The 12th International Collaborative Forum of
Human Gene Therapy for Genetic Disease
Expanding clinical application of gene therapy for genetic diseases

Jan. 20（Thu.）, 2022 9：00−17：40 ※Hybrid Meeting

Date

The Jikei University School of Medicine, Building 1 Hall

Venue
President
Registration FeeTakanori Yamagata (Professor, Department of Pediatrics, Center for Gene Therapy Research, Jichi Medical University School of Medicine)
Regular: 3,000 JPY

Company: 10,000 JPY Student: Admission free (with veriﬁcation card)※In regarding to the registration, please check above URLor ask to the Secretariat.
※Registration: Dec. 15, 2021−Jan. 19, 2022
TimeProgram

9：00〜 9：15

Opening Remarks

9：15〜10：15

Clinical experience of AAV-based gene therapy1) Onasemnogene abeparvovec (ZolgensmaⓇ) for Spinal Muscular Atrophy:
Clinical and Real World Experience
Sandra P. Reyna (Novartis Gene Therapies, USA)

Takanori Yamagata (President of the 12th GT Forum)
Yoshikatsu Eto (Executive Chairperson of the Committee)

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
Gene Therapy and Genome Editing for Human Genetic Diseases
James M. Willson (University of Pennsylvania, USA)

11：00〜11：40

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

１１:40〜12：40

Gene therapy for Haemophilia
1) Clinical Application of Gene Therapy for Hemophilia: Experience withﬁdanacogene elaparvovec and giroctocogene ﬁtelparvovec
Gregory Di Russo (Pﬁzer Inc., USA)2) Gene therapy of hemophilia A

Kevin Eggan (BioMarin Pharmaceutical Inc., USA)12：45〜13：45

Luncheon Seminar
Takara Bio's eﬀorts to address gene therapy vectors
Tatsuji Enoki (Takara Bio Inc., Japan)

13：55〜14：25

Eﬀorts on Gene and Cell Therapy Projects at AMED
Michiko Takakura (AMED, Japan)

１4:25〜15：25

Gene therapy for AADC deﬁciency
1) Gene therapy for AADC deﬁciency in Japan -Improved motor and mental
function of various phenotype patients
Karin Kojima (Jichi Medical University, Japan)
2) Gene therapy for AADC deﬁciency in Taiwan
Wuh-Liang Hwu (National Taiwan University Hospital, Taiwan)

15：40〜16：40

Ex vivo Gene therapy
1) Hematopoietic stem cell gene therapy with a lentiviral gene delivery
Naoya Uchida (The Institute of Medical Science, The University of Tokyo, Japan)

2) Orchard Therapeutics and its Gene Therapy Programs
Christopher Dott (Orchard Therapeutics Ltd., UK)

16：40〜17：20

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

17：20〜17：40

Closing Remarks

Ryuichi Morishita (President of the JSGCT)【Organizing】 International Collaborative Forum of Human Gene Therapy for Genetic Disease【Support】
Japan Society of Gene and Cell Therapy【Co-sponsored】NPO Japan Lysosomal Disease Research Center
Organizer:
Secretariat:

Department of Pediatrics, Jichi Medical University School of Medicine
3311-1 Yakushiji, Shimotsuke-shi, Tochigi 329-0496 Japan
c/o SK Apex Plan Co., Ltd.
SS Bldg. 5F, 1-8-6 Shintomi, Chuo-ku, Tokyo 104-0041, Japan Tel：+81-3-3523-3722 Fax：+81-3-3523-3723 E-mail：info@skap.jp

http://www.gt-forum.net/[International Collaborative Forum of
Human Gene Therapy for Genetic Disease]