Cellular and Gene Therapy
- Name
- Cellular and Gene Therapy
- Accession Number
- DBCAT005459
- Description
Not Available
- Drugs
Drug Drug Description 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. Elivaldogene autotemcel A gene replacement therapy for treating early cerebral adrenoleukodystrophy comprising autologous hematopoietic stem cells transduced with a viral vector encoding ABCD1 complementary DNA for human adrenoleukodystrophy protein. 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. Etranacogene dezaparvovec An adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX 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). Beremagene geperpavec Gene therapy used to treat wounds associated with dystrophic epidermolysis bullosa. Donislecel An allogeneic pancreatic islets cellular therapy indicated for the treatment of type 1 diabetes - Drugs & Drug Targets
Drug Target Type Valoctocogene roxaparvovec Coagulation factor VIII (F8) target