Autologous Hematopoietic Stem Cell-Based Gene Therapy

Autologous Hematopoietic Stem Cell-Based Gene Therapy
Accession Number

Genetic therapy consisting of autologous hematopoietic stem cells (HSCs).

DrugDrug Description
Betibeglogene autotemcelA 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 autotemcelA gene therapy comprising autologous CD34+ enriched stem cells encoding the human arylsulfatase A (ARSA) gene used to treat metachromatic leukodystrophy (MLD).
Lovotibeglogene autotemcelAn autologous CD34+ cell therapy used to treat sickle cell disease by providing a functional copy of the beta-globin gene
Drugs & Drug Targets
Betibeglogene autotemcelAlpha globintarget
Atidarsagene autotemcelHuman Arylsulfatase A mRNAtarget
Lovotibeglogene autotemcelHemoglobin subunit betatarget