Autologous Hematopoietic Stem Cell-Based Gene Therapy
- Name
- Autologous Hematopoietic Stem Cell-Based Gene Therapy
- Accession Number
- DBCAT005722
- Description
Genetic therapy consisting of autologous hematopoietic stem cells (HSCs).
- Drugs
Drug Drug Description Betibeglogene autotemcel A hematopoietic stem cell-based gene therapy used in the treatment of β-thalassemia in adult and pediatric patients who require regular red blood cell transfusions. Atidarsagene autotemcel A gene therapy comprising autologous CD34+ enriched stem cells encoding the human arylsulfatase A (ARSA) gene used to treat metachromatic leukodystrophy (MLD). Lovotibeglogene autotemcel An autologous CD34+ cell therapy used to treat sickle cell disease by providing a functional copy of the beta-globin gene - Drugs & Drug Targets
Drug Target Type Betibeglogene autotemcel Alpha globin target Atidarsagene autotemcel Human Arylsulfatase A mRNA target Lovotibeglogene autotemcel Hemoglobin subunit beta target