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    CRISPR Therapeutics

    Also known as CRISPR Therapeutics AG, CRSP

    CRISPR Therapeutics is a gene-editing company headquartered in Zug, Switzerland, that co-developed Casgevy, the first approved CRISPR medicine, with Vertex Pharmaceuticals.[1][2] Its in vivo program CTX310 lowered blood lipids in a Phase 1 trial published in 2025.[3]

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

    CRISPR Therapeutics AG develops medicines based on crispr-cas9 gene editing. It is headquartered in Zug, Switzerland, and has a US subsidiary and research operations in Boston and San Francisco.[1] This page describes company disclosures and published data. It is not investment or medical advice.

    Casgevy

    The company’s approved product is casgevy, an ex vivo, non-viral CRISPR/Cas9-edited cell therapy for sickle cell disease and transfusion-dependent beta-thalassemia.[4] Vertex leads Casgevy’s development, manufacturing and commercialization. The two companies share profits and program costs 60/40.[2] The FDA approved Casgevy in December 2023 as the first therapy using CRISPR/Cas9.[5] It extended the approval to children aged 2 and older on 1 July 2026.[6] Casgevy revenue was $76 million in the second quarter of 2026, up 78% on the prior quarter.[7]

    In vivo programs

    CRISPR Therapeutics has moved into in vivo editing, delivering the editor into the patient’s body rather than editing cells outside it.

    • CTX310 (ANGPTL3). An LNP-delivered CRISPR/Cas9 therapy that edits the ANGPTL3 gene in liver cells after a single course of infusions. In Phase 1 results published in NEJM in November 2025, the highest dose lowered ANGPTL3 by a mean 73%, triglycerides by 55% and LDL cholesterol by 49%. No treatment-related serious adverse events were reported.[3] Two of 15 participants had serious adverse events that were not attributed to the therapy.[8] At one year, the company reported that the highest dose lowered ANGPTL3 by a mean 79%, triglycerides by 48% and LDL cholesterol by 53%, with no further treatment-related adverse events.[9] Phase 1b data in severe hypertriglyceridemia are due at the American Heart Association meeting on 9 November 2026.[10]
    • New trials in 2026. The company started Phase 1 trials of CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency.[11]
    • Liver portfolio. As of October 2026 its disclosed in vivo liver programs were CTX310 (ANGPTL3), CTX340 (angiotensinogen), CTX321 (LPA, for raised lipoprotein(a)) and CTX460 (SERPINA1), which uses the company’s SyNTase editing technology.[12]
    • Stem cells in the body. The company is working on editing blood-forming stem cells inside the body with lipid nanoparticles, which it says could reach more sickle cell and beta-thalassemia patients than ex vivo therapy.[13]

    CTX310 uses lipid-nanoparticle-delivery to reach liver cells.[3] Another in vivo CRISPR therapy, Intellia’s lonvo-z, is under FDA review.[14]

    Cell therapies

    The company also develops gene-edited cell therapies. Zugo-cel (formerly CTX112) is in Phase 1 trials in autoimmune disease and B-cell cancers, and the company is building an in vivo CAR-T platform using antibody-targeted lipid nanoparticles.[15] Related approaches are covered in car-t-cell-therapy and in-vivo-car-t.

    For Casgevy, the partners had filed in Saudi Arabia and the UK by August 2026 to extend use to children aged 5 to 11.[16] Vertex says it is doing preclinical work on gentler conditioning regimens, which could widen eligibility.[17]

    What to watch

    • Casgevy uptake after the pediatric label expansion, which Vertex estimates adds about 5,500 eligible patients.[18]
    • Whether CTX310’s lipid-lowering effects hold up in larger trials.[3]
    • Safety in in vivo editing generally. A competitor’s in vivo CRISPR trials were put on clinical hold in 2025 after a severe liver event.[19]

    Questions readers ask

    What is CRISPR Therapeutics' relationship to Casgevy?

    It co-developed Casgevy with Vertex Pharmaceuticals. Vertex leads development, manufacturing and commercialization, and the two companies share profits and program costs 60/40.[2]

    What did the CTX310 trial show?

    In Phase 1 data published in NEJM in November 2025, the highest dose lowered ANGPTL3 by a mean 73%, triglycerides by 55% and LDL cholesterol by 49%, with no treatment-related serious adverse events reported.[3]

    Where is CRISPR Therapeutics based?

    Zug, Switzerland, with a US subsidiary and research operations in Boston and San Francisco.[1]

    Sources

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

    1. [1]

      CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with a US subsidiary and research operations in Boston and San Francisco. confirmedas of 2026-10-10

    2. [2]

      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

    3. [3]

      In November 2025 CRISPR Therapeutics reported Phase 1 data, published in NEJM, for CTX310, an LNP-delivered CRISPR therapy that edits the ANGPTL3 gene in liver cells; at the highest dose mean ANGPTL3 fell 73%, triglycerides 55% and LDL cholesterol 49%, with no treatment-related serious adverse events reported. confirmedas of 2026-10-10

    4. [4]

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

    5. [5]

      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

    6. [6]

      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

    7. [7]

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

    8. [8]

      In the published CTX310 Phase 1 trial, serious adverse events occurred in two of 15 participants (13%): one had a spinal disk herniation and the other died suddenly 179 days after treatment with the lowest, 0.1 mg per kilogram dose; the authors reported no dose-limiting toxic effects related to CTX310. confirmedas of 2025-11-08

    9. [9]

      At the European Society of Cardiology Congress on 28 August 2026, CRISPR Therapeutics reported one-year Phase 1a follow-up for CTX310, with mean reductions from baseline at the highest dose of 79% for ANGPTL3, 48% for triglycerides and 53% for LDL cholesterol, and said no additional treatment-related adverse events had occurred since the previous update; the data were published in NEJM the same day. confirmedas of 2026-08-28

    10. [10]

      On 8 October 2026 CRISPR Therapeutics said Phase 1b data for CTX310 in severe hypertriglyceridemia would be presented in a late-breaking oral session at the American Heart Association Scientific Sessions on 9 November 2026. confirmedas of 2026-10-08

    11. [11]

      In 2026 CRISPR Therapeutics started Phase 1 trials of CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency, and presented CTX310 durability data at the European Society of Cardiology Congress in August 2026. confirmedas of 2026-10-10

    12. [12]

      CRISPR Therapeutics' disclosed in vivo liver-editing portfolio in October 2026 comprises CTX310 (ANGPTL3), CTX340 (angiotensinogen, for refractory hypertension), CTX321 (LPA, for elevated lipoprotein(a)) and CTX460 (SERPINA1, for alpha-1 antitrypsin deficiency), the last using the company's proprietary SyNTase editing technology. confirmedas of 2026-10-08

    13. [13]

      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

    14. [14]

      On 8 September 2026 Intellia said the FDA had accepted its lonvo-z application with Priority Review and a target action date of 10 March 2027; the company says lonvo-z would be the world's first in vivo CRISPR-based therapy if approved. confirmedas of 2026-10-10

    15. [15]

      CRISPR Therapeutics is also developing gene-edited cell therapies beyond blood disorders: zugocabtagene geleucel (zugo-cel, formerly CTX112) is in Phase 1 trials in autoimmune disease and B-cell malignancies, and the company is building an in vivo CAR-T platform using an antibody-conjugated lipid nanoparticle system to deliver editing to immune cells. confirmedas of 2026-08-03

    16. [16]

      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

    17. [17]

      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

    18. [18]

      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

    19. [19]

      On 29 October 2025 the FDA placed clinical holds on Intellia's Phase 3 MAGNITUDE and MAGNITUDE-2 trials of nex-z after a patient dosed in MAGNITUDE had Grade 4 liver transaminase elevations and increased bilirubin. confirmedas of 2026-10-10

    20. [20]

      Lonvo-z (lonvoguran ziclumeran) is Intellia's one-time, in vivo CRISPR/Cas9 treatment for hereditary angioedema, designed to permanently lower kallikrein by inactivating the KLKB1 gene, and given in an outpatient setting. confirmedas of 2026-10-10

    Revision history (2)
    1. Page created.
    2. Added CTX310 safety, one-year and upcoming data, the in vivo portfolio, cell-therapy programs and Casgevy filings.

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

    Cite this page

    "CRISPR Therapeutics." ContentLora, updated Oct 10, 2026. https://contentlora.com/wiki/crispr-therapeutics

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    Casgevy Co-developer ofCRISPR-Cas9 UsesLipid nanoparticle … UsesIntellia Therapeuti… Competitor
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