Dr. Zhang Lei is a distinguished physician-scientist and Professor of Hematology at the Institute of Hematology & Blood Diseases Hospital, CAMS. His research focuses on the pathogenesis, precise diagnosis, and targeted therapy of hemorrhagic and thrombotic diseases, including hemophilia and immune thrombocytopenia (ITP). He has made a series of groundbreaking contributions to the field.
Dr. Zhang is a pioneer of gene therapy in China. He led the team that conducted Asia's first successful clinical trial of AAV-mediated gene therapy for Hemophilia B, achieving long-term stable factor IX expression and enabling the first major surgery (total knee arthroplasty) without factor infusion in a gene therapy patient worldwide. This work culminated in him spearheading the development and approval of Asia's first gene therapy product for a genetic disease in 2024. Concurrently, his team has developed novel protein therapeutics, such as a world-first Factor X activator inspired by snake venom.
In the realm of immunotherapy, Dr. Zhang's team identified CD38 as a novel therapeutic target for ITP. They conducted the first-in-human clinical trials of a novel anti-CD38 monoclonal antibody (CM313) for adult ITP and of daratumumab for pediatric ITP, demonstrating rapid, durable platelet responses. This work, published in the New England Journal of Medicine, represents a paradigm shift in ITP treatment.
His foundational research has significantly advanced the understanding of myeloproliferative neoplasms (MPN), elucidating mechanisms of JAK2V617F-driven hematopoietic stem cell heterogeneity, megakaryocytic bias, and bone marrow microenvironment dysfunction. Dr. Zhang has authored over 80 SCI-indexed papers as a corresponding author in top-tier journals including N Engl J Med, BMJ, Nat Med, Lancet Haematol, Cell Stem Cell, and Blood, with numerous publications selected for China's Top 10 Advances in Hematology. He holds 8 authorized national patents. His work exemplifies a powerful "bench-to-bedside" translational research model, establishing him as a national leader in hematology.
M.D., Clinical Medicine (Hematology), Peking Union Medical College, Beijing, China, 2000-2004
Deputy Director, National Clinical Research Center for Blood Diseases, Tianjin, China, 2022-
Deputy Director, Institute of Hematology & Blood Diseases Hospital, CAMS, Tianjin, China, 2020-
Deputy Director, State Key Laboratory of Experimental Hematology, Tianjin, China, 2018-
Director, CAMS/Tianjin Key Laboratory of Gene Therapy, Tianjin, China, 2018-
Chief Physician, Institute of Hematology & Blood Diseases Hospital, CAMS, Tianjin, China, 2013-
Visiting Scholar, Hematology, University of Pittsburgh Medical Center, Pittsburgh, PA, USA, 2010-2011
PRINCIPAL HONORS, AWARDS:
2025 Special Government Allowance, State Council of China
2024 Tianjin Outstanding Talent
2023 The National Ten Thousand Talents Program
2023 National Health Commission Outstanding Contribution Young and Middle-Aged Expert
2023 First Prize, Tianjin Science and Technology Progress Award (Rank 1)
2022 Special Prize, Tianjin Natural Science Award (Rank 7)
2021 Third Prize, Huaxia Medical Science and Technology Award (Rank 1)
2009 Second Prize, National Science and Technology Progress Award (Rank 3)
MAJOR NATIONAL LEADERSHIP POSITIONS, SOCIETIES AND BOARDS:
2024-Present Member, 12th Hematology Society, Chinese Medical Association
2023-Present Standing Member, 8th Tianjin Hematology Society
2022-Present Council Member, 8th Tianjin Medical Association
2021-Present Chairman & Legal Representative, Tianjin Society of Blood and Regenerative Medicine
2021-Present Editor-in-Chief, Global Medical Genetics
2020-Present Member, 10th Editorial Board, Chinese Journal of Hematology
2022-Present Secretary-General, 1st Committee of Hematology Institutions, Chinese Hospital Association
2020-Present Standing Committee Member & Secretary-General, Hemophilia Group, China Rare Diseases Alliance
SELECTED PUBLICATIONS:
1. Xue F, Ju M, Zhu T, Zhou Z, Sun J, Yang L, Yan Z, Zhou H, Du X, Zheng C, Zheng J, Wu X, Du Z, Jiang W, Yang C, Xiao X, Liu W, Yang R, Zhang L*. Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials. Nat Med. 2025 Nov 20. Online.
2. Chen Y, Xu Y, Dai J, Sun T, Li H, Hua Z, Zhou Z, Zhou H, Yan Z, Zhao X, Xue F, Liu W, Liu X, Fu R, Wang W, Chi Y, Dong H, Ju M, Dai X, Gu W, Pei X, Yang R, Zhang L*. Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial. BMJ. 2025 Oct 21;391:e084314.
3. 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.
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 Apr 23;9(1):102.
6. 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.
7. 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.
8. 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. doi: 10.1016/j.stem.2021.01.018. Epub 2021 Feb 22. Erratum in: Cell Stem Cell. 2021 Apr 1;28(4):780.
9. 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.
10. 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.