Genetic Therapy
- Name
- Genetic Therapy
- Accession Number
- DBCAT001637
- Description
Not Available
- Drugs
Drug Drug Description Sitimagene ceradenovec Intended for the treatment of brain cancer. Onasemnogene abeparvovec A gene therapy used to treat neonatal and infant patients with spinal muscular atrophy caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. 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. Valoctocogene roxaparvovec An adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A. Eladocagene exuparvovec A recombinant AAV2 vector containing the human aromatic L-amino acid decarboxylase (AADC) gene indicated for the treatment of AADC deficiency. Nadofaragene firadenovec A non-replicating adenoviral vector-based gene therapy used to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). BAX 335 BAX 335 is an adeno-associated virus vector 8 (AAV8)–based FIX gene therapy being investigated for the treatment of hemophilia B. 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 Valoctocogene roxaparvovec Coagulation factor VIII (F8) target Lovotibeglogene autotemcel Hemoglobin subunit beta target