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Revolutionizing genetic disease treatment: The case of exagamglogene autotemcel

  • 1.

    State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China

  • 2.

    Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing 100044, China

  • 3.

    Research Unit of Key Technique for Diagnosis and Treatments of Hematologic Malignancies, Chinese Academy of Medical Sciences (2019RU029), Beijing 100044, China

  • 4.

    Department of Hematology & Cancer Biology Research Center, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China

  • 5.

    Department of Hematology & Hematopoietic Stem Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA

More Information

  • 6These authors contributed equally

  • Corresponding authors: raoshuquan@ihcams.ac.cn (S.R);  ebudde@coh.org (L.E.B.)
  • Received Date:  29 January 2024
    Accepted Date:  27 March 2024
    Published Online:  30 March 2024
The Innovation  5(3),  https://doi.org/10.1016/j.xinn.2024.100619  |  Cite this article
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    • REFERENCES
  • [1] Kato, G.J., Piel, F.B., Reid, C.D., et al. (2018). Sickle cell disease. Nat. Rev. Dis. Primers 4: 18010. https://doi.org/10.1038/nrdp.2018.10.

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    [2] Origa, R. (2017). β-Thalassemia. Genet. Med. 19: 609-619. https://doi.org/10.1038/gim.2016.173.

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    [3] Frangoul, H., Altshuler, D., Cappellini, M.D., et al. (2021). CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia. N. Engl. J. Med. 384: 252-260. https://doi.org/10.1056/NEJMoa2031054.

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    [4] (2023). FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. (FDA News release). https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease.

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    [5] (2024). CASGEVY (exagamglogene autotemcel) suspension for intravenous infusion. https://pi.vrtx.com/files/uspi_exagamglogene_autotemcel.pdf.

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    • CITED BY

  • Cite this article:

    Rao S., Yao Y., Huo W., et al., (2024). Revolutionizing genetic disease treatment: The case of exagamglogene autotemcel. The Innovation 5(3), 100619. https://doi.org/10.1016/j.xinn.2024.100619
    Rao S., Yao Y., Huo W., et al., (2024). Revolutionizing genetic disease treatment: The case of exagamglogene autotemcel. The Innovation 5(3), 100619. https://doi.org/10.1016/j.xinn.2024.100619

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