第12回国際協力遺伝病遺伝子治療フォーラム

Live配信　2022年1月20日(木) 09:00〜18:00

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Changed Program

Information（開催概要）

President 当番幹事: Takanori Yamagata
山形崇倫（自治医科大学小児科学主任教授）
Date 開催日: Thursday, January 20th, 2022 ＊Hybrid Meeting
2022年1月２０日（木）　＊現地開催& Live配信
Venue 会　場: The Jikei University School of Medicine, Building 1 Hall
東京慈恵会医科大学 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: Expanding clinical application of gene therapy for genetic diseases
飛躍する、遺伝病に対する遺伝子治療の臨床応用
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: Japan Society of Gene and Cell Therapy
NPO法人日本ライソゾーム病研究センター
Co-sponsored 後　援: NPO Japan Lysosomal Disease Research Center
一般社団法人日本遺伝子細胞治療学会

Greetings（ご挨拶）

The 12th International Collaborative Forum of Human Gene Therapy for Genetic Disease

Welcome Greetings

It is our honor to organize the 12th International Collaborative Forum of Human Gene Therapy for Genetic Disease.

The development of gene therapy for a wide range of genetic diseases has been on the rise worldwide. The main procedures available at present are in vivo gene therapy using an adeno-associated viral (AAV) vector and ex vivo gene therapy using a lentiviral vector. Intravenous Onasemnogene abeparvovec (zorgensma) treatment using an AAV9 vector for spinal muscular atrophy was approved in Japan last year and has since shown marked efficacy for patients treated early. In addition, the advent of this treatment has helped familiarize many physicians with gene therapy as a whole. Following the development of Onasemnogene abeparvovec, progress in AAV gene therapies for several diseases, including neurological diseases, such as AADC deficiency, congenital metabolic diseases (mainly lysosomal diseases), and hemophilia has been achieved, and clinical trials are underway. Ex vivo gene therapies using lentiviral vector have been developed for immune deficiency as well as for neuronal and metabolic diseases, including adrenoleukodystrophy, metachromatic leukodystrophy and Hurler disease.

We invited global front-runners in the clinical application of gene therapy to participate in this forum. It is our great pleasure to have them here to inform us about the progress and benefits of gene therapy.

Gene therapy is a promising therapy, but some issues remain to be addressed. Massive intravenous AAV gene therapy induced liver dysfunction and thrombocytopenia in some patients, albeit transiently. Myelodysplastic syndrome was observed in some patients in lentiviral gene therapy. We have also invited prominent researchers to tell us more about gene therapy, including its mechanism of action, expression control, and associated adverse events. We also hope to learn more about the available support from the government for gene therapy.

We would enjoy hearing lectures directly from researchers and discussing their work in a face-to-face manner, but we understand that is difficult in the current global situation. Therefore, we plan to hold this forum in a hybrid system. Please join the forum either in person or attend via the web. We hope this forum will be a good chance to learn about the worldwide progress of gene therapy, improving international/academic and pharmaceutical cooperation and stimulating further research in this field in Japan.

We thank the participants as well as the individuals and companies who have supported this forum.

Sincerely Yours,

Takanori Yamagata
President of the 12th International Collaborative Forum of Human Gene Therapy for Genetic Disease．
Professor,
Department of Pediatrics, Jichi Medical University

Download of Program & Abstracts（抄録集ダウンロード）

Program（プログラム）

9:00～9:15　Opening Remarks (開会挨拶)

Takanori Yamagata（President of the 12th GT Forum）
山形　崇倫（第12回国際協力遺伝病遺伝子治療フォーラム当番幹事）
Yoshikatsu Eto(Executive Chairperson of the Committee)
衞藤　義勝（国際協力遺伝病遺伝子治療フォーラム代表幹事）

9:15～10:15　Clinical experience of AAV-based gene therapy（AAVを用いた遺伝子治療の臨床）

Chairs:: Torayuki Okuyama (National Center for Child Health and Development) /
奥山虎之（国立成育医療研究センターライソゾーム病センター）; Kazuhiro Muramatsu (Jichi Medical University) /
村松　一洋（自治医科大学小児科学）

1) Onasemnogene abeparvovec (Zolgensma®) for Spinal Muscular Atrophy:
Clinical and Real World Experience
Sandra P. Reyna（Novartis Gene Therapies, USA）
2) Intracisternal AAV9-based Gene Therapy for Neuronopathic Lysosomal Storage Diseases:
Experience from a Clinical Trial Program
Marie-Laure Névoret (REGENXBIO, Inc., USA)

10:15～10:55　Special lecture（特別講演）

Chair:: Keiya Ozawa (Jichi Medical University) /
小澤　敬也（自治医科大学免疫遺伝子細胞治療学（タカラバイオ）講座）

Gene therapy using AAV vector
James M. Willson（University of Pennsylvania, USA）

11:00～11:40　Special lecture（特別講演）

Chair:: Yoshikatsu Eto (Institute of Neurological Disorders) /
衛藤　義勝（脳神経疾患研究所附属先端医療センター＆遺伝病治療研究所）

Advancing gene therapies for the brain
Beverly L. Davidson(University of Pennsylvania, USA)

11:40～12:40　Gene therapy for Haemophilia（血友病の遺伝子治療）

Chairs:: Toya Ohashi(The Jikei University School of Medicine) /
大橋　十也（東京慈恵会医科大学医学部看護学科）; Toshinao Kawai(National Center for Child Health and Development) /
河合　利尚（国立成育医療研究センター小児内科系専門診療部　免疫科）

1) Clinical Application of Gene Therapy for Hemophilia:
Experience with fidanacogene elaparvovec and giroctocogene fitelparvovec
Gregory Di Russo (Pfizer Inc., USA)
2) Gene therapy of hemophilia A
Kevin Eggan(BioMarin Pharmaceutical Inc., USA)

12:40～13:30　Lunch Break(昼食)

13:30～14:15　Luncheon Seminar（ランチョンセミナー）

Chair:: Takanori Yamagata(Jichi Medical University) /
山形　崇倫（自治医科大学小児科学）

Takara Bio's efforts to address gene therapy vectors（遺伝子治療用ベクターに対するタカラバイオの取り組み）
Tatsuji Enoki(Takara Bio Inc., Japan)
榎　竜嗣

14:20～14:50　Efforts on Gene and Cell Therapy Projects at AMED

Chair:: Takashi Shimada(AMED/PMDA) /
島田　隆（AMEDプログラムオフィサー、PMDA専門委員）

Michiko Takakura (AMED, Japan)
高倉美智子（国立研究開発法人日本医療研究開発機構）

14:50～15:50　Gene therapy for AADC deficiency

Chairs:: Shinichi Muramatsu(Jichi Medical University) /
村松　慎一（自治医科大学神経遺伝子治療部）; Hitoshi Osaka(Jichi Medical University) /
小坂　仁（自治医科大学小児科学）

1) Gene therapy for AADC deficiency in Japan -Improved motor and mental function of various phenotype patients
Karin Kojima (Jichi Medical University, Japan)
小島　華林（自治医科大学小児科学）
2) Gene therapy for AADC deficiency in Taiwan
Wuh-Liang Hwu(National Taiwan University Hospital, Taiwan)

16:00～17:00　Ex vivo Gene therapy（Ex vivo 遺伝子治療）

Chairs:: Hiroshi Kobayashi(The Jikei University School of Medicine) /
小林　博司（東京慈恵会医科大学遺伝子治療研究部）; Toru Uchiyama(National Center for Child Health and Development) /
内山　徹（国立成育医療研究センター成育遺伝研究部）

1) Hematopoietic stem cell gene therapy with a lentiviral gene delivery
Naoya Uchida (The Institute of Medical Science, The University of Tokyo, Japan)
内田　直也（東京大学医科学研究所遺伝子・細胞治療センター）
2) Orchard Therapeutics and its Gene Therapy Programs
Christopher Dott (Orchard Therapeutics Ltd., UK)

17:00～17:40　Special lecture（特別講演）

Chair:: Masafumi Onodera(National Center for Child Health and Development) /
小野寺　雅史（国立成育医療研究センター遺伝子細胞治療推進センター）

Hematopoietic stem cell gene therapy for inborn errors of immunity and metabolism
Aiuti Alessandro (San Raffaele Telethon Institute for Gene Therapy, Italy)

17:40～18:00　Closing Remarks（閉会挨拶）

Ryuichi Morishita(President of the JSGCT, Osaka University)
森下　竜一（一般社団法人日本遺伝子細胞治療学会理事長、大阪大学大学院医学系研究科臨床遺伝子治療学）

A little bit of respect（敬称略）

Download of Poster（ポスターダウンロード）

English version

日本語版

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Registration Fee（参加費）

Regular （一般）: ￥ 3,000.-
Student （学生）: Admission free (with verfication card)
無料（要・学生証提示）
Company （企業）: ￥ 10,000.-

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