Program :: ICIEM 2025 (International Congress of Inborn Errors of Metabolism 2025)

Program

Time Table

*Please note that the program is subject to change.

Time Table

Keynote Speech 1

September 3 (Wed), 10:00-10:45 [Room 1]

My Life in the Mitochondria
Chair	Yoshikatsu Eto (Southern Tohoku Institute for Neuroscience/Tokyo Jikei University School of Medicine, Japan)
Speaker	John Walker, Nobel Laureate in Chemistry (1997), (University of Cambridge, UK)

Keynote Speech 2

September 4 (Thu), 9:15-10:00 [Room 1]

A Systems Approach to Mitochondrial Disorders
Chair	Kimitoshi Nakamura (Kumamoto University, Japan)
Speaker	Vamsi Krishna Mootha (Massachusetts General Hospital, Harvard Medical School, USA)

Keynote Speech 3

September 5 (Fri), 9:15-10:00 [Room 1]

Molecular mechanisms and physiological roles of autophagy
Chair	Fumio Endo (Kumamoto Ezuko Medical Center, Japan)
Speaker	Noboru Mizushima (The University of Tokyo, Japan)

Plenary Session 1

September 4 (Thu), 10:00-10:30 [Room 1]

Empowering Biomedical Discovery with "AI Scientists"
Chair	Marshall Summar(Children's National, Vanderbilt University , Uncommon Cures LLC, USA)
Chair	Barbara Yu (Citrin Foundation, Singapore and UK)
Speaker	Marinka Zitnik (Harvard University, Harvard Medical School, USA)

Plenary Session 2

September 4 (Thu), 10:30-11:00 [Room 1]

Harnessing allosteric regulation of metabolic enzymes for novel drug discovery
Chair	Sean Froese (University Children’s Hospital Zurich, Switzerland)
Speaker	Wyatt Yue (Newcastle University, UK)

Plenary Session 3

September 4 (Thu), 11:00-11:30 [Room 1]

Role of urea cycle flux studies for preclinical research
Chair	Nicholas Ah Mew (Children's National Hospital, USA)
Speaker	Johannes Häberle (Division of Metabolism and Children’s Research Center, University Children’s Hospital Zurich, Switzerland)

Plenary Session 4

September 4 (Thu), 11:30-12:00 [Room 1]

The Future of Specific Advanced Treatments for Inborn Errors of Metabolism
Chair	Charles P. Venditti (National Institutes of Health, USA)
Speaker	Emil D. Kakkis (Ultragenyx, USA)

Plenary Session 5

September 6 (Sat), 9:00-9:30 [Room 1]

Finding mitochondrial disease-associated proteins through systems biochemistry
Chair	Kei Murayama (Department of Pediatrics and Diagnostics and Therapeutics of Intractable Disease, Intractable Disease Research Center, Juntendo University Faculty of Medicine, Japan)
Speaker	David J. Pagliarini (HHMI/Washington University School of Medicine in St. Louis, USA)

Plenary Session 6

September 6 (Sat), 9:30-10:00 [Room 1]

Long-Term Efficacy of Genome Editing in Infant Mice With Glycogen Storage Disease Type Ia
Chair	Takashi Hamazaki (Metropolitan University School of Medicine, Japan)
Speaker	Dwight Koeberl (Duke University School of Medicine, USA )

Plenary Session 7

September 6 (Sat), 10:00-10:30 [Room 1]

Mucolipidosis II-related diseases: impact of the mannose 6-phosphate pathway
Chair	Eva Morava (Icahn School of Medicine at Mount Sinai, USA)
Speaker	Thomas Braulke (University Medical Center Hamburg-Eppendorf; Institute of Osteology & Biomechanics; Cell Biology of Rare Diseases, Germany)

Symposium 1 : Cutting-Edge Basic Research

September 3 (Wed), 11:00-12:30 [Room 1]

Chair	Yair Anikster (Safra Children's Hospital, Sheba Medical Center, Israel)
Chair	Ljubica Caldovic (Children's National Hospital, USA)
Speaker	Hudson H. Freeze (Sanford Burnham Prebys Medical Discovery Institute, USA)
	Hirohide Saito (RNP Synthetic Biology and Biotechnology Institute for Quantitative Biosciences, The University of Tokyo, and Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan)
	Knut Woltjen (Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan)
	Yousuke Katsuda (Faculty of Advanced Science and Technology, Kumamoto University, StapleBio Inc., Japan)

Symposium 2 : Gene therapy from Japan, the Land of Tradition

September 3 (Wed), 11:00-12:30 [Room 2]

Chair	Kimihiko Oishi (Department of Pediatrics, The Jikei University School of Medicine, Japan)
Chair	Gerard Berry (Boston Children's Hospital/Harvard Medical School, USA)
Speaker	Masafumi Onodera (Graduate School of Engineering, The University of Osaka)
	Kazuhiro Muramatsu (Jichi Medical University, Department of Pediatrics and Center for Gene Therapy Research, Japan)
	Hiroshi Kobayashi (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Japan)
	Akihiro Kume (Jichi Medical University, Japan)

Symposium 3 : Global Trends in Gene Therapy

September 3 (Wed), 14:00-15:30 [Room 1]

Chair	Mireia Del Toro (Pediatric Neurology, Hospital Universitario Vall d'Hebron, Spain)
Chair	Gerry Lipshutz (University of California, Los Angeles, USA)
Speaker	Stacy E Croteau (Boston Children's Hospital, USA)
	Haiyan Zhou (University College London, UK)
	J Lawrence Merritt, II (Ultragenyx Pharmaceutical, USA)
	Kent Lai (University of Utah, USA)

Symposium 4 : Transformation in Newborn Screening

September 3 (Wed), 14:00-15:45 [Room 2]

Chair	Susan Berry (University of Minnesota, USA)
Chair	Sarah Viall (Oregon Health & Science University, USA)
Speaker	Patrick Forny (Washington University School of Medicine in St. Louis, USA)
	Osamu Ohara (Kazusa DNA Research Institute, Japan)
	Rohit Sharma (Massachusetts General Hospital, USA)
	Dau-Ming Niu (Institute of Clinical medicine, National Yang Ming Chiao Tung University / Rare Disease Medical Research Center, Taipei Veterans General Hospital, Taiwan)
	Yin-Hsiu Chien (National Taiwan University Hospital, Taiwan)

Symposium 5 : Cutting-Edge Advances in Lysosomal Disorders

September 4 (Thu), 15:15-17:30 [Room 1]

Chair	Marc Patterson (Mayo Clinic Rochester MN; IntraBio Inc, USA)
Chair	Nicola Brunetti-Pierri (Telethon Institute of Genetics and Medicine, Italy)
Speaker	Pramod K. Mistry (Yale University School of Medicine, USA)
	Priya S. Kishnani (Duke University Medical Center, USA)
	Roberto Giugliani (UFRGS, HCPA, DASA, CASA DOS RAROS, Brazil)
	Aurora Pujol (Bellvitge Biomedical Research Insitute (IDIBELL), Spain)
	Maurizio Scarpa (Azienda Sanitaria Universitaria Friuli Centrale, Italy)
	Isaac Canals (University of Zurich - Kinderspital Zürich, Switzerland)

Symposium 6 : Frontiers in Amino Acid Metabolism Disorders

September 4 (Thu), 15:15-17:30 [Room 2]

Chair	Tetsuya Ito (Department of Pediatrics, Fujita Health University School of Medicine, Japan)
Chair	Philippa Mills (University College London, Great Ormond Street Institute of Child Health, UK)
Speaker	Nicola Longo (University of California Los Angeles (UCLA), USA)
	Johan L.K. Van Hove (University of Colorado, USA)
	Juan Francisco Cabello (INTA, University of Chile, Chile)
	Yukihito Ishizaka (Japan Institute for Health Security, Japan)
	Ayelet Erez (Weizmann Institute of Science, Israel)
	Rebecca Ahrens-Nicklas (The Children’s Hospital of Philadelphia and University of Pennsylvania, USA)

Symposium 7 : Frontiers in Organic Acidemias and Fatty Acid Oxidation Disorders

September 5 (Fri), 10:15-11:55 [Room 1]

Chair	Chong Kun Cheon (Pusan National University School of Medicine, Republic of Korea)
Chair	Shirou Matsumoto (Kumamoto University Hospital, Japan)
Speaker	Jerry Vockley (University of Pittsburgh School of Medicine, USA)
	Stefan Kölker (Heidelberg University Hospital and Heidelberg University, Medical Faculty, Centre for Pediatrics and Adolescent Medicine, Germany)
	Sufin Yap (Sheffield Children’s Hospital, UK)
	Sean Froese (University Children’s Hospital Zurich, Switzerland)
	Charles P. Venditti (National Institutes of Health, USA)

Symposium 8 : Advances in iPS Cell Research

September 5 (Fri), 10:15-11:45 [Room 1]

Chair	Hiroshi Kobayashi (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Japan)
Chair	Andrea Dardis (University Hospital of Udine, Italy)
Speaker	Hideyuki Okano (Keio University Regenerative Medicine Research Center (KRM), Japan)
	Haruhisa Inoue (Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan)
	Takumi Era (Department of Cell Modulation, Institute of Molecular Embryology and Genetics, Kumamoto University, Japan)
	Norifumi Shioda (Kumamoto University, Japan)

Symposium 9 : Late-Breaking Research

September 6 (Sat), 10:30-12:00 [Room 1]

Chair	TBD
Speaker	Gerry Lipshutz (University of California, USA)
	Simon Allan Jones (Manchester University NHS Foundation Trust, UK)
	Peter J. McGuire (National Human Genome Research Institute, NIH, USA)
	Tomoko Nakanishi (Center for Biomedical Research Resources, Juntendo University Graduate School of Medicine, Japan)
	Kristina L. Elvidge (Childhood Dementia Initiative, Australia)
	Abdullah Hoter (Department of Biochemistry, University of Veterinary Medicine Hannover, Germany)

Symposium 10 : Asian Young Investigator Award

September 6 (Sat), 10:30-12:00 [Room 2]

Chair	TBD
Speaker	Yu-Ting Chiang (Institute of Clinical Medicine, National Yang Ming Chiao Tung University, Taiwan)
	Mai Thi Thanh Do (Vietnam National Children's Hospital, Vietnam)
	Mildrid Yeo (Genetics Service, Department of Paediatrics, KK Women's and Children's Hospital, Singapore)
	Ji-Hee Yoon (Department of pediatrics, Bundang Jesaeng Hospital, South Korea)
	Parith Wongkittichote (Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Thailand)
	Swasti PAL (Sir Ganga Ram Hospital, India)

Special Symposium : From Pathogenesis to Therapy, From Japan to the World: Global Translation, Metabolic Flux, and Gene Editing for Citrin Deficiency

September 3 (Wed), 14:00-16:00 [Room 6]

Chair	Kimitoshi Nakamura (Kumamoto University, Japan)
Chair	Barbara Yu (Citrin Foundation, Singapore and UK)
Speaker	Johannes Häberle (University Children’s Hospital, University of Zürich, Switzerland)
	Marc Hellerstein (University of California, Berkeley, USA)
	Gerald Schwank (University of Zürich, Switzerland)
	Jun Kido (Kumamoto University, Japan)
	Yoichi Wada (Tohoku University, Japan)
	Barbara Yu (Citrin Foundation, Singapore and UK)

Memorial Session for Prof. Toshiyuki Fukao

September 6 (Sat), 8:30-10:00 [Room 5]

Chair	Hiroyuki Ida (The Trustee of The Jikei University, Japan)
Chair	Grant Mitchell (Division of Medical Genetics and Genomics, Department of Pediatrics, CHU Sainte Justine and Université de Montréal, Canada)
Speaker	Kimitoshi Nakamura (Kumamoto University, Japan)
	Grant Mitchell (Division of Medical Genetics and Genomics, Department of Pediatrics, CHU Sainte Justine and Université de Montréal, Canada)
	Sasai Hideo (Department of Pediatrics/ Department of Early Diagnosis and Preventive Medicine for Rare and Intractable Pediatric Diseases, Graduate School of Medicine, Gifu University, Japan)
	Jörn Oliver Sass (RG Inborn Errors of Metabolism, Department of Natural Sciences & Institute for Functional Gene Analytics (IFGA), Bonn-Rhein-Sieg University of Applied Sciences, Rheinbach, Germany)

Garrod Award Lecture

September 3 (Wed), 17:30-18:00 [Room 1]