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Exa-cel
Exa-cel is an in vivo CRISPR gene-editing therapy that modifies the BCL11A gene to reactivate fetal hemoglobin production in patients with sickle cell disease or beta-thalassemia.
Exa-cel is an in vivo CRISPR gene-editing therapy that modifies the BCL11A gene to reactivate fetal hemoglobin production in patients with sickle cell disease or beta-thalassemia. Used for Sickle cell disease, Beta-thalassemia major.
At a glance
| Generic name | Exa-cel |
|---|---|
| Also known as | Exagamglogene autotemcel, CTX001 |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CRISPR gene-editing therapy |
| Target | BCL11A gene |
| Modality | Biologic |
| Therapeutic area | Hematology/Rare Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
Exa-cel uses CRISPR-Cas9 technology to edit the BCL11A gene in patient hematopoietic stem cells, which normally suppresses fetal hemoglobin (HbF) expression after birth. By disrupting BCL11A, the therapy allows reactivation of HbF production, which compensates for defective adult hemoglobin and reduces sickling or transfusion dependence. The edited cells are reinfused into the patient where they engraft and provide long-term therapeutic benefit.
Approved indications
- Sickle cell disease
- Beta-thalassemia major
Common side effects
- Myelosuppression
- Infection
- Infusion-related reactions
- Cytopenias
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)
- 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:
- Exa-cel CI brief — competitive landscape report
- Exa-cel updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI