第14回国際協力遺伝病遺伝子治療フォーラム

What’s New（お知らせ）

2024.01.26: 第14回国際協力遺伝病遺伝子治療フォーラムは盛会裏にて終了いたしました。
たくさんのご参加ありがとうございました。
2023.10.12: ホームページを公開しました。

Information（開催概要）

President 当番幹事: Yasuhiro Ikeda
池田康博（宮崎大学医学部眼科学）
Date 開催日: Thursday, January 25th, 2024 ＊Venue Meeting
2024年1月25日（木）　＊現地開催
Venue 会　場: The Jikei University School of Medicine, Building 1 Hall
東京慈恵会医科大学大学1号館（3階）講堂
Location 場　所: 3-25-8, Nishi-Shimbashi, Minato-ku, Tokyo, 105-8461
東京都港区西新橋3-25-8
Theme テーマ: Clinical practice of genomic medicine for intractable genetic diseases - The light at the end of the tunnel for patients
遺伝性難病に対するゲノム医療の実践～患者の希望の光～
Organizing 主　催: International Collaborative Forum of Human Gene Therapy for Genetic Disease
国際協力遺伝病遺伝子治療フォーラム実行委員会
Support 共　催: NPO Japan Lysosomal Disease Research Center
NPO法人日本ライソゾーム病研究センター
Co-sponsored 後　援: Japan Society of Gene and Cell Therapy (JSGCT)
一般社団法人日本遺伝子細胞治療学会（JSGCT） Japanese Society for Genetic Counseling
一般社団法人日本遺伝カウンセリング学会 The Japan Society of Human Genetics
一般社団法人日本人類遺伝学会 Japanese Society for Gene Diagnosis and Therapy
日本遺伝子診療学会 Japanese Retina and Vitreous Society
日本網膜硝子体学会

Greetings（ご挨拶）

The 14th International Collaborative Forum of Human Gene Therapy for Genetic Disease

Welcome Greetings

It is our honor to organize the 14^th International Collaborative Forum of Human Gene Therapy for Genetic Disease on January 25^th, 2024, at Tokyo Jikei University School of Medicine.

In my field of ophthalmology, many clinical trials of gene therapy for ocular genetic diseases are now underway worldwide. Voretigene neparvovec-rzyl (Luxturna^TM) was approved as the first US Food and Drug Administration (FDA)-approved gene therapy product for a genetic disease on December, 2017. Luxtuna^TM has already been administrated to over 400 patients with RPE65-associated retinal dystrophy (Leber congenital amaurosis) all over the world. Finally, Luxtuna^TM has recently been approved, and is now available, in Japan. In addition to this approval, the gene panel testing system for inherited retinal dystrophy (PrismGuide^TM IRD Panel System) was also approved in Japan.

The main theme of the forum is “Clinical practice of genomic medicine for intractable genetic diseases - The light at the end of the tunnel for patients”. This forum will focus on the clinical application of gene and cell therapy and genetic testing for intractable genetic diseases, including not only ocular diseases, but also neuromuscular diseases, inherited metabolic diseases, and genetic blood disorders. We will invite the leading researchers and clinician scientists in each field to inform us about the most up-to-date information and problems in their clinical settings. We hope that this forum will provide fruitful opportunity to learn about developments of the treatment for patients with intractable genetic diseases. Therefore, we are looking forward to welcoming many participants, including researchers and clinicians from various fields.

Sincerely yours,

Yasuhiro Ikeda
President of the 14th International Collaborative Forum of Human Gene Therapy for Genetic Disease,
Department of Ophthalmology, Faculty of Medicine, University of Miyazaki

Program（プログラム）

9:00-9:05　
Opening Remarks

Yasuhiro Ikeda (President of the 14th GT Forum)

9:10-10:10　
神経疾患の遺伝子治療：Gene therapy for neurological diseases

Chairs:: Karin Kojima (Jichi Medical University); Shin-Ichi Muramatsu (Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University)

1) Gene Therapy for ALS: The Future is Coming Fast
Defne Amado (Perelman School of Medicine, University of Pennsylvania)
2) Breaking down barriers: Bringing a gene therapy for AADC deficiency
Rafael Sierra (PTC Therapeutics Inc Exec. Director, Head of Gene Therapy Global Medical Affairs)

10:15-11:15　
ベクター製造：Vector manufacturing

Chairs:: Takashi Okada (Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo); Masafumi Onodera (Gene ＆ Cell Therapy Promotion Center, National Center for Child Health and Development)

1) Viral Vector manufacturing processes for Gene Therapy
Margherita Neri (AGC Biologics）
2) Two-way strategies for comprehensive AAV mass production
Taichi Muraguchi (Bio Science & Engineering Laboratories, FUJIFILM)

11:20-12:10　
Special Lecture

Chairs:: Yasuhiro Ikeda (Department of Ophthalmology, Faculty of Medicine, University of Miyazaki)

1) Ocular Gene Therapy and Surgery: Today and Future Horizons
Andreas K. Lauer (Casey Eye Institute）

12:20-13:20　
Luncheon Seminar (Sysmex Corp.)

Chairs:: Mineo Kondo (Department of Ophthalmology, Mie University Graduate School of Medicine); Takashi Nakagawa (Department of Otorhinolaryngology, Graduate School of Medical Sciences, Kyushu University)

1) Standardized genetic analysis for inherited retinal disease in Japan
Koji M. Nishiguchi (Department of Ophthalmology Nagoya University Graduate School of Medicine)
2) Innovation of clinical practice for hearing loss by implementation of large-scale genetic test
Tatsuo Matsunaga (Div. of Hearing and Balance Research, National Institute of Sensory Organs / Medical Genetics Center, NHO Tokyo Medical Center)

13:30-14:30　
血液凝固疾患の遺伝子治療：Gene therapy for hemophilia and congenital neutropenia

Chairs:: Hiroshi Kobayashi (The Jikei University School of Medicine); Toru Uchiyama (Gene ＆ Cell Therapy Promotion Center, National Center for Child Health and Development)

1) Molecular Characterization of AAV Gene Therapy, and International Cooperative Investigations
Kevin Eggan (BioMarin Pharmaceutical Inc.)
2) “OMNITM Technology Platform” Genome editing solution to make any gene targetable
Takeshi Harada (AnGes inc.)

14:40-15:40　
Afternoon Seminar (Takara Bio Inc.)

Chairs:: Motomichi Kosuga (Gene ＆ Cell Therapy Promotion Center, National Center for Child Health and Development)

1) Takara Bio’s CDMO service of viral vector manufacturing
Hideto CHONO (General Manager, CDM Center 5, Takara Bio Inc.)

15:50-16:50　
代謝性疾患の遺伝子治療：Gene therapy for metabolic diseases

Chairs:: Torayuki Okuyama (Saitama Medical University); Kazuhiro Muramatsu (Department of Pediatrics, Jichi Medical University)

1) INTERIM CLINICAL OUTCOMES AFTER HEMATOPOIETIC STEM CELL-GENE THERAPY for MUCOPOLYSACCHARIDOSIS TYPE I HURLER
Maria Ester Bernardo ("Vita-Salute San Raffaele" University Medical School)
2) Gene Therapy for Menkes Disease: Pre-Clinical Data and First-in-Human Study
Bruce T. Lahn (VectorBuilder Inc. and Lantu biopharma (Guangzhou) Co., Ltd.)

16:55-17:55　
Sponsored Seminar (Novartis Pharma) 眼疾患の遺伝子治療：Gene therapy for ocular diseases

Chairs:: Koji M Nishiguchi (Department of Ophthalmology, Nagoya University Graduate School of Medicine); Tsutomu Igarashi (Department of Ophthalmology, Nippon Medical School Chiba Hokusoh Hospital)

1) Inherited retinal dystrophy: diagnosis and treatment in Japan
Kaoru Fujinami（Laboratory of Visual Physiology, Division of Vision Research, National Institute of Sensory Organs, NHO Tokyo Medical Center, Tokyo, Japan.)
2) Ocular gene therapy in Europe: Voretigene neparvovec and beyond
Bart Peter Leroy (Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium)

18:00-18:10　
Closing Remarks

Ryuichi Morishita (President of the JSGCT / Osaka University)
Hiroshi Kobayashi (President of the 15th GT Forum / The Jikei University School of Medicine)

Registration Fee（参加費）

Regular （一般）: ￥5,000.-
Student （学生）: Admission free (with verification card)
無料（要・学生証提示）
Company （企業）: ￥20,000.-

Reception （懇親会）: ￥5,000.-　*25^th January (Doors open 18:30) 　[THE CORE KITCHEN]

※参加費は「不課税」です（免税事業者のため事業者登録番号無し）

Registration（登録方法）

Please register from above Registration button.
We are kindly ask you to pay by credit.
Registration from Octorber 1, 2023 to January 22, 2024 untill 17:00.

Registration from here（参加登録はこちらから）

Inquiry（問い合わせ）