The 29th Annual Meeting of Japan Society of Gene and Cell Therapy

Main Program

Program

JSGCT Chairman’s LectureJapanese

Sep. 11, 9:30-10:00, Room1

Chair
Hideki Mochizuki（Department of Neurology,Osaka University Graduate School of Medicine）
CLCurrent Status and Details of Gene and Cell Therapy
Speaker
Ryuichi Morishita（Department of Clinical Gene Therapy, School of Medicine, Osaka University）

Special Lecture

Special Lecture1Japanese

Sep. 13, 10:00-10:30, Room1

Chair
Ryuichi Morishita（Department of Clinical Gene Therapy, School of Medicine, Osaka University）
Hideki Mochizuki（Department of Neurology, Osaka University Graduate School of Medicine）
SL1
Speaker
Katsunobu Kato（Minister of Health, Labour and Welfare）

Special Lecture2English

Sep. 13, 10:05-10:55, Room2

Chair
Ryuichi Morishita（Department of Clinical Gene Therapy, School of Medicine, Osaka University）
SL2OMNITM Triple-platform technology - Removing the barriers in CRISPR-based gene editing therapy
Speaker
David Baram（EmendoBio, Inc., USA）

（Sponsor：AnGes, Inc.）

Dialogue SessionJapanese

Sep. 12, 13:30-14:10, Room1

Chair
Hideki Mochizuki（Department of Neurology, Osaka University Graduate School of Medicine）
Speaker
Takenori Emoto（Fomer member of the House of Councilors）

JSGCT Special Program

JSGCT Special Program1Japanese
– What is missing to bring distinguish research seeds of / related gene therapy in Japan into clinical innovations in the global market? –

Sep. 12, 15:00-16:30, Room1

Chair
Yasushi Kajii（R&D Japan Region, Takeda Pharmaceutical Company Limited）
Yasuko Terao（Janssen Pharmaceutical Company, Global Government Grant Office）
SP1-1Gene therapy technologies to be expected by global pharma in the future
Speaker
Yasushi Kajii（Takeda Pharmaceutical Company Limited）
SP1-2Genome editing technology innovation : from Japan to Global
Speaker
Tomoji Mashimo（The University of Tokyo, Institute of Medical Science）
SP1-3The success key to develop academia- oriented gene therapy technologies to global innovative products
Speaker
Takeshi Takahashi（Catalys Pacific）
SP1-4Building ecosystem to accelerate commercialization of innovative technology
Speaker
Toshio Fujimoto（iPark Institute, Co. Ltd.）
Panelist
Yoshinori Ikeura（Axcelead, Inc.）

JSGCT Special Program2English
– Policy initiatives from the Ministries and AMED for gene and cell therapy in Japan –

Sep. 12, 17:20-18:50, Room1

Chair
Hideki Mochizuki（Department of Neurology,Osaka University Graduate School of Medicine）
Masafumi Onodera （Gene & Cell Therapy Promotion Center, National Center for Child Health and Development ）
SP2-1MEXT’s R&D promotion for regenerative medicine and cell and gene therapy
Speaker
Hiroyuki Kamai（Ministry of Education, Culture, Sports, Science and Technology (MEXT))
SP2-2Amendment of the Act of the Safety of Regenerative Medicine related to in vivo gene therapy
Speaker
Keigo Sano（Office for Promotion of Regenerative Medicine, Research and Development Policy Division, Health Policy Bureau, Ministry of Health, Labour and Welfare.）
SP2-3The METI's policy in the field of the cell and gene therapy
Speaker
Hirokazu Shimoda（Bio-Industry Division, Commerce and Service Industry Policy Group, METI)
SP2-4TDB
Speaker
Kentaro Oki（Japan Agency for Medical Research and Development）

JSGCT Special Program3Japanese
– Implementation of gene and cell therapy products. Voices from Clinical Sites –

Sep. 13, 10:25-11:55, Room1

Chair
Masafumi Onodera（Gene & Cell Therapy Promotion Center, National Center for Child Health and Development ）
Hideki Mochizuki（Department of Neurology,Osaka University Graduate School of Medicine）
SP3-1Forefront of critical care by regenrative medicine　～Treatment strategies for extensive burns using autologus cultured epidermis
Speaker
Takahiro Ueda（Tottori University Faculty of Medicine）
SP3-2Utilization Experience of JACC® : Advancing Cartilage Injury Treatment in the Knee Joint
Speaker
Yuki Kato（Department of Sports Medicine, Kameda Medical Center）
SP3-3How to use CAR-T cell therapy in practice ? The past and the future from the view point of Cytotherapy Operation
Speaker
Yasuyuki Arai（Kyoto University Hospital）
SP3-4Gene therapy for hereditary neuromuscular diseases in real clinical practice
Speaker
Yuko Shimizu-Motohashi（National Center of Neurology and Psychiatry）

Vice-Presidential Special ProgramJapanese

Sep. 12, 9:00-10:30, Room1

Chair
Naoki Hosen（Department of Hematology and Oncology, Osaka University Graduate School of Medicine）
Keiya Ozawa（Department of Molecular Biology, Jichi Medical University）
PSP1Identification of new targets for CAR-T cell therapy against hematological cancers
Speaker
Naoki Hosen（Osaka University Graduate School of Medicine）
PSP2Recent advances of CAR-T cell technology for the treatment of solid cancers
Speaker
Koji Tamada（Yamaguchi University Graduate School of Medicine）
PSP3Lights and shadows in CAR-T cell therapy - Expectation and exhaustion of the clinical team
Speaker
Yasuyuki Arai（Kyoto University Hospital）
PSP4Development of allogeneic iPSC-derived Regenerative CAR-T cell therapy
Speaker
Shin Kaneko（Center for iPS cell Research and Application, Kyoto University ／ Transborder Medical Research Center, University of Tsukuba）

Debate SessionJapanese

Sep. 13, 9:25-10:25, Room1

Chair
Aya Kubota（Nikkei Business Publications, Inc.）
Speaker
Ryosuke Takahashi（Kyoto University Graduate School of Medicine）
Hideki Mochizuki（Department of Neurology, Osaka University Graduate School of Medicine）

ASGCT/ESGCJSGCT Joint SymposiumEnglish

Sep. 11, 17:15-18:45, Room1

Chair
Noriyuki Kasahara（Departments of Neurological Surgery and Radiation Oncology University of California）
Takafumi Nakamura（Division of genomic Medicine, Tottori University Faculty of Medicine）
JS-1Next generation gene therapies for Duchenne muscular dystrophy (DMD)
Speaker
Jeffrey S. Chamberlain（University of Washington School of Medicine）
JS-2How to empower current gene and cell therapy approaches by next generation AAV vectors
Speaker
Hildegard Büning（Hannover Medical School, Hannover, Germany）
JS-3Plasmid DNA-based Gene Therapy: From Regenerative Medicine to Vaccine
Speaker
Ryuichi Morishita（Department of Clinical Gene Therapy, School of Medicine, Osaka University）

Invited Lectures

Invited Lectures1English

Sep. 12, 9:00-9:45, Room2

Chair
Toya Ohashi（The Jikei University School of Nursing, Department of Human Health Science and Therapeutics）
IL1Cholesterol Pathway Gene Therapy to treat Neurodegenerative Diseases
Speaker
Natalie Cartier（Asklepios BioPharmaceutical, Inc, Institut du Cerveau (ICM), France）

Invited Lectures2English

Sep. 12, 10:30-11:20, Room1

Chair
Yoshikatsu Eto（Advanced Clinical Research Center, Southern Tohoku Institute for Neuroscience/ Prof. (Emeritus) Tokyo JikeiUniversity School of Medicine）
IL2Improving efficacy and safety of AAV vectors for in vivo gene therapy
Speaker
Hildegard Büning（Hannover Medical School, Hannover, Germany）

（Sponsor：AnGes, Inc.）

Invited Lectures3English

Sep. 12, 14:10-15:00, Room1

Chair
Hideki Mochizuki（Department of Neurology, Osaka University Graduate School of Medicine）
Haruhiko Kishima（Department of Neurosurgery, Osaka University Graduate School of Medicine）
IL3Focal brain delivery of gene therapy via blood brain barrier opening in neurodegenerative diseases
Speaker
José A. Obeso（Fundación HM Hospitales. Universidad CEU-San Pablo, Spain）

Invited Lectures4English

Sep. 13, 8:35-9:25, Room1

Chair
Shin-ichi Muramatsu（Division of Neurological Gene Therapy, Jichi Medical University）
Masahiro Toda（Department of Neurosurgery, Keio University School of Medicine）
IL4A GENE THERAPY DELIVERED GROWTH FACTOR APPROACH TO TREATING PARKINSON’S DISEASE AND MULTIPLE SYSTEM ATROPHY
Speaker
Adrian P Kells（Asklepios BioPharmaceutical, Inc. (AskBio))

Educational Lecture

Educational Lecture1Japanese

Sep. 11, 11:30-12:20, Room1

Chair
Teruhide Yamaguchi（Kanazawa institute of technology）
ED1PMDA’s Vision for the Future
Speaker
Haruko Yamamoto（National Cerebral and Cardiovascular Center）

Educational Lecture2Japanese

Sep. 12, 16:30-17:20, Room1

Chair
Takashi Shimada（Nippon Medical School）
ED2What I think about as overviewing current gene therapy in Japan
Speaker
Masafumi Onodera（Gene & Cell Therapy Promotion Center, National Center for Child Health and Development）

Educational Lecture3Japanese

Sep. 13, 15:05-15:55, Room1

Chair
Seiichi Nagano（Department of Neurotherapeutics, Osaka University Graduate School of Medicine）
ED3Ethics and governance of human genome editing and gene therapy
Speaker
Kazuto Kato（Graduate School of Medicine, Osaka University）

Educational Lecture4Japanese

Sep. 13,15:55-16:45, Room1

Chair
Ryuichi Morishita（Department of Clinical Gene Therapy, School of Medicine, Osaka University）
ED4Organoid based regenerative therapy
Speaker
Takanori Takebe（Osaka University / Tokyo Medical and Dental University）

The Japanese Society for Regenerative MedicineJapanese

Sep. 11, 14:15-15:45, Room1

Chair
Yoshikazu Yonemitsu（Kyushu University Graduate School of Pharmaceutical Sciences）
Katsuto Tamai（Osaka University Graduate School of Medicine）
RM-1Development of regeneration-inducing gene therapy for recessive dystrophic epidermolysis bullosa
Speaker
Katsuto Tamai（Osaka University Graduate School of Medicine）
RM-2Development of iPS regenerative therapy using iPS derived cardiac cell patch
Speaker
Yoshiki Sawa（Osaka Police Hospital）
RM-3Challenges for Stem Cell Gene Therapy for CNS Disorders
Speaker
Hideyuki Okano（Department of Physiology, Keio University School of Medicine）
RM-4GAIA-NK: Its biological properties and current supply chain of PBMC sources
Speaker
Yoshikazu Yonemitsu（Kyushu University Graduate School of Pharmaceutical Sciences）

Nucleic Acids Therapeutics Related ProgramEnglish

Sep. 12, 9:45-11:15, Room2

Chair
Satoshi Obika（Graduate School of Pharmaceutical Sciences Osaka UniversityOsaka University）
Makoto Koizumi（Modality Research Laboratories, Daiichi Sankyo Co., Ltd.）
NA-1Advancing Oligonucleotide Therapeutics: Unveiling a New Era of Precision Gene Medicines
Speaker
Hideaki Sato（Luxna Biotech Co., Ltd.）
NA-2Development of Nucleic Acid Derivatives for Application to Antisense Oligonucleotides
Speaker
Satoshi Obika（Graduate School of Pharmaceutical Sciences, Osaka University）
NA-3Development of intracellular environment-responsive lipid-like material (ssPalm) as a DDS platform for the acceleration of nucleic acid/RNA-based medication
Speaker
Hidetaka Akita（Graduate School of Pharmaceutical Sciences、Tohoku University）
NA-4Exon skipping therapeutic strategy using 2'-O,4'-C-ethylene-bridged nucleic acid (ENA) oligonucleotides
Speaker
Makoto Koizumi（Modality Research Laboratories, Daiichi Sankyo Co., Ltd.）

The Japanese Society of Virology Joint ProgramJapanese

Sep. 12, 15:50-17:20, Room2

Chair
Takafumi Nakamura（Division of genomic Medicine, Tottori University Faculty of Medicine）
Keizo Tomonaga（Institute for Life and Medical Sciences, Kyoto University）
JSV-1Ex vivo gene-cell therapy using bornavirus vector
Speaker
Ryo Komorizono（Lab. of RNA viruses, Institute for Life and Medical Sciences, Kyoto University）
JSV-2Recent advances in the development of segmented double-stranded RNA virus vectors
Speaker
Takeshi Kobayashi（Research Institute for Microbial Diseases, Osaka University）
JSV-3Molecular basis for the development of a novel LCMV vector capable of controlling foreign gene expression
Speaker
Masaharu Iwasaki（Research Institute for Microbial Diseases, Osaka University）
JSV-4Generation of a photocontrollable recombinant bovine parainfluenza virus type 3
Speaker
Takashi Okura（Department of Virology 3, National Institute of Infectious Diseases）

The Japanese Society for Genome Editing Joint ProgramJapanese

Sep. 13, 13:35-15:05, Room1

Chair
Hiromi Kanegae（Jikei University of Medicine）
Yasuji Kitabatake（Osaka University Graduate School of Medicine）
GE-1Gene editing for gene therapy ~ Overview
Speaker
Tsukasa Ohmori（Department of Biochemistry, Jichi Medical University）
GE-2Development of novel gene therapy via in vivo gene knock-in technology
Speaker
Keiichiro Suzuki（Osaka University）
GE-3Development of gene therapy for Down syndrome using genome editing technology
Speaker
Yasuji Kitabatake（Osaka University Graduate School of Medicine）
GE-4Induction of gigantic deletion and expansion of applicability to DMD by CRISPR-Cas3
Speaker
Akitsu Hotta（Center for iPS cell Research and Application, Kyoto University）

The Japanese Society of Child Neurology Joint ProgramEnglish

Sep. 13, 10:55-12:25, Room2

Chair
Hitoshi Osaka（Jichi Med Univ, Dept of Pediatrics）
Takanori Yamagata（Tochigi Prefectural Rehabilitation Center）
1Future perspectives on gene therapy for genetic epilepsy
Speaker
Mitsuhiro Kato（Department of Pediatrics, Showa University School of Medicine）
2Gene therapy for intellectual disability, autism and imprinting disorders
Speaker
Shinji Saitoh（Department of Pediatrics and Neonatology, Nagoya City University Graduate School of Medical Sciences）
3Gene therapy for hereditary neuromuscular diseases
Speaker
Hifumi Komaki（National Center of Neurology and Psychiatry）
4Development for Gene therapy of Neurodegenerative Diseases
Speaker
Kazuhiro Muramatsu（Jichi Medical University）
5Establishing a Model for N-of-1 Oligonucleotide Therapeutics: Prospect and Challenge in Japan
Speaker
Tojo Nakayama（Neurology and Neurological Science Tokyo Medical and Dental University）

Genetic Counseling in Gene Therapy

Genetic counseling and gene therapyJapanese

Sep. 13, 9:35-11:05, Room 3（1008, 10F）

Chair
Atsushi Watanabe（Division of Clinical Genetics and Support Center for Genetic Medicine, Kanazawa University Hospital）
Masayoshi Nakakuni（National Center for Child Health and Development）
GC-1Genetic counseling for issues of gene therapy
Speaker
Masayoshi Nakakuni（National Center for Child Health and Development）
GC-2Current status and trend of genetic counseling and genetic/genomic medicine
Speaker
Atsushi Watanabe(Division of Clinical Genetics and Support Center for Genetic Medicine, Kanazawa University Hospital)
GC-3Roles and issues in genetic counseling for treatable genetic diseases: From the perspective of Certified Genetic Counselors in the pharmaceutical industry
Speaker
Mio Tsuchiya（Amicus Therapeutics K.K.）
GC-4Significance of early and pre-symptomatic diagnosis following early intervention by newborn screening
Speaker
Torayuki Okuyama（Saitama Medical University）

Symposium

Symposium1 Cancer Gene TherapyJapanese

Sep. 11, 10:30-12:20, Room2

Chair
Kentaro Ohki（Japan Agency for Medical Research and Development）
Yozo Nakazawa（Department of Pediatrics, Shinshu University School of Medicine）
S1-1Academic drug discovery of CAR-T cells: current status and issues
Speaker
Yozo Nakazawa（Department of Pediatrics, Shinshu University School of Medicine）
S1-2New Developments in Gene Therapy and Regenerative Medicine
Speaker
Masahiro Tsuji（JST-CRDS）
S1-3Translating Japanese CAR-T Cell Therapeutics into Clinical Practice - From Academic Breakthroughs to Promising Clinical Trials in Collaboration with Biotech Venture
Speaker
Shigeki Yagyu（Shinshu University Innovative Research & Liaison Organization）
S1-4Role of the CDMO in CAR-T Product Development
Speaker
Shin Kawamata（Cyto-Facto inc）

Symposium2 Oncolytic virus and cell-based therapyJapanese

Sep. 11, 14:15-15:45, Room2

Chair
Toshiyoshi Fujiwara（Okayama University, Institute of Academic and Research, Medicine, Dentistry and Pharmaceutical Sciences）
Hirouiki Mizuguchi（Graduate School and School of Pharmaceutical Sciences, Osaka University）
S2-1Stem cell therapy for leptomeningeal disease
Speaker
Yohei Kitamura（Department of Neurosurgery, Keio University School of Medicine）
S2-2Gene edited and engineered stem cell based virotherapy for melanoma leptomeningeal metastasis
Speaker
Nobuhiko Kanaya（Department of Gastroenterological Surgery, Okayama University Graduate School of Medicine, Dentistry, and Pharmaceutical Sciences）
S2-3Development of a novel oncolytic adenovirus composed of an adenovirus serotype 35
Speaker
Fuminori Sakurai（Graduate School of Pharmaceutical Sciences, Osaka University）
S2-4Next-generation oncolytic vaccinia virus
Speaker
Takafumi Nakamura（Tottori University Faculty of Medicine）

Symposium3 RegulationJapanese

Sep. 11, 15:45-17:15, Room1

Chair
Kume Akihiro（Jichi Medical University Hospital, Clinical Research Center）
Eriko Uchida（National Institute of Health Sciences）
S3-1PMDA's efforts to promote the development of gene therapy products and recent approval status
Speaker
Jun Matsumoto（Pharmaceuticals and Medical Devices Agency）
S3-2Points to consider on non-clinical safety assessment for gene therapy products
Speaker
Kazushige Maki（Pharmaceuticals and Medical Devices Agency）
S3-3Update on the implementation of the Cartagena Act and the points of quality control of Living Modified Organisms
Speaker
Akira Sakurai（Pharmaceuticals and Medical devices Agency）

Symposium4 Neuromuscular DisordersEnglish

Sep. 11, 15:45-17:15, Room2

Chair
Shin-ichi Muramatsu（Division of Neurological Gene Therapy, Jichi Medical University）
José A. Obeso（Fundación HM Hospitales. Universidad CEU-San Pablo, Spain）
S4-1Advances and Challenges of Next-Generation CRISPR 4.0 Gene-Editing Technology for Neurological Diseases
Speaker
Alvin Luk（HuidaGene Therapeutics, Inc.）
S4-2Gene Therapy for ALS
Speaker
Shin Kwak（Division of Neurology, Tokyo Medical University）
S4-3Establishment of a mouse model of inherited PIGO deficiency and therapeutic potential of AAV-based gene therapy
Speaker
Yoshiko Murakami（Research Institute for Microbial Diseases, Osaka University）
S4-4A Roche reflection on our experiences with gene therapy in Duchenne muscular dystrophy
Speaker
Alexander P. Murphy（Roche Products Ltd, Welwyn Garden City, UK）

Symposium5 Genetic DiseasesEnglish

Sep. 11, 17:15-18:45, Room2

Chair
Hiroshi Kobayashi（Division of Gene Therapy, Research Cenetr for Medical Sciences, The Jikei University School of Medicine）
Makoto Migita（Nippon Medical School Pediatrics）
Nathalie Cartier（Asklepios BioPharmaceutical, Inc, Institut du Cerveau (ICM), France）
S5-1Efficacy and Safety of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia
Speaker
Tae Matsumoto（Department of Nippon Medical School）
S5-2Gene therapy for patients with Niemann-Pick disease type C1 (NPC1) using AAV.GTX-NPC1
Speaker
Karin Kojima（Dept. of Pediatrics, Jichi Medical University）
S5-3Gene therapy for lysosomal storage diseases using lentiviral vectors
Speaker
Yohta Shimada（Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine）
S5-4Gene therapy for inborn errors of immunity
Speaker
Toru Uchiyama（Division of Molecular Pathogenesis, Department of Human Genetics, National Center for Child Health and Development）
S5-5Hematopoietic stem cell-targeted gene therapy with lentiviral vectors to treat sickle cell disease
Speaker
Naoya Uchida（National Institutes of Health）

Symposium6 non viral vector・Advanced medicineJapanese

Sep. 12, 14:20-15:50, Room2

Chair
Hironori Nakagami（Osaka University Graduate School of Medicine）
Tatsushi Toda（Department of Neurology, Graduate School of Medicine, The University of Tokyo）
S6-1Application of DNA nanotechnology for Antisense Therapeutics
Speaker
Takeshi Yamamoto（Graduate School of Biomedical Sciences, Nagasaki University）
S6-2Department of General Medicine, Niigata University School of Medicine
Speaker
Kenya Kamimura（Department of General Medicine, Niigata University School of Medicine）
S6-3Development of Gene Therepy for Loss-of-function Cardiomyopathy
Speaker
Yoshihiro Asano（Director of Genomic Medicine, National Cerebral and Cardiovascular Center/Osaka University）
S6-4Development of therapeutic vaccine for common diseases and intractable diseases
Speaker
Hironori Nakagami（Osaka University Graduate School of Medicine）

Symposium7 Vector DevelopmentEnglish

Sep. 12, 17:20-18:50, Room2

Chair
Takashi Okada（Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo）
Hiroyuki Nakai（Oregon Health & Science University）
S7-1AAV9, intracoronary artery infusion
Speaker
Kiyotake Ishikawa（Mount Sinai）
S7-2Development of Recombinant AAV for Human Gene Therapy: Vector Design and Manufacturing Strategies to Reduce Immune Responses
Speaker
John Fraser Wright（Stanford University School of Medicine）
S7-3The safety and efficacy of pre-treatment with imlifidase prior to adeno associated virus (AAV)-based gene therapy in non-human primates with pre-existing anti-AAVrh74 antibodies
Speaker
Rachael A Potter（Sarepta Therapeutics, Inc., Cambridge, MA, USA）

Symposium8 CancerEnglish

Sep. 13, 8:35-10:05, Room2

Chair
Yasutomo Nasu（Okayama University）
Hiroshi Fukuhara（Kyorin University）
S8-1Alteration of the surrounding immunological tumor environment by Oncolytic viruses
Speaker
Hideki Kasuya（Nagoya University Graduate School of Medicine、Cancer Immune Therapy Center）
S8-2Clinical trials and next-generation technology development of Survivin-responsive conditionally replicating denovirus targeting and treating with multiple factors (Surv.m-CRA)
Speaker
Kenichiro Kosai（Kagoshima University Graduate School of Medical and Dental Sciences / Kagoshima University Hospital）
S8-3The Role of Myeloid-derived Suppressor Cells in Construction of Immune Suppressive Tumor Microenvironment of Pancreatic Cancer
Speaker
Kazunori Aoki（National Cancer Center Research Institute）
S8-4Oncolytic Adenoviruses for Pancreatic Cancers
Speaker
Masato Yamamoto（University of Minnesota）

Symposium9 Vector ManufacturingEnglish

Sep. 13, 15:05-16:35, Room2

Chair
Toshihiko Okazaki（Medical Center for Translational Research Osaka University Hospital）
Susumu Uchiyama（Department of Biotechnology, Graduate School of Engineering, Osaka University）
S9-1GMP viral vector manufacturing for academia R&D
Speaker
Mikako Wada（Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo）
S9-2Quantitative analysis of GOI and impurity nucleic acids of AAV viral vectors by droplet digital PCR
Speaker
Kazuhisa Uchida（Kobe University Graduate School of Science,Technology and Innovation）
S9-3Establishment of new human amniotic epithelial cell lines for scalable production of recombinant adeno-associated viral vectors for gene therapy
Speaker
Yugo Hirai（Chitose Laboratory Corp.）
S9-4Analytical development and quality control of AAV vectors
Speaker
Susumu Uchiyama（Osaka University）

Young Scientists Symposium

Explore a new era of gene therapyJapanese

Sep. 13, 8:35-9:35, Room2

Chair
Makoto Otsu（Kitasato University School of Allied Health Sciences）
Kenya Kamimura（Department of General Medicine Niigata University School of Medicine）
YS-1Department of Molecular Life Science, Division of Basic Medical Science and Molecular Medicine, Tokai University School of Medicine
Speaker
Hiromi Miura（Department of Molecular Life Science, Division of Basic Medical Science and Molecular Medicine, Tokai University School of Medicine）
YS-2Neonatal genome editing for protein C deficiency in mice
Speaker
Tomoki Togashi（Department of Clinical Laboratory Science, Division of Health Sciences, Graduate School of Medical Science, Kanazawa University）
YS-3Graduate School of Pharmaceutical Sciences, Osaka University
Speaker
Ikuho Ishigami（Graduate School of Pharmaceutical Sciences, Osaka University）
YS-4Devision of Molecular Target and Gene Therapy Products, National Institute of Health Sciences
Speaker
Takenori Yamamoto（Devision of Molecular Target and Gene Therapy Products, National Institute of Health Sciences）

Plenary sessionJapanese

Sep. 11, 10:30-11:30, Room1

Chair
Makoto Otsu（Kitasato University School of Allied Health Sciences）
Yumi Kanegae（Jikei University of Medicine）
PS-1A blood-brain barrier-penetrating AAV2 capsid variant BR1N that evades neutralizing antibodies against AAV9 capsid protein
Speaker
Yuto Kawabata（Department of Neurophysiology & Neural Repair, Gunma University Graduate School of Medicine）
PS-2Long-term efficacy of gene therapy for AADC deficiency, including patients with a moderate phenotype.
Speaker
Marina Mizobe（Department of Pediatrics, Jichi Medical University）
PS-3TBD
Speaker
Kashiwakura Yuji （Department of Biochemistry, Jichi Medical University）
PS-4First in human clinical study of ex vivo gene therapy using autologous adipocytes for familial LCAT deficiency syndrom
Speaker
Masayuki Kuroda（Center for Advanced Medicine, Chiba University Hospital）

Corporate Seminar

Luncheon Seminar

Luncheon Seminar 1

Sep. 11, 12:25-13:25, Room1

Chair
Makoto Otsu（Division of Hematology, Department of Medical Laboratory Sciences, Kitasato University School of Allied Health Sciences）
LS1Development and Clinical translation of piggyBac transposon CAR-T cell therapy
Speaker
Shigeki Yagyu（Shinshu University, Innovative Research & Liaison Organization）

（Sponsor：MaxCyte, Inc. & Kiko Tech., Ltd）

Luncheon Seminar 2
Research and Clinical Experience of Genetic Disorders – Development of Nucleic

Sep. 11, 12:25-13:25, Room2

Chair
Akihiro Kume（Jichi Medical University Hospital, Clinical Research Center）
LS2-1Development of Therapeutic Drug for Spinal Muscular Atrophy – From Basic Research to Clinical Trials and Insurance Coverage
Speaker
Reiko Arakawa（Center Hospital of the National Center for Global Health and Medicine, Department of Genomic Medicine）
LS2-2Pathology and Treatment of Spinal Muscular Atrophy Viewed Through a Patient-derived Cell Model
Speaker
Michiko Yoshida（Kyoto City Hospital, Department of Pediatrics）

（Sponsor：Biogen Japan Ltd.）

Luncheon Seminar 3

Sep. 11, 12:25-13:25, Room3

Chair
Yoshikazu Yonemitsu（R&D Laboratory for Innovative Biotherapeutics, Kyushu University Graduate School of Pharmaceutical Sciences）
LS3ELEVECTA, the next generation of AAV manufacturing that combines high scalability and increased safety by reducing encapsidated host cell DNA
Speaker
Michelle Hussong（Head of Molecular Biology and Analytics, Viral Vectors - Genomics Medicine, Cytiva, Germany）

（Sponsor：Cytiva）

Luncheon Seminar 4

Sep. 12, 11:30-12:30, Room1

Chair
Masayuki Nakamori（Department of Neurology and Clinical Neuroscience, Yamaguchi University Graduate School of Medicine）
LS4A disease in 21st century: ATTRv amyloidosis, its pathogenesis, therapies, and perspective
Speaker
Yukio Ando（Department of Amyloidosis Research, Nagasaki International University, Kumamoto University）

（Sponsor：Alnylam Japan K.K.）

Luncheon Seminar 5

Sep. 12, 11:30-12:30, Room2

Chair
Hiroshi Fujiwara（Department of Personalized Cancer Immunotherapy, Graduate School of Medicine, Mie University）
LS5Research and Clinical Development of CAR-T Cell Therapy -including manufacturing in Academia-
Speaker
Naoki Hosen（Department of Hematology and Oncology, Graduate School of Medicine, Osaka University）

（Sponsor：Miltenyi Biotec K.K.）

Luncheon Seminar 6

Sep. 12, 11:30-12:30, Room3

Chair
Masafumi Onodera（Gene & Cell Therapy Promotion Center, National Center for Child Health and Development）
LS6-1Current status and perspectives of analytical development for viral vectors
Speaker
Susumu Uchiyama（Department of Biotechnology, Graduate School of Engineering, Osaka University）
LS6-2Cell culture process development for AAV vector production in HEK293 suspension cells
Speaker
Yoshinori Takagi（Medical Business Unit, Synplogen Co., Ltd.）

（Sponsor：Synplogen Co., Ltd.）

Luncheon Seminar 7

Sep. 13, 12:30-13:30, Room1

Chair
Tatsuji Enoki（Takara Bio Inc.）
LS7Gene therapy for neuroprotection and axon regeneration.
Speaker
Kazuhiko Namekata（Tokyo Metropolitan Institute of Medical Science）

（Sponsor：Takara Bio Inc.）

Luncheon Seminar 8

Sep. 13, 12:30-13:30, Room2

Chair
Takanori Yamagata（Department of Pediatrics, Tochigi Rehabilitation Center）
LS8-1Our experience of gene replacement therapy with Zolgensma : Importance of early treatment for spinal muscular atrophy
Speaker
Tomokazu Kimizu（Department of Pediatric Neurology, Osaka Women's and Children's Hospital）
LS8-2Gene therapy for Inherited retinal dystrophy
Speaker
Kaoru Fujinami（Laboratory of Visual Physiology, Division of Vision Research, National Institute of Sensory Organs, National Hospital Organization Tokyo Medical Center）

（Sponsor：Novartis Pharma K.K. Gene Therapies Business Unit Medical Division）

Luncheon Seminar 9

Sep. 13, 12:30-13:30, Room3

Chair
Hitoshi Osaka（Department of Pediatrics, Jichi Medical University）
LS9-1Driving pharmaceutical open innovation by T-CiRA program
Speaker
Yasushi Kajii（R&D Japan Region, Takeda Pharmaceutical Company Limited）
LS9-2Toward finding a cure for NGLY1 deficiency
Speaker
Tadashi Suzuki（Glycometabolic Biochemistry Laboratory, RIKEN Cluster for Pioneering Research, RIKEN）

（Sponsor：Takeda Pharmaceutical Company Limited）

Morning seminar

Morning seminar 1

Sep. 12, 8:15-8:55, Room1

Chair
Hideki Mochizuki（Department of Neurology, Osaka University Graduate School of Medicine）
MS1Pathogenesis of thymoma-associated myasthenia gravis
Speaker
Tatsusada Okuno（Department of Neurology, Osaka University Graduate School of Medicine）

（Sponsor：Alexion Pharma GK）

Morning seminar 2

Sep. 12, 8:15-8:55, Room2

Chair
Atsushi Miyamura（Nepa Gene Co., Ltd.）
MS2Cell and Gene Therapy Product Management, Precision NanoSystems
Speaker
Angela Zhang（Cell and Gene Therapy Product Management, Precision NanoSystems）

（Sponsor：Nepa Gene Co., Ltd.）

Morning seminar 3

Sep. 12, 8:15-8:55, Room3

Chair
Seiichi Nagano（Department of Neurotherapeutics, Osaka University Graduate School of Medicine）
MS3Development of nucleic acid therapeutics for ALS and latest topics in ALS treatment
Speaker
Yoshitaka Nagai（Department of Neurology, Kindai University Faculty of Medicine）

（Sponsor：Mitsubishi Tanabe Pharma Corporation）

Morning seminar 4

Sep. 13, 7:45-8:25, Room1

Chair
Akiko Ishii（National Hospital Organization Iwaki Hospital, Japan.）
MS4Pathophysiology and new therapeutic approaches in myasthenia gravis
Speaker
Masanori Takahashi（Department of Clinical Laboratory and Biomedical Sciences, Division of Health Sciences, Osaka University Graduate School of Medicine, Japan）

（Sponsor：argenx Japan K.K.）

Morning seminar 5

Sep. 13, 7:45-8:25, Room2

Chair
Yuko Kasahara（The Institute of Medical Science, The University of Tokyo）
MS5High-throughput and accurate AAV Capsid quantitation using biomolecular interaction analysis
Speaker
Yusuke Maruyama（Sartorius Japan K.K. Field Application Scientist）

（Sponsor：Sartorius Japan K.K.）

Morning seminar 6

Sep. 13, 7:45-8:25, Room3

Chair
Hiroyuki Nakai（Department of Molecular and Medical Genetics, Oregon Health & Science University）
MS6Towards Developing Best-In-Class rAAV Vectors: Guidance for a successful Go-to Market Gene Therapy Strategy
Speaker
Kathrin Schneider（Department of Molecular and Medical Genetics, Oregon Health & Science University）

（Sponsor：Revvity, Inc.）

Evening seminar

Evening seminar 1

Sep. 11, 18:50-19:30, Room1

Chair
Fuminori Sakurai（Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Japan.）
ES1Development of gene and cell therapy for intractable diseases in childhood
Speaker
Toru Uchiyama（Department of Human Genetics, National Center for Child Health and Developemt, Japan.）

（Sponsor：Bio-Rad Laboratories K.K.）

Evening seminar 2
HEK Media and Manufacturing portfolio for Gene and Cell Therapy offered by Sartorius

Sep. 11, 18:50-19:30, Room2

Chair
Takashi Okada（Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo）
ES2-1
Speaker
Toru Tsuji（Sartorius Stedim Japan K.K. Sales department, Field Application Specialist）
ES2-2
Speaker
Shiori Akiyama（Sartorius Stedim Japan K.K. Sales department, Field Application Specialist）

（Sponsor：Sartorius Stedim Japan K.K.）

Evening seminar 3

Sep. 11, 18:50-19:30, Room3

Chair
Shin-ichi Muramatsu（Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University）
ES3Advances in Treatment of Spinal Muscular Atrophy
Speaker
Toshio Saito（Division of Child Neurology, Department of Neurology, National Hospital Organization Osaka Toneyama Medical Center）

（Sponsor：CHUGAI PHARMACEUTICAL CO., LTD.）

Sponsored seminar

Sep. 11, 17:55-19:45, Room3

Chair
Tatsushi Toda（Department of Neurology, Graduate School of Medicine, The University of Tokyo）
SSStatistical genetics elucidates disease biology and drug discovery
Speaker
Yukinori Okada（Department of Genome Informatics, Graduate School of Medicine, the University of Tokyo / Department of Statical Genetics, Osaka University Graduate School of Medicine / Laboratory for Systems Genetics, RIKEN Center for Integrative Medical Sciences）

（Sponsor：Scrum Inc.）