| Casgevy (exagamglogene autotemcel) |
Vertex/CRISPR Therapeutics |
Sickle cell disease (SCD) β-thalassemia |
Approved by US FDA and European Commision (EC) |
Autologous genetically modified CD34+ cells |
| Lyfgenia (lovotibeglogene autotemcel) |
bluebird bio |
Sickle cell disease (SCD) |
Approved by US FDA |
Autologous genetically modified CD34+ cells |
| Libmeldy (OTL-200) |
Orchard Therapeutics |
Metachromatic leukodystrophy (MLD) |
Approved by EC, In review by US FDA |
Autologous genetically modified CD34+ cells |
| Skysona (elivaldogene autotemcel) |
bluebird bio |
Cerebral adrenoleukodystrophy |
Approved by US FDA and EC |
Autologous genetically modified CD34+ cells |
| Zynteglo (betibeglogene autotemcel) |
bluebird bio |
β-thalassemia |
Approved by US FDA and EC |
Autologous genetically modified CD34+ cells |
| MDR-101 |
Medeor Therapeutics |
Kidney transplant rejection |
Phase 3 |
Allogeneic unmodified CD34+ hematopoietic stem cells and CD3+ T-cells |
| OTL-203 |
Orchard Therapeutics |
Mucopolysaccharidosis type I (MPS-I) |
Phase 3 |
Allogeneic genetically modified CD34+ cells |