Skip to content
ContentLora

    Tip: press / anywhere to search.

    product

    Casgevy (exa-cel)

    Also known as exagamglogene autotemcel, exa-cel, CTX001

    Casgevy (exagamglogene autotemcel) is a one-time therapy that uses CRISPR-Cas9 to edit a patient's own blood stem cells so they make more fetal hemoglobin.[1] It became the first approved CRISPR-based medicine in 2023, and in July 2026 the FDA extended it to children aged 2 and older.[2][3][4]

    Editor reviewedStrict sourcingUpdated Gene editing and gene therapyHealth and medicineLife sciences
    Key facts

    Casgevy is the first medicine built on crispr-cas9 gene editing to reach patients. It treats sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta-thalassemia (TDT).[3][5] This page describes evidence and regulatory status. It is not medical advice.

    How it works

    Casgevy does not fix the sickle cell mutation itself. It edits an enhancer of the BCL11A gene in blood stem cells. BCL11A normally represses fetal hemoglobin, so disrupting it lets red cells make more fetal hemoglobin.[1] Treatment involves collecting the patient’s stem cells and editing them outside the body. The patient then receives myeloablative conditioning (high-dose chemotherapy) to clear the bone marrow, and the edited cells are infused back once.[6] CRISPR Therapeutics describes it as a non-viral, ex vivo edited cell therapy.[7]

    Approvals

    • UK, 16 November 2023. The MHRA authorised Casgevy for patients aged 12 and over with sickle cell disease or TDT.[2]
    • US, 8 December 2023. The FDA approved it for sickle cell disease from age 12, calling it the first FDA-approved therapy using CRISPR/Cas9. On the same day it approved Lyfgenia, a lentiviral gene therapy.[3][8]
    • US, 16 January 2024. The FDA approved the TDT indication.[5]
    • US, 1 July 2026. The FDA approved Casgevy from age 2, 53 days after filing, under the Commissioner’s National Priority Voucher pilot.[4] Data came from children aged 5 to 12, and use in ages 2 to 5 relied on extrapolation.[9]

    Evidence and safety

    In the data supporting the 2023 US approval, 29 of 31 evaluable sickle cell patients (93.5%) were free of severe vaso-occlusive crises for at least 12 consecutive months.[10] In the pediatric trials, all 8 evaluable children with sickle cell disease met the primary endpoint, and 8 of 9 with TDT became transfusion-independent.[9] The label warns of neutrophil engraftment failure, delayed platelet engraftment, hypersensitivity reactions and off-target genome editing risk.[11]

    Commercial status (2026)

    Vertex leads manufacturing and commercialization and splits profits and costs 60/40 with crispr-therapeutics.[12] Second-quarter 2026 revenue was $76 million, up 78% on the prior quarter.[13] Casgevy is approved in 39 countries,[14] and Germany agreed reimbursement in May 2026.[15] Vertex estimates the pediatric approval makes about 5,500 more patients eligible.[16] In the US, a Medicaid access model run by CMS negotiates outcomes-based agreements for sickle cell gene therapies.[17] Vertex’s 2026 guidance assumes $500 million or more in revenue from Casgevy and its pain drug Journavx combined.[18]

    Price and access

    Vertex set Casgevy’s US list price at $2.2 million per patient at approval in December 2023. Lyfgenia, approved the same day, was priced at $3.1 million.[19] CMS notes that cell and gene therapies can cost millions of dollars, and designed its access model to tie payment to outcomes.[20] About 100,000 people in the US have sickle cell disease.[21] WHO estimates that sub-Saharan Africa accounts for nearly 80% of cases worldwide.[22] The access debate page sets out the arguments.

    What comes next

    By August 2026 the companies had filed in Saudi Arabia and the UK to extend Casgevy to children aged 5 to 11.[23] Conditioning chemotherapy is one of the main burdens of treatment, and Vertex says preclinical work on gentler regimens could broaden eligibility.[24] CRISPR Therapeutics is exploring editing blood stem cells inside the body, which would avoid cell collection.[25] Beam plans to file risto-cel, a base-edited sickle cell therapy, as early as year-end 2026.[26]

    Questions readers ask

    How does Casgevy work?

    A patient's own blood stem cells are collected and edited with CRISPR-Cas9 at an enhancer of the BCL11A gene, which represses fetal hemoglobin. After high-dose chemotherapy conditioning, the edited cells are infused back as a one-time treatment.[1][6]

    How well did Casgevy work in the trials behind its approval?

    In the data behind the 2023 FDA approval, 29 of 31 evaluable sickle cell patients (93.5%) had no severe vaso-occlusive crises for at least 12 consecutive months.[10]

    What safety warnings does Casgevy carry?

    Its prescribing information includes warnings for neutrophil engraftment failure, delayed platelet engraftment, hypersensitivity reactions and off-target genome editing risk.[11]

    Who makes Casgevy?

    Vertex Pharmaceuticals leads development, manufacturing and commercialization, and shares profits and costs 60/40 with CRISPR Therapeutics.[12]

    Sources

    Each numbered claim is a statement we checked against the sources listed with it. Status shows how well established it is.

    1. [1]

      Casgevy's approach edits a patient's blood stem cells outside the body by electroporating them with CRISPR-Cas9 aimed at an enhancer of the BCL11A gene, a gene that represses fetal hemoglobin, so that the cells make more fetal hemoglobin. confirmedas of 2026-10-10

    2. [2]

      On 16 November 2023 the UK medicines regulator, the MHRA, authorised Casgevy for patients aged 12 and over with sickle cell disease or transfusion-dependent beta-thalassemia, the first authorisation of a CRISPR-based medicine. confirmedas of 2026-10-10

    3. [3]

      On 8 December 2023 the FDA approved Casgevy, the first FDA-approved therapy using CRISPR/Cas9, for people aged 12 and older with sickle cell disease and recurrent vaso-occlusive crises. confirmedas of 2026-10-10

    4. [4]

      On 1 July 2026 the FDA expanded Casgevy's approval to patients aged 2 years and older with sickle cell disease or transfusion-dependent beta-thalassemia, 53 days after filing, under the Commissioner's National Priority Voucher pilot program. confirmedas of 2026-10-10

    5. [5]

      The FDA approved Casgevy for transfusion-dependent beta-thalassemia on 16 January 2024. confirmedas of 2026-10-10

    6. [6]

      Treatment with Casgevy involves collecting the patient's own stem cells, editing them, giving myeloablative conditioning (high-dose chemotherapy) to clear the bone marrow, and infusing the edited cells back as a one-time treatment. confirmedas of 2026-10-10

    7. [7]

      CRISPR Therapeutics describes Casgevy as a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy. confirmedas of 2026-10-10

    8. [8]

      On the same day as Casgevy, the FDA approved Lyfgenia, a sickle cell gene therapy that uses a lentiviral vector rather than gene editing; 28 of 32 patients (88%) had complete resolution of vaso-occlusive events in its trial. confirmedas of 2026-10-10

    9. [9]

      The pediatric approval rested on trials in which all 8 evaluable children with sickle cell disease (ages 5 to 12) had no severe crises for 12 months and 8 of 9 with beta-thalassemia became transfusion-independent; use in children aged 2 to 5 relied on extrapolation. confirmedas of 2026-10-10

    10. [10]

      In the data supporting the 2023 FDA approval, 29 of 31 evaluable sickle cell patients (93.5%) were free of severe vaso-occlusive crises for at least 12 consecutive months. confirmedas of 2026-10-10

    11. [11]

      Casgevy's prescribing information carries warnings for neutrophil engraftment failure, delayed platelet engraftment, hypersensitivity reactions and off-target genome editing risk. confirmedas of 2026-10-10

    12. [12]

      Vertex leads global development, manufacturing and commercialization of Casgevy, and Vertex and CRISPR Therapeutics share its profits and program costs 60/40. confirmedas of 2026-10-10

    13. [13]

      Casgevy generated $76 million in revenue in the second quarter of 2026, up 78% from the prior quarter. confirmedas of 2026-10-10

    14. [14]

      As of August 2026, Casgevy was approved in 39 countries across North America, Europe and the Middle East. confirmedas of 2026-08-03

    15. [15]

      In May 2026 Vertex secured reimbursement for Casgevy in Germany for eligible patients aged 12 and older. confirmedas of 2026-10-10

    16. [16]

      Vertex estimates that about 5,500 more patients with sickle cell disease or beta-thalassemia became eligible for Casgevy with the 2026 pediatric approval. confirmedas of 2026-10-10

    17. [17]

      The US Cell and Gene Therapy Access Model is a voluntary CMS program in which CMS negotiates outcomes-based agreements with manufacturers, initially for sickle cell gene therapies from Vertex and Genetix Biotherapeutics; 34 state Medicaid programs plus Washington DC and Puerto Rico joined between January 2025 and January 2026. confirmedas of 2026-10-10

    18. [18]

      Vertex's 2026 revenue guidance assumes $500 million or more from non-cystic-fibrosis products, namely Casgevy and Journavx, within total revenue guidance of $13.1 billion to $13.2 billion. confirmedas of 2026-08-03

    19. [19]

      Vertex set Casgevy's US list price at $2.2 million per patient when it was approved in December 2023; Lyfgenia's wholesale acquisition cost was set at $3.1 million. confirmedas of 2026-10-10

    20. [20]

      CMS notes that cell and gene therapies can cost millions of dollars, and designed its access model to lower prices for states and tie payment to outcomes. confirmedas of 2026-10-10

    21. [21]

      Sickle cell disease affects approximately 100,000 people in the United States. confirmedas of 2026-10-10

    22. [22]

      WHO estimates that 7.74 million people were living with sickle cell disease in 2021, with 515,000 new births with the disease, and that sub-Saharan Africa accounts for nearly 80% of global cases. confirmedas of 2026-10-10

    23. [23]

      As of August 2026 Vertex and CRISPR Therapeutics had completed regulatory submissions in Saudi Arabia and the United Kingdom to extend Casgevy to children aged 5 to 11. confirmedas of 2026-08-03

    24. [24]

      Vertex says it is advancing preclinical work on gentler conditioning regimens for Casgevy, which it says could broaden the eligible patient population. confirmedas of 2026-08-03

    25. [25]

      CRISPR Therapeutics says it is advancing in vivo editing of blood-forming stem cells using lipid nanoparticle delivery, an approach it says could expand the treatable population for sickle cell disease and beta-thalassemia beyond ex vivo therapy. confirmedas of 2026-08-03

    26. [26]

      Beam plans to submit a Biologics License Application for risto-cel (formerly BEAM-101), a base-edited cell therapy for sickle cell disease, as early as year-end 2026. reportedas of 2026-08-04· forecast

    Revision history (2)
    1. Page created.
    2. Added list prices, Vertex guidance, disease burden, pending pediatric filings and next-generation approaches.

    Created Oct 10, 2026. Last reviewed by an editor on Oct 10, 2026. Next scheduled review: Jan 10, 2027.

    Cite this page

    "Casgevy (exa-cel)." ContentLora, updated Oct 10, 2026. https://contentlora.com/wiki/casgevy

    Spotted an error? Suggest a correction or emailcorrections@contentlora.com.

    Keep exploring

    Connections
    CRISPR-Cas9 Based onCRISPR Therapeutics Co-developed by
    ☰ Contents