17:40-18:10 Evening Seminar
Chair:Shin'ichi Takeda (National Center of Neurology and Psychiatry)
"Recent advances of lysosomal storage diseases- Toward to gene therapy"
Yoshikatsu Eto (Advanced Clinical Research Center & Asian LSD Center, Institute of Neurological Disorders) 18:10-18:30 Closing Remarks and Welcome Greetings from the President of JSGCT Tomoki Todo (The Chairman of the Board of Directors of JSGCT, The Institute of Medical Science The University of Tokyo) Yoshikatsu Eto (10th ICFHGTGD2020 President Elect)
19:00-21:00 Welcome Reception (WINE FACTORY)
The 9th International Collaborative Forum of Human Gene Therapy for Genetic Disease2019 Date:January 17 (Thu.), 2019
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日本遺伝子細胞治療学会(JSGCT)事務局 [Japan Society of Gene and Cell Therapy,JSGCT Administrative Office]