CTX001 Uses, Dosage, Side Effects and more
CTX001 is being developed by CRISPR Therapeutics and Vertex. It is an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in clinical trials for the treatment of severe sickle cell disease and severe beta-thalassemia.
| Attribute | Details |
|---|---|
| Trade Name | CTX001 |
| Generic | CTX001 |
| Type | |
| Groups | Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | |
| Last Updated: | January 7, 2025 at 1:49 am |
