Prof. Lei Zhang is a tenured Associate Professor and doctoral supervisor at Peking Union Medical College, currently serving as Vice Secretary of the Party Committee and Secretary of the Discipline Inspection Commission at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (CAMS). He also holds concurrent leadership roles as Deputy Director of the National Clinical Research Center for Blood Diseases, the State Key Laboratory of Hematology and Health, and the Center for Thrombosis and Hemostasis, as well as Director of the Key Laboratory for Gene Therapy of Hematologic Diseases (CAMS and Tianjin).
Prof. Zhang has long been engaged in both basic and clinical research on hematologic diseases. He has led more than 20 national and provincial research projects, including serving as Principal Investigator of the National Key R&D Program and the National Natural Science Foundation of China (NSFC) Key Program. As a corresponding author, he has published over 200 SCI-indexed papers in leading international journals such as The New England Journal of Medicine (3 articles), The Lancet Haematology (2 articles), Cell Stem Cell, Nature Communications, Signal Transduction and Targeted Therapy, and Blood. He holds 7 authorized national invention patents. He received the First Prize of the Tianjin Science and Technology Progress Award and the Third Prize of the Huaxia Medical Science and Technology Award as the first contributor; he also received the Second Prize of the National Science and Technology Progress Award and seven other provincial and ministerial-level awards.
In recent years, Prof. Zhang has pioneered new pathogenic mechanisms and treatment strategies in coagulation and bleeding disorders. His original clinical research includes the world's first application of mesenchymal stem cells and thrombopoietin receptor agonists for refractory immune thrombocytopenia, and he led the first international multicenter randomized controlled trial of rituximab for acquired hemophilia. He developed a novel anti-CD38 monoclonal antibody that has achieved long-term remission in immune thrombocytopenia. As the principal investigator of the first gene therapy clinical trial for hemophilia in Asia, he reported the world's first case of a gene therapy recipient undergoing major surgery with normal hemostasis. He has led 8 gene therapy clinical studies to date, curing over 100 severe hemophilia patients and saving tens of millions of RMB in healthcare costs. His team's efforts contributed to the completion of China's first Phase III clinical trial of a gene therapy product for hemophilia.
Prof. Zhang has also made significant advances in the mechanisms of platelet diseases, including megakaryocytic differentiation from hematopoietic stem cells, unique molecular mutations, and the critical role of acylglycerol kinase in platelet function. His work has had a major international impact and has been recognized among China's "Top Ten Medical Science News" and "Top Ten Advances in Hematology."
He currently serves as President of the Tianjin Society of Hematology and Regenerative Medicine, Member of the Hematology Branch of the Chinese Medical Association, Standing Member of the Hematology Branch of the Tianjin Medical Association, and Editor-in-Chief of Global Medical Genetics. He has received numerous honors, including Leading Talent in Scientific and Technological Innovation, Outstanding Young and Middle-aged Expert by the National Health Commission, State Council Special Government Allowance, Tianjin Medical Talent, and Distinguished Professor of Peking Union Medical College. He also leads teams that have been recognized as Tianjin Innovation Team and PUMC Innovation Team.
Ph.D., Internal Medicine, Peking Union Medical College, Beijing, China, 2000–2004
B.M., Clinical Medicine, Weifang Medical University, Weifang, China, 1993–1998
Vice Secretary of the Party Committee & Secretary of the Discipline Inspection Commission, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2023 – Present
Vice President, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2020 – 2023
Deputy Director, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2020 – Present
Deputy Director, State Key Laboratory of Hematology and Health, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2018 – Present
Deputy Director, Center for Thrombosis and Hemostasis, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2016 – Present
Director, Key Laboratory for Gene Therapy of Hematologic Diseases (CAMS and Tianjin), Tianjin, 2018 – Present
Visiting Scholar, University of Pittsburgh, Pittsburgh, PA, USA, 2010 – 2011
Chief Physician, Center for Thrombosis and Hemostasis, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2013 – Present
Associate Chief Physician, Center for Thrombosis and Hemostasis, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2007 – 2013
Attending Physician, Center for Thrombosis and Hemostasis, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2004 – 2007
Resident Physician, Affiliated Hospital of Weifang Medical University, 1998 – 2000
PRINCIPAL HONORS, AWARDS:
2024 Excellent Teacher Award, Peking Union Medical College
2024 State Council Special Government Allowance Recipient
2024 Outstanding Talent of Tianjin
2023 Leading Talent in Scientific and Technological Innovation
2023 Outstanding Young and Middle-aged Expert (10th Selection), National Health Commission
2023 First Prize, Tianjin Science and Technology Progress Award, for original gene therapy technologies and novel hemophilia treatment systems (First Contributor)
2022 Special Prize, Tianjin Natural Science Award, for blood cell molecular mapping and ecosystem research (Seventh Contributor)
2011 First Prize, Tianjin Science and Technology Progress Award, for pathogenesis and treatment strategies of immune thrombocytopenia (Fifth Contributor)
2011 Second Prize, Ministry of Education Science and Technology Progress Award, for research on immune thrombocytopenia (Fifth Contributor)
2009 Second Prize, National Science and Technology Progress Award, for hematopoietic stem cell technology and its application (Third Contributor)
MAJOR NATIONAL LEADERSHIP POSITIONS, SOCIETIES AND BOARDS:
2024–2027 Member, 12th Committee of the Hematology Branch, Thrombosis and Hemostasis Group, Chinese Medical Association
2022–2027 Council Member, 8th Council, Tianjin Medical Association
2023–2026 Standing Member, 8th Committee of Hematology Branch, Tianjin Medical Association
2021–2026 President, 1st Council, Tianjin Society of Hematology and Regenerative Medicine
2020–2025 Standing Member & Secretary-General, Hemophilia Group, China Alliance for Rare Diseases
2021–Present Editor-in-Chief, Global Medical Genetics
2022–Present Executive Editorial Board Member, Journal of Thrombosis and Hemostasis (8th Editorial Board)
2024–Present Editorial Board Member, Blood Science (English) (1st Editorial Board)
2025–Present Deputy Editor-in-Chief, Chinese Journal of Hematology (11th Editorial Board)
SELECTED PUBLICATIONS:
1. Sun T#, Chen Y#, Zhang L*. Daratumumab in Relapsed or Refractory Pediatric Immune Thrombocytopenia. N Engl J Med. 2025 May 29;392(20):2069-2071.
2. Fu R#, Dong H#, Gu W#, Meng K#, Sun T, Liu X, Qu X, Chen J, Xue F, Liu W, Chen Y, Ju M, Dai X, Chi Y, Wang W, Pei X, Zhu X*, Yang R*, Li H*, Zhang L*. Childhood and adolescent essential thrombocythemia and prefibrotic primary myelofibrosis: insights into diagnosis, outcomes, and treatment from a large Chinese cohort. Leukemia. 2025 Jan;39(1):155-165.
3. Dong H#, Chen J#, Zhang J#, Xue F, Li H, Zhang D, Zhou H, Zhang X, Huang Y, Liu X, Chen Y, Liu W, Chi Y, Wang W, Sun T, Ju M, Dai X, Gu W, Yang R, Fu R*, Zhang L*. Reduced platelet activation in triple-negative essential thrombocythemia compared with JAK2V617F-mutated essential thrombocythemia. Clin Cancer Res. 2024 Dec 2;30(23):5473-5482.
4. Chen Y#, Xu Y#, Li H#, Sun T#, Cao X#, Wang Y, Xue F, Liu W, Liu X, Dong H, Fu R, Dai X, Wang W, Ma Y, Song Z, Chi Y, Ju M, Gu W, Pei X, Yang R, Zhang L*. A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia. N Engl J Med. 2024 Jun 20;390(23):2178-2190.
5. Chen Y#, Xu Y#, Chi Y#, Sun T#, Gao Y#, Dou X, Han Z, Xue F, Li H, Liu W, Liu X, Dong H, Fu R, Ju M, Dai X, Wang W, Ma Y, Song Z, Gu J, Gong W, Yang R, Zhang L*. Efficacy and safety of human umbilical cord-derived mesenchymal stem cells in the treatment of refractory immune thrombocytopenia: a prospective, single arm, phase I trial. Signal Transduct Target Ther. 2024 April 23;9(1):102.
6. Zhang P#, You N#, Ding Y, Zhu W, Wang N, Xie Y, Huang W, Ren Q, Qin T, Fu R, Zhang L*, Xiao Z*, Cheng T*, Ma X*. Gadd45g insufficiency drives the pathogenesis of myeloproliferative neoplasms. Nat Commun. 2024 April 06;15(1):2989.
7. Li Y#, Chen J#, Sun T#, Chen Y, Fu R, Liu X, Xue F, Liu W, Ju M, Dai X, Dong H, Li H, Wang W, Chi Y, Zhang L*. Genetically determined telomere length and risk for haematologic diseases: results from large prospective cohorts and Mendelian Randomization analysis. Blood Cancer J. 2024 Mar 18;14(1):48.
8. Chen Y#, Xu Y#, Huang Y#, Sun T#, Hu Y#, Xue F, Peng X, Lai Y, Zhou Z, Zhou H, Zhou R, Liu W, Liu X, Dong H, Fu R, Ju M, Dai X, Li H, Liu X, Ma Y, Song Z, Peng J*, Yang R*, Zhang L*. Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase II trial. Am J Hematol. 2024 Jan;99(1):E22-E25.
9. Wang P#, Zhou R#, Xue F#, Zhou H, Bai J, Wang X, Ma Y, Song Z, Chen Y, Liu X, Fu R, Sun T, Ju M, Dai X, Dong H, Yang R, Liu W*, Zhang L*. Single-dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: A multicenter, open-label, randomized noninferiority trial. Am J Hematol. 2024 Jan;99(1):28-37.
10. An ZY#, Wu YJ#, Hou Y#, Mei H#, Nong WX#, Li WQ#, Zhou H, Feng R, Shen JP, Peng J, Zhou H, Liu Y, Song YP, Yang LH, Fang MY, Li JY, Cheng YF, Liu P, Xu YJ, Wang Z, Luo Y, Cai Z, Liu H, Wang JW, Li J, Zhang X, Sun ZM, Zhu XY, Wang X, Fu R, Huang L, Wang SY, Yang TH, Su LP, Ma LM, Chen XQ, Liu DH, Yao HX, Feng J, Zhang HY, Jiang M, Zhou ZP, Wang WS, Shen XL, Baima Y, Li YY, Wang QF, Huang QS, Fu HX, Zhu XL, He Y, Jiang Q, Jiang H, Lu J, Zhao XY, Chang YJ, Wu T, Pan YZ, Qiu L, Gao D, Jin AR, Li W, Gao SJ, Zhang L*, Hou M*, Huang XJ*, Zhang XH*; National Cooperative ITP Working Group. A life-threatening bleeding prediction model for immune thrombocytopenia based on personalized machine learning: a nationwide prospective cohort study. Sci Bull (Beijing). 2023 Sep 30;68(18):2106-2114.
11. Gao Y#, Chi Y#, Chen Y#, Wang W#, Li H, Zheng W, Zhu P, An J, Duan Y, Sun T, Liu X, Xue F, Liu W, Fu R, Han Z, Zhang Y, Yang R, Cheng T, Wei J*, Zhang L*, Zhang X*. Multi-omics analysis of human mesenchymal stem cells shows cell aging that alters immunomodulatory activity through the downregulation of PD-L1. Nat Commun. 2023 Jul 20;14(1):4373.
12. Jin J#*, Zhang L#*, Qin A*, Wu D, Shao Z, Bai J, Chen S, Duan M, Zhou H, Xu N, Zhang S, Zuo X, Du X, Wang L, Li P, Zhang X, Li Y, Zhang J, Wang W, Shen W, Zagrijtschuk O, Urbanski R, Sato T, Xiao Z. A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. Exp Hematol Oncol. 2023 Jun 21;12(1):55.
13. Zhang L#*. Gene therapy in China: past, present, and future. Lancet Haematol. 2022 Dec;9(12):e869-e871.
14. Xue F#, Wang P#, Yuan Z#, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L*. Total Knee Arthroplasty after Gene Therapy for Hemophilia B. N Engl J Med. 2022 Oct 27;387(17):1622-1624.
15. Xue F#, Li H#, Wu X#, Liu W#, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R*, Xiao X*, Zhang L*. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022 Jul;9(7):e504-e513.
16. Tong J#, Sun T#, Ma S#, Zhao Y#, Ju M#, Gao Y, Zhu P, Tan P, Fu R, Zhang A, Wang D, Wang D, Xiao Z, Zhou J, Yang R, Loughran SJ, Li J, Green AR, Bresnick EH, Wang D, Cheng T*, Zhang L*, Shi L*. Hematopoietic Stem Cell Heterogeneity Is Linked to the Initiation and Therapeutic Response of Myeloproliferative Neoplasms. Cell Stem Cell. 2021 Mar 4;28(3):502-513.e6.
17. Fu YW, Dai XY, Wang WT, Yang ZX, Zhao JJ, Zhang JP, Wen W, Zhang F, Oberg KC, Zhang L*, Cheng T*, Zhang XB*. Dynamics and competition of CRISPR-Cas9 ribonucleoproteins and AAV donor-mediated NHEJ, MMEJ and HDR editing. Nucleic Acids Res. 2021 Jan 25;49(2):969-985.
18. Jiang H, Yu Z, Ding N, Yang M, Zhang L, Fan X, Zhou Y, Zou Q, Hou J, Zheng J, Zhang L*, Xu Y*, Liu J*. The role of AGK in thrombocytopoiesis and possible therapeutic strategies. Blood. 2020 Jul 2;136(1):119-129.