Cellular and Gene Therapy

Name
Cellular and Gene Therapy
Accession Number
DBCAT005459
Description

Not Available

Drugs
DrugDrug Description
Onasemnogene abeparvovecA 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 autotemcelA 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 roxaparvovecAn adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A.
Etranacogene dezaparvovecAn adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX deficiency).
Nadofaragene firadenovecA 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 geperpavecGene therapy used to treat wounds associated with dystrophic epidermolysis bullosa.
DonislecelAn allogeneic pancreatic islets cellular therapy indicated for the treatment of type 1 diabetes
Drugs & Drug Targets
DrugTargetType
Valoctocogene roxaparvovecCoagulation factor VIII (F8)target