Sung Hee ChoiProf.

Professor Sung-Hee Choi, MD, PhD, is Chief Professor of Medicine at Seoul National University College of Medicine and Seoul National University Bundang Hospital (SNUBH), where she specializes in Endocrinology and Metabolism. She also serves as Director of the International Health Care Center at SNUBH. Professor Choi received her MD, MSc, and PhD degrees from Yonsei University College of Medicine and completed her clinical training at Yonsei University College of Medicine. She joined Seoul National University College of Medicine as a faculty member in 2004 and has held progressive academic appointments, becoming Associate Professor in 2012 and Professor in 2017. She undertook advanced research training as a Research Scholar at the Irving Institute for Clinical and Translational Research, Columbia University Medical Center, New York, USA (2009–2011), under Henry Ginsberg, where she focused on translational and clinical research in metabolic diseases. Professor Choi’s research interests encompass obesity, diabetes, cardiometabolic risk, and lipid disorders, on population-based epidemiology, biomarkers, adipose tissue biology, and clinical trials evaluating novel therapeutic strategies. Currently, her lab has been focused on human adipose tissue single cell and multi-omics research. She has played a leading role in national-scale epidemiological studies, including the Diabetes Fact Sheet in Korea, and has contributed significantly to research on insulin resistance, biomarkers for cardiovascular risk prediction, and metabolic complications. She has published more than 300 peer-reviewed articles in high-impact international journals and has delivered invited lectures at numerous national and international scientific meetings. Professor Choi is actively engaged in global professional leadership and currently serves as an Executive Board Member of the International Diabetes Federation – Western Pacific Region (IDF-WPR), an Executive Board Member of the Asia-Pacific region of the International Atherosclerosis Society, and a former strategic committee member of the World Health Organization. In addition, she has held multiple leadership roles within the Korean Diabetes Association, Korean Endocrine Society, Korean Obesity Society (KSSO), and the Korean Society of Lipid and Atherosclerosis. Professor Choi also serves in key editorial roles, including Associate Editor of Diabetes & Metabolism Journal and Editorial Board Member of Scientific Reports etc. She remains deeply committed to education, research excellence, and international collaboration in the field of endocrinology.

21 MARCH

Time	Session

08:30 10:00	[Symposium] Dyslipidemia New Development in Dyslipidemia Management Abbas RazaPakistan Moderator Obesity - MASLD Perspectives in Asia-Oceania Jun-Sing WangTaiwan Moderator Continuous Glucose Monitoring (CGM) in Asia: Behavior Change, Physician Workflow, and New Care Models Lipoprotein(a): What Endocrinologists Need to Know Kathryn TanHong Kong, China Speaker Lipoprotein(a): What Endocrinologists Need to KnowLipoprotein(a) [Lp(a)] is a cholesterol-rich LDL-like particle with apolipoprotein(a) covalently linked to apolipoprotein B100 via a disulfide bond. Lp(a) is synthesized within the liver and there is a general inverse correlation between Lp(a) isoform size and plasma Lp(a) concentrations. About 90% of plasma Lp(a) concentration is genetically determined and plasma levels can modestly rise after menopause in women, and in conditions like hypothyroidism, nephrotic syndrome. It has been shown that elevated Lp(a) is an independent risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve stenosis. Although Lp(a) concentration does vary with ethnicity, relationships between Lp(a) concentration and ASCVD risk remain similar across different ethnic groups. Elevated Lp(a) is considered a cardiovascular risk-enhancing or amplification factor, and recent guidelines and consensus have increasingly recommended universal screening for Lp(a). There are as yet, no approved therapies for elevated Lp(a). Current management focuses on intensifying control of concurrent risk factors, particularly LDL-C, to reduce ASCVD risk. Amongst existing lipid-lowering drugs, only proprotein convertase subtilisin/kexin type 9 inhibitors can lower Lp(a) levels modestly. Emerging RNA-based and small-molecule therapeutics are under development and are showing promising Lp(a)-lowering effects up to 80-90%. Ongoing phase 3 cardiovascular outcomes trials will determine whether effectively lowering Lp(a) can translate to cardiovascular benefit. A Novel Therapeutic Concept for Familial LCAT Deficiency: Long-Term Enzyme Replacement Using Genetically Modified Adipocytes Masayuki KurodaJapan Speaker A Novel Therapeutic Concept for Familial LCAT Deficiency: Long-Term Enzyme Replacement Using Genetically Modified AdipocytesFamilial lecithin:cholesterol acyltransferase (LCAT) deficiency is a rare autosomal recessive disorder marked by defective HDL maturation, leading to corneal opacity, hemolytic anemia, and progressive renal dysfunction. No disease-modifying treatment has been established to date. Conventional enzyme replacement requires repeated administration with limited durability. Glybera, the first AAV1-based gene therapy for lipoprotein lipase deficiency, was withdrawn after limited clinical use and modest benefit. More broadly, in vivo AAV gene therapies face challenges including immune responses, hepatotoxicity at high vector doses, and considerable inter-patient variability in transgene expression. Our therapeutic approach originated from studies in diabetic mouse models, where adipocytes were explored as platforms for sustained protein delivery. These cells demonstrated endocrine-like properties and long-term protein secretion in vivo. Adipocytes are particularly suited for this purpose due to their longevity, secretory capacity, and low tumorigenic risk. Building on these findings, we established an ex vivo gene and cell therapy platform using genetically modified adipocytes (GMAC), autologous adipocyte-derived cells engineered to express therapeutic proteins. As its first application, we targeted familial LCAT deficiency. These cells were expected to engraft upon subcutaneous implantation, re-differentiate into functional adipocytes, and provide long-lasting and therapeutically relevant LCAT secretion. In a first-in-human clinical trial conducted under Japan’s regulatory framework for regenerative medicine, mature adipocytes were collected from the patient’s subcutaneous fat, converted into proliferative cells via ceiling culture, and transduced to express therapeutic human LCAT, then administered subcutaneously to the patient. Single administration of LCAT-GMAC was well tolerated with no serious adverse events. Sustained increases in serum LCAT activity were observed, accompanied by improvements in lipoprotein profiles and hemolytic anemia. A marked reduction in proteinuria was noted, and renal function remained stable throughout the follow-up period. Remarkably, serum LCAT activity persisted for over eight years after the single administration, the longest durability ever reported for enzyme replacement. This provides the first clinical evidence that ceiling culture-derived, ex vivo modified adipocytes can achieve lasting correction of systemic enzyme deficiencies. LCAT-GMAC therapy thus offers a potentially curative strategy for familial LCAT deficiency and a new paradigm for treating dyslipidemias and other lifelong plasma protein deficiencies. Novel and Future Lipid-Lowering Therapy Sung Hee ChoiSouth Korea Speaker Novel and Future Lipid-Lowering TherapyIn this lecture, I want to introduce the mechanism of current developing lipid lowering drugs. Small molecular inhibitors such as bempedoic acid, oral forms of newer drugs, Anti-sense oligonucleotide drugs, and siRNA technique based new lipid lowering drugs and its clinical trials. These agents target diverse pathways such as proprotein convertase subtilisin/kexin type 9 (PCSK9), angiopoietin-like protein 3 (ANGPTL3), apolipoprotein C-III (apoC-III), and Lp(a), achieving potent lipid modulation in different mechanistic approach. 101

Time

Session

08:30

10:00

[Symposium] Dyslipidemia

New Development in Dyslipidemia Management

Abbas RazaPakistan Moderator Obesity - MASLD Perspectives in Asia-Oceania

Jun-Sing WangTaiwan Moderator Continuous Glucose Monitoring (CGM) in Asia: Behavior Change, Physician Workflow, and New Care Models

Lipoprotein(a): What Endocrinologists Need to Know

Kathryn TanHong Kong, China Speaker Lipoprotein(a): What Endocrinologists Need to KnowLipoprotein(a) [Lp(a)] is a cholesterol-rich LDL-like particle with apolipoprotein(a) covalently linked to apolipoprotein B100 via a disulfide bond. Lp(a) is synthesized within the liver and there is a general inverse correlation between Lp(a) isoform size and plasma Lp(a) concentrations. About 90% of plasma Lp(a) concentration is genetically determined and plasma levels can modestly rise after menopause in women, and in conditions like hypothyroidism, nephrotic syndrome. It has been shown that elevated Lp(a) is an independent risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve stenosis. Although Lp(a) concentration does vary with ethnicity, relationships between Lp(a) concentration and ASCVD risk remain similar across different ethnic groups. Elevated Lp(a) is considered a cardiovascular risk-enhancing or amplification factor, and recent guidelines and consensus have increasingly recommended universal screening for Lp(a). There are as yet, no approved therapies for elevated Lp(a). Current management focuses on intensifying control of concurrent risk factors, particularly LDL-C, to reduce ASCVD risk. Amongst existing lipid-lowering drugs, only proprotein convertase subtilisin/kexin type 9 inhibitors can lower Lp(a) levels modestly. Emerging RNA-based and small-molecule therapeutics are under development and are showing promising Lp(a)-lowering effects up to 80-90%. Ongoing phase 3 cardiovascular outcomes trials will determine whether effectively lowering Lp(a) can translate to cardiovascular benefit.
A Novel Therapeutic Concept for Familial LCAT Deficiency: Long-Term Enzyme Replacement Using Genetically Modified Adipocytes

Masayuki KurodaJapan Speaker A Novel Therapeutic Concept for Familial LCAT Deficiency: Long-Term Enzyme Replacement Using Genetically Modified AdipocytesFamilial lecithin:cholesterol acyltransferase (LCAT) deficiency is a rare autosomal recessive disorder marked by defective HDL maturation, leading to corneal opacity, hemolytic anemia, and progressive renal dysfunction. No disease-modifying treatment has been established to date. Conventional enzyme replacement requires repeated administration with limited durability. Glybera, the first AAV1-based gene therapy for lipoprotein lipase deficiency, was withdrawn after limited clinical use and modest benefit. More broadly, in vivo AAV gene therapies face challenges including immune responses, hepatotoxicity at high vector doses, and considerable inter-patient variability in transgene expression. Our therapeutic approach originated from studies in diabetic mouse models, where adipocytes were explored as platforms for sustained protein delivery. These cells demonstrated endocrine-like properties and long-term protein secretion in vivo. Adipocytes are particularly suited for this purpose due to their longevity, secretory capacity, and low tumorigenic risk. Building on these findings, we established an ex vivo gene and cell therapy platform using genetically modified adipocytes (GMAC), autologous adipocyte-derived cells engineered to express therapeutic proteins. As its first application, we targeted familial LCAT deficiency. These cells were expected to engraft upon subcutaneous implantation, re-differentiate into functional adipocytes, and provide long-lasting and therapeutically relevant LCAT secretion. In a first-in-human clinical trial conducted under Japan’s regulatory framework for regenerative medicine, mature adipocytes were collected from the patient’s subcutaneous fat, converted into proliferative cells via ceiling culture, and transduced to express therapeutic human LCAT, then administered subcutaneously to the patient. Single administration of LCAT-GMAC was well tolerated with no serious adverse events. Sustained increases in serum LCAT activity were observed, accompanied by improvements in lipoprotein profiles and hemolytic anemia. A marked reduction in proteinuria was noted, and renal function remained stable throughout the follow-up period. Remarkably, serum LCAT activity persisted for over eight years after the single administration, the longest durability ever reported for enzyme replacement. This provides the first clinical evidence that ceiling culture-derived, ex vivo modified adipocytes can achieve lasting correction of systemic enzyme deficiencies. LCAT-GMAC therapy thus offers a potentially curative strategy for familial LCAT deficiency and a new paradigm for treating dyslipidemias and other lifelong plasma protein deficiencies.
Novel and Future Lipid-Lowering Therapy

Sung Hee ChoiSouth Korea Speaker Novel and Future Lipid-Lowering TherapyIn this lecture, I want to introduce the mechanism of current developing lipid lowering drugs. Small molecular inhibitors such as bempedoic acid, oral forms of newer drugs, Anti-sense oligonucleotide drugs, and siRNA technique based new lipid lowering drugs and its clinical trials. These agents target diverse pathways such as proprotein convertase subtilisin/kexin type 9 (PCSK9), angiopoietin-like protein 3 (ANGPTL3), apolipoprotein C-III (apoC-III), and Lp(a), achieving potent lipid modulation in different mechanistic approach.

101

Sung Hee ChoiProf. South Korea

21 MARCH