희귀유전질환심포지엄조직위원회
Rare Genetic Disease Symposium Organizing Committee
PROFILE
김형섭 연구팀장
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Towards a Disease-Modifying Therapy for Charcot–Marie–Tooth Disease: Preclinical and Clinical Development of EN001
EN001 is a high-potency, umbilical cord tissue-derived mesenchymal stromal cell therapy being developed for Charcot–Marie–Tooth disease (CMT), with multimodal regenerative effects on Schwann cells, peripheral nerves, and skeletal muscle. A Phase 1a/b study and an investigator-initiated trial in a total of 18 patients with CMT demonstrated favorable safety and tolerability with exploratory signals of efficacy, and a Phase 2a study is currently ongoing in Korea. As a leading clinical-stage cell therapy pipeline for CMT, this presentation will highlight the key preclinical and clinical findings supporting the therapeutic potential of EN001.
ENCell 신약 R&BD본부
EDUCATION
2015-2020, M.S./Ph.D. in Health Sciences and Technology, Sungkyunkwan University, Seoul, Korea
2011-2015, B.S. in Biological Sciences and Psychology, Sungkyunkwan University, Suwon, Korea
PROFESSIONAL EXPERIENCE
2020-Present, Team Leader, New Drug R&BD Division / Translational Science, ENCell Co., Ltd., Seoul, Korea
2015-2020, Researcher, Department of Neurology, Samsung Medical Center, Seoul, Korea
RESEARCH ACTIVITIES & OTHERS
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Led the preclinical and translational development of ENCell’s EN001 pipeline across CMT, DMD, and sarcopenia, including mechanism-of-action, efficacy, IND-enabling, and translational studies. Led to IND- enabling studies and supported regulatory submissions leading to IND approval and clinical advancement of MSC-based therapeutic programs.
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Published a study in Nano Letters (2023) on iron oxide nanoparticle-incorporated mesenchymal stromal cells for Alzheimer’s disease treatment, highlighting an advanced approach to enhance MSC-based therapeutic delivery and tracking.
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Published translational research in Neurobiology of Disease (2024) demonstrating the therapeutic potential of Wharton’s jelly-derived MSCs in CMT1A, including enhancement of Schwann cell proliferation.
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Contributed to the clinical translation of MSC therapy for Duchenne muscular dystrophy, with Phase 1 safety and tolerability results published in the Journal of Clinical Neurology (2025).
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Published mechanistic and translational research on Wharton’s jelly-derived MSCs across multiple disease areas, including preterm premature rupture of membranes (2026) and neuromuscular disorders.
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Engaged in business development and external collaboration activities with global pharmaceutical and biotechnology companies, as well as CMT patient advocacy groups and nonprofit organizations, leveraging expertise in cell therapy and translational science.