Main Program

Day 1,
July 26, 2018 Day 2,
July 27, 2018 Day 3,
July 28, 2018

General Session:Oral General Session:Poster

Day 1, July 26, 2018

11:10-12:00 (Main Hall)

Presidential Special Program with AMED & PMDA (J)

1. Mission of AMED:Empowering gene therapy for IRUD Beyond
   Chair: Jun Yoshida (Professor Emeritus, Nagoya University)
   Speaker: Makoto Suematsu (Japan Agency for Medical Research and Development)
2. PMDA’s Initiatives Based on Regulatory Science－In Pursuit of Developing Gene Therapies－
   Co chair: Takashi Shimada (Professor Emeritus, Nippon Medical School)
   Speaker: Tatsuya Kondo (Pharmaceuticals and Medical Devices Agency)

15:10-17:00 (Main Hall)

Symposium-I:JCA-JSGCT joint Symposium" All about CAR-T cell therapy: current status and future perspective" (E)
Chairs: Keiya Ozawa (Jichi Medical University)
Koji Tamada (Yamaguchi University Graduate School of Medicine)

1. Overview of CAR-T Cell Therapy
   Keiya Ozawa (Jichi Medical University)
2. CAR T cell Therapy: The CD19 Paradigm and beyond
   Isabelle Rivière(Michael G. Harris Cell Therapy and Cell Engineering Facility Center for Cell Engineering)
3. The activated conformation of integrin β7 is a novel multiple myeloma-specific target for CAR T-cell therapy
   Naoki Hosen (Osaka University Graduate School of Medicine)
4. A novel platform technology of CAR-T cell therapy for solid tumors
   Koji Tamada ( Yamaguchi University Graduate School of Medicine)

17:00-18:50 (Main Hall)

Symposium II: Genetic Diseases(E)
Chairs: Toya Ohashi (The Jikei University School of Medicine)
Torayuki Okuyama (National Center for Child Health and Development)

1. Exon Skipping Therapy for Muscular Dystrophy
   Shin'ichi Takeda (National Center of Neurology and Psychiatry)
2. Developing AAV Gene Therapies for Rare Genetic Diseases
   Juan Ruiz (Abeona Therapeutics, Inc.)
3. Gene therapy for child neurological diseases: Effective for AADC deficiency, new challenges for Glut1-deficiency syndrome and potential for other Gene therapy for child neurological diseases
   Karin Kojima (Jichi Medical University)
4. Genetic Engineering of Hematopoiesis to Treat Inherited Diseases
   Alessandro Aiuti (San Raffaele Institute, Milan, Italy)

18:50-19:30(Main Hall)

Presidential Evening Symposium I (E)
Chair: Masafumi Onodera (President of JSGCT2018/National Center for Child Health and Development)

1. Orchard Therapeutics and its Gene Therapy Programs
   Speaker: Christopher Dott (Orchard Therapeutics)
2. " Development of gene therapy related treatments in Novartis"
   Speaker: Tohru Hirose (Novartis Pharma K.K.)

Day 2, July 27, 2018

08:30-09:50 (Main Hall)

Symposium-III: Regulatory Science (J)
Chairs: Akihiro Kume (Jichi Medical University)
Eriko Uchida (National Institute of Health Sciences)

1. Revised Ethical Guideline for Clinical Trials on Gene Therapy
   Takashi Ichikawa (Ministry of Health, Welfare and Labour)
2. Revision of guideline for gene therapy clinical study based on the novel technology of gene editing
   Teruhide Yamaguchi (Kanazawa Institute of Technology/ Nihon Pharmaceutical University)
3. Points to consider in quality and preclinical aspects of gene therapy products
   Megumi Kawamoto (Pharmaceuticals and Medical Devices Agency)

08:30-09:30 (Hall-1)

Symposium IV: JSGCT Working Group Symposium (J)
" The Advancement in Innovative Approaches for Next-generation Gene Therapy "
Chairs: Makoto Otsu (The Institute of Medical Science, The University of Tokyo)
Fuminori Sakurai (Graduate School of Pharmaceutical Sciences, Osaka University)

1. First-in-human investigator-initiated clinical trial of survivin-respoinsive conditionally replicating adenovirus for treating bone and soft tissue sarcoma
   Satoshi Nagano (Kagoshima University)
2. Chimeric Antigen Receptor T cells redirecting EPHB4 for the Treatment of Human Rhabdomyosarcoma
   Kayoko Nakamura (Shinshu University Hospital)
3. Precise Gene Editing by SNGD
   Shinichiro Nakada (Graduate School of Medicine, Osaka University)

09:50-11:10 (Main Hall)

Special Symposium I (E)
" Gene therapy for Wiskott-Aldrich Syndrome"
Chair: Masafumi Onodera (President of JSGCT2018/National Center for Child Health and Development)
Speaker: Alessandro Aiuti (San Raffaele Institute, Milan, Italy, ESGCT)

" Human Gene Therapy - The Road to Today and Beyond"
Chairs: Tomoki Todo (President-Elect /The Institute of Medical Science, The University of Tokyo)
Speaker: Guangping Gao (University of Massachusetts Medical School, ASGCT President Elect)

11:10-11:40 (Main Hall)

JSGCT Chairman’s Lecture (J)
" Japan should accelerate human gene therapy without delay"
Chair: Shigetaka Asano (Professor Emeritus: The University of Tokyo and Waseda University)
Speaker: Yasufumi Kaneda (Graduate School of Medicine, Osaka University, Chairman of JSGCT)

11:40-12:00 (Main Hall)

Presidential Lecture (E)
" Gene and Cell Therapy, To Take One Step Forward"
Chair: Yoshikatsu Eto (Advanced Clinical Research Center & Asian LSD Center, Institute of Neurological Disorders)
Speaker: Masafumi Onodera (President of JSGCT2018/National Center for Child Health and Development)

13:00-13:40 (Main Hall)

Educational Lecture (E)
Chairs: Yoshiro Niitsu (Professor Emeritus, Sapporo Medical University)

1. Molecular mechanism of CRISPR and structure-based development of genome editing tool towards medical applications
   Osamu Nureki (The University of Tokyo)
2. Genome editing for therapeutic applications
   Ko Mitani (Saitama Medical University)

13:00-14:10 (Hall-1)

Symposium V: Vector" GeneRide and HITI: Cutting Edge Technologies in Vectors and Genome Editing" (E)
Chairs: Takashi Okada (Nippon Medical School)
Hiroyuki Nakai (Oregon Health & Sciences University)

1. GeneRideTM, a novel AAV-mediated strategy to treat pediatric indications
   Nelson Chau (LogicBio Therapeutics)
2. Genome-editing therapy via HITI (Homology-Independent Targeting Integration) method
   Keiichiro Suzuki (Osaka University)

13:40-15:20 (Main Hall)

Special Symposium II" Biosafety Regulations in Gene Therapy" (J)
Chairs: Masafumi Onodera (President of JSGCT2018/National Center for Child Health and Development)
Teruhide Yamaguchi (Nihon Pharmaceutical University)

1. Environmental assessments and shedding studies for gene therapy products in the US and in the EU
   Eriko Uchida (National Institute of Health Sciences)
2. Points to consider for environmental risk assessment of the use of living modified organisms; PMDA perspectives on type 1 use approval and type 2 use confirmation under" Cartagena Law"
   Kazunobu Oyama (Pharmaceuticals and Medical Devices Agency)

14:10-15:20 (Hall-1)

Symposium-VI: Genome Editing" Genome editing: Step to gene therapy" (E)
Chairs: Yumi Kanegae (The Jikei University School of Medicine)
Akitsu Hotta (CiRA, Kyoto University)

1. CRISPR-mediated correction of CD34+ hematopoietic stem cells from patients with chronic granulomatous disease
   Harry L. Malech (National Institutes of Health)
2. Development of Genome-editing treatment for hemophilia
   Tsukasa Ohmori (Jichi Medical University School of Medicine)
3. Genome editing and iPS cell mediated gene therapy: how to overcome in vivo delivery and ex vivo immunorejection
   Akitsu Hotta (CiRA, Kyoto University)

15:30-17:20 (Main Hall)

International Symposium" Steps forward to Clinic in Gene and Cell Therapy of Genetic Diseases" (E)
Chairs: Noriyuki Kasahara (University of Miami)
Masato Yamamoto (University of Minnesota)

1. Development of Luxturna™ (voretigene neparvovec-rzyl): Gene Therapy for RPE65 Biallelic Mutation Associated Inherited Retinal Disease
   Daniel C. Chung (Spark Therapeutics)
2. YAP/TAZ-dependent depletion of human epidermal stem cells in Junctional Epidermolysis Bullosa
   Michele De Luca (University of Modena and Reggio Emilia)
3. Lentivector Gene Therapy for X-linked Chronic Granulomatous Disease.
   Harry L. Malech (National Institutes of Health)

18:00-18:20(Main Hall)

Presidential Evening Symposium II
" Gene Therapy (GTx): A Path to Deliver Transformative Medicine to Patients"
Chair: Masafumi Onodera (President of JSGCT2018/National Center for Child Health and Development)
Speaker: Takayuki Imaeda (Pfizer Inc.)

Day 3, July 28, 2018

08:30-10:15 (Main Hall)

Symposium-VII: Regenerative Medicine" Interface between gene therapy and regenerative medicine" (E)
Chairs: Katsuto Tamai (Osaka University)
Toru Uchiyama (National Center for Child Health and Development)

1. Life-saving regeneration of the entire human epidermis by transgenic stem cells
   Michele De Luca (University of Modena and Reggio Emilia)
2. Circulating mesenchymal stem cells: their function and possibility as a target of gene therapy.
   Katsuto Tamai (Osaka University)
3. Origin and development of mesenchymal stem cells
   Takumi Era (Institute of Molecular Embryology and Genetics, Kumamoto University)
4. Interspecies organogenesis generates autologous functional islets
   Tomoyuki Yamaguchi (Institute of Medical Science, University of Tokyo)

08:30-10:15 (Hall-1)

Symposium-VIII: GCTP Marketing" Extracting issues toward Social Implementation of Cell & Gene Therapy: What is proper price of the products?" (J)
Chairs: Toshihiko Okazaki (Kyushu University Hospital)
Masanobu Kimura (Takara Bio Inc)

1. Roadmap for industrialization of cell-based therapy from academia to clinical sites.
   Importance of the design of the cell manufacturing system by academia applicable to industrialization.
   Hisako Hashimoto (Foundation for Biomedical Research and Innovation at Kobe)
2. Manufacturing of Cellular and Tissue-based Products
   Junichi Mineno (TAKARA BIO INC.)
3. Development of HeartSheet and current issue
   Tadashi Samejima (Terumo Corp.)
4. Quality of Biological Raw Materials for Manufacturing of Cell-Based and Gene Therapy Products
   Yoji Sato (National Institute of Health Science)

10:15-12:00 (Main Hall)

Symposium-IX: Neurologic Diseases" Nervous system" (E)
Chairs: Shin-ichi Muramatsu (Jichi Medical School)
Hideki Mochizuki (Osaka University)

1. Gene therapy for GM2 gangliosidosis with CNS involvement
   Kohji Itoh (Tokushima University)
2. Gene therapy for ALS
   Seiichi Nagano (Osaka University)
3. Effects of overexpression of TMEM-induced by AAV on the behavioral changes of the drug dependence and anxiety
   Atsumi Nitta (Toyama University)
4. Mouse modeling by genome and epigenome editing
   Takuro Horii (Gunma University)

10:15-12:00 (Hall-1)

Symposium-X: Oncolytic Virus" For clinical implementation of oncolytic virotherapy" (E)
Chairs: Kenzaburo Tani (The Institute of Medical Science, The University of Tokyo)
Toshiyoshi Fujiwara (Okayama Univerisity)

1. Current issues on quality and safety of Oncolytic virus products
   Teruhide Yamaguchi (Kanazawa Institute of Technology/ Nihon Pharmaceutical University)
2. The current preparative status of oncolytic coxsackievirotherapy toward clinical trial
   Shohei Miyamoto (IMS, The University of Tokyo)
3. Recent advances in clinical application of telomerase-specific oncolytic viruses
   Hiroshi Tazawa (Okayama Univerisity Hospital)
4. Clinical evaluation of a novel oncolytic viral immunotherapy agent, Canerpaturev (HF10)
   Maki Tanaka (Takara Bio Inc)
5. Clinical development of third generation oncolytic HSV-1 G47Δ
   Tomoki Todo (The Institute of Medical Science, The University of Tokyo)

13:00-14:50 (Main Hall)

Symposium-XI: Cardiovascular Diseases" Cardiovascular Gene & Cell Therapy and Genome Editing: a recent progress" (E)
Chairs: Ryuichi Morishita (Osaka University Graduate School of Medicine)
Yoshikazu Yonemitsu (Kyushu University Graduate School of Pharmaceutical Sciences)

1. Opening Remarks: Recent Progress of Cardiovascular Gene & Cell Therapy and Gene Editing
   Yoshikazu Yonemitsu (Kyushu University Graduate School of Pharmaceutical Sciences)
2. Cell Therapy Trials in Congenital Heart Disease
   Hidemasa Oh (Okayama University Hospital)
3. Identification of candidate genes for gene therapy against heart failure
   Yuichi Oike (Kumamoto University)
4. Development of novel immunotherapy for cardiovascular diseases
   Hironori Nakagami (Osaka University Graduate School of Medicine)
5. Frontline of gene editing application
   Haruhiko Morita (EdiGENE Corporation)
6. Closing Remarks
   Ryuichi Morishita (Osaka University Graduate School of Medicine)

13:00-14:50 (Hall-1)

Symposium-XII: Cancer Gene Therapy" Gene medicine going into a tranlational phase" (E)
Chairs: Yasutomo Nasu (Okayama University)
Masatoshi Tagawa (Chiba Cancer Center Research Institute)

1. Toca 511 & Toca FC: Evaluation of durable response rate in the post-resection setting and association with survival in patients with recurrent high grade glioma
   Nicholas A. Boyle (Tocagen Inc.,)
2. Clinical development of non-replicating oncolytic virus (HVJ-E/GEN0101) for the treatment of solid cancers
   Toshihiro Nakajima (Genomidea, Inc.)
3. A phase1 clinical trial of inhibiting the HGF/c-Met pathway for malignant pleural mesothelioma with NK4 gene-expressing adenoviral vectors
   Yuji Tada (School of Medicine Chiba University)
4. A phase I/II clinical trial of Ad-SGE-REIC for malignant brain tumor
   Kazuhiko Kurozumi (Okayama University)
5. The current status of clinical trials of Ad-SGE-REIC gene therapy
   Hitoshi Shiomi (Momotaro Gene Inc.,)