.Pharmacolibrary.Drugs.B_BloodAndBloodFormingOrgans.B06A_OtherHematologicalAgents.B06AX05_ExagamglogeneAutotemcel.ExagamglogeneAutotemcel .Pharmacolibrary.Drugs.B_BloodAndBloodFormingOrgans.B06A_OtherHematologicalAgents.B06AX05_ExagamglogeneAutotemcel.ExagamglogeneAutotemcel| name: | ExagamglogeneAutotemcel |
| ATC code: | B06AX05 | route: | intravenous |
| n-compartments | 0 |
Exagamglogene autotemcel is an autologous, genetically modified CD34+ hematopoietic stem and progenitor cell product in which the erythroid-specific enhancer region of the BCL11A gene is disrupted by CRISPR/Cas9 technology. It is developed for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia and was approved in 2023 in the US and UK.
No pharmacokinetic parameters are available from published literature. As a gene-modified autologous cell therapy, traditional pharmacokinetic parameters (such as volume of distribution, clearance, bioavailability) are generally not applicable.