We hope that you are enjoying your time and doing well. I would also like to express my deepest gratitude for your continued support and cooperation.
We are pleased to announce that the 15th International Collaborative Forum of Human Gene Therapy for Genetic Disease will be held on January 24, 2025, at the auditorium of the Jikei university school of medicine. The COVID-19 pandemic has been temporarily under control, and we are planning to hold this year's forum mainly in person.
The main theme of this forum is “Toward the Cure of Hereditary Diseases”. Gene therapy for genetic diseases is currently being steadily approved worldwide. Gene therapy product for spinal muscular atrophy have already been approved and marketed in Japan, and those for Inherited Retinal Dystrophy, hemophilia, β-Thalassemia, sickle cell anemia, ADA deficiency, adrenoleukodystrophy, and metachromatic leukodystrophy have been approved in the United States and Europe. On the other hand, serious complications have been reported with lentiviral vectors and Adeno-associated viral vectors, which were previously considered safe, and it is time to discuss safety as well as efficacy again.
In this forum, we hope to share the latest knowledge and development information on gene and cell therapy for genetic diseases and related fields, and to discuss the possibility of cure. And in the current situation where gene therapy is becoming more practical than ever before, we also hope to share a lot of information and have meaningful discussions with medical staff such as certified genetic counselor or certified nurse specialist in genetics nursing, as well as with corporate staff who are considering the development of gene therapy.
Thank you in advance for your cooperation.
Hiroshi Kobayashi M.D.
The Jikei University School of Medicine
9:10-9:40 session 1 神経疾患に対する遺伝子治療：Gene Therapy for Neurological disorder
Chairs:
山形 崇倫
Gene therapy for AADC deficiency Paul Whu-Liang Hwu（National Taiwan University）
9:40-10:40 session 2 遺伝子編集とmRNA医薬：Gene Editing and mRNA Therapeutics
Chairs:
村松 一洋、村松 慎一
1) In vivo genome editing treatment for monogenic diseases 大森 司（自治医科大学）
2) ｍRNA Therapeutics（モデルナ社） ① mRNA Platform Introduction Patrick Finn（VP, Rare Research & Preclinical Development, Moderna, Inc.）WEB ② Development of mRNA therapeutics for propionic acidemia and methylmalonic acidemia 斎藤 格 Kaku Saito（Senior Director, Clinical Development, Moderna Japan Co., Ltd.）
10:40-11:40 session 3 遺伝性網膜疾患に対する遺伝子治療：Gene Therapy for Inherited Retinal Dystrophy
Chairs:
池田 康博、西口 康二
1) Startup-driven development of gene therapy for vision restoration using optogenetics 堅田 侑作（慶應大学医学部）
2) Social implementation of gene therapy for inherited retinal dystrophy in Japan 藤波 芳（東京医療センター）
11:40-11:50 準備
11:50-12:40 session 4（共催セミナー1） 遺伝性難聴に対するAAV ベクターを用いた遺伝子治療：Luncheon Seminar 【Takara Bio Inc.】: AAV mediated Gene Therapy for Congenital Deafness
Chairs:
小野寺 雅史
Development of AAV Gene Therapy Targeting GJB2 Related Hearing Loss by Capsid and Promoter Modification 神谷 和作（順天堂大学耳鼻咽喉科学講座）
12:40-12:50 休憩 / 回収
12:50-13:50 session 5 先天代謝異常症に対する ex vivo 遺伝子治療：Ex vivo Gene Therapy for Inborn Error of Metabolism
Chairs:
樋口 孝、奥山 虎之
1) Hematopoietic stem cell gene therapy for mucopolysaccharidosis type II 嶋田 洋太（東京慈恵会医科大学 遺伝子治療研究部）
2) Atidarsagene Autotemcel (Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-up Kent Christopherson（Executive Director and Head, Global Medical Affairs, Orchard Therapeutics）協和キリン社
13:50-14:50 session 6 先天代謝異常症に対するIn vivo 遺伝子治療：In vivo Gene Therapy for Inborn Error of Metabolism
Chairs:
大橋 十也、鐘ヶ江裕美
1) Results from ongoing gene therapy clinical trials in patients with rare diseases: MPS IIIa, GSDIa, OTC Deficiency, and Wilson Disease Eric Crombez（Ultragenyx Pharmaceutical Inc. Novato, California United States）
2) Complete normalisation of GM1 ganglioside storage through AAV expressing a blood-brain barrier-penetrating enzyme in mice 松島 小貴（東京慈恵会医科大学 遺伝子治療研究部）
14:50-15:00 準備・休憩
15:00-15:50 session 7（共催セミナー２） 銅代謝異常に対する遺伝子治療の試み：Sweets Seminar【Vector Builder Japan Inc.】 ：Trial of Gene Therapy for Inborn Error of Copper Metabolism
Chairs:
小須賀 基通
1) Potent Gene Therapy development for Menkes Disease: Preclinical Data and First-in-Human Study. Miho Matakatsu（VectorBuilder Inc., VectorBuilder Japan Inc.）
2) Accelerating Gene Therapy Development with VectorBuilder. Miho Matakatsu（VectorBuilder Inc., VectorBuilder Japan Inc.）
15:50-16:50 session 8 神経筋疾患に対する遺伝子治療：Gene Therapy for neuromuscular disorder
Chairs:
小坂 仁、岡田 尚巳
1) Roche's reflection on preparing for a future with gene therapy in Duchenne muscular dystrophy Alex Murphy（ロシュ社臨床開発担当者）WEB
2) Clinical development of Onasemnogene abeparvovec and Voretigen neparvovec （オナセムノゲン アベパルボベク、ボレチゲン ネパルボベクの臨床開発） 土田 健（Ken Tsuchida）、伊藤 紗織（Saori Ito） （ノバルティスファーマ株式会社 神経科学・遺伝子治療臨床開発） （Novartis Pharma K.K Neuroscience & GTx Clinical Development）
Guiding the Path of Gene Therapy -Supporting Patients and Families- 稲垣 夏子（Natsuko Inagaki）
（東京医科大学 循環器内科学分野 Tokyo Medical University, Department of Cardiology 東京医科大学病院 遺伝子診療センター Tokyo Medical University Hospital, Clinical Genetics Center）
17:40-18:30 session 10 特別講演 ヨーロッパ遺伝子細胞治療学会理事長Naldini教授による特別講演：Special Lecture by Professor Luigi Naldini
Chairs:
：衛藤 義勝
Precision Genetic Engineering of Hematopoiesis to Treat Human Disease Prof. Luigi Naldini
Please register from above Registration button.
We are kindly ask you to pay by credit.
Registration from November 13th, 2024 to January 23, 2025 until 5:00 P.M.
