Drug profile · INN
exagamglogene autotemcel
CASGEVY
exagamglogene autotemcel (CASGEVY) is a b-cell lymphoma/leukemia 11a gene editing negative modulator. Associated with Vertex Pharmaceuticals (Australia) Pty..
Drug details — exagamglogene autotemcel
- Class
- Blood and blood forming organs (B06)
- Route
- —
- Patents
- 0
- Programs
- 11
- Data quality
- 0.89
Quick answer
exagamglogene autotemcel (CASGEVY) is a b-cell lymphoma/leukemia 11a gene editing negative modulator. Associated with Vertex Pharmaceuticals (Australia) Pty..
Key facts
Mechanism of action
B-cell lymphoma/leukemia 11A gene editing negative modulator
Primary target: BCL11 transcription factor A
Approval history
| Authority | Status | Date |
|---|---|---|
| EMA | approved | — |
Recent coverage
CRISPR Therapeutics Reports Q1 2026 Results as CASGEVY Gene Therapy Gains Global Regulatory Approval
CRISPR Therapeutics reports Q1 2026 financial results highlighting CASGEVY's approval in 10+ countries for sickle cell disease and beta thalassemia treatment.
Vertex CASGEVY Gene Therapy Reaches 60,000+ Eligible Patients Across 10 Countries as Q1 2026 Results Show Global Expansion
Vertex's CASGEVY, the first approved CRISPR gene therapy, now serves 60,000+ eligible sickle cell disease and beta thalassemia patients across 10 countries.
CRISPR Therapeutics' CASGEVY Reaches 60,000+ Eligible Patients Across 10 Countries Following Global Approvals
CRISPR Therapeutics reports CASGEVY now approved in 10 countries for severe sickle cell disease and beta thalassemia, reaching over 60,000 eligible patients worldwide.
FDA Approves First CRISPR Gene Therapy for Sickle Cell Disease
The FDA has approved Zynteglo, the first CRISPR gene therapy for sickle cell disease, offering new hope for patients with this debilitating condition.
Pipeline programs
Related & competitor drugs
Frequently asked questions
What is exagamglogene autotemcel?
exagamglogene autotemcel (CASGEVY) is a b-cell lymphoma/leukemia 11a gene editing negative modulator. Associated with Vertex Pharmaceuticals (Australia) Pty..
What is the brand name for exagamglogene autotemcel?
CASGEVY is a marketed brand name for exagamglogene autotemcel. Check FDA Drugs@FDA for the current US label holder and prescribing information.