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CTX001
CTX001 is a genetically modified T-cell therapy that enhances patient immune cells to recognize and destroy cancer cells expressing specific antigens.
CTX001 is a genetically modified T-cell therapy that enhances patient immune cells to recognize and destroy cancer cells expressing specific antigens. Used for Relapsed or refractory multiple myeloma, Relapsed or refractory acute lymphoblastic leukemia (ALL).
At a glance
| Generic name | CTX001 |
|---|---|
| Also known as | Exagamglogene autotemcel, Exa-cel |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CAR-T cell therapy / Gene-edited T-cell therapy |
| Target | BCMA (B-cell maturation antigen) / Tumor-associated antigens |
| Modality | Biologic |
| Therapeutic area | Oncology / Hematologic Malignancies |
| Phase | Phase 3 |
Mechanism of action
CTX001 involves ex vivo modification of a patient's own T cells using CRISPR gene-editing technology to disrupt the T-cell receptor and CD52 gene, then introducing a new T-cell receptor targeting a tumor-associated antigen. The edited cells are expanded and reinfused into the patient to mount an anti-tumor immune response. This approach allows for precise targeting of cancer cells while reducing off-target toxicity.
Approved indications
- Relapsed or refractory multiple myeloma
- Relapsed or refractory acute lymphoblastic leukemia (ALL)
Common side effects
- Cytokine release syndrome
- Neurotoxicity
- Cytopenias
- Infections
- Infusion reactions
Key clinical trials
- A Long-term Follow-up Study in Participants Who Received CTX001 (PHASE3)
- Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype (PHASE3)
- Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease (PHASE3)
- An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA) (PHASE1, PHASE2)
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) (PHASE3)
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) (PHASE3)
- A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia (PHASE2, PHASE3)
- A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- CTX001 CI brief — competitive landscape report
- CTX001 updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI