Genetic Therapy

Name
Genetic Therapy
Accession Number
DBCAT001637
Description

Not Available

Drugs
DrugDrug Description
Sitimagene ceradenovecIntended for the treatment of brain cancer.
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.
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.
Valoctocogene roxaparvovecAn adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A.
Eladocagene exuparvovecA recombinant AAV2 vector containing the human aromatic L-amino acid decarboxylase (AADC) gene indicated for the treatment of AADC 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).
BAX 335BAX 335 is an adeno-associated virus vector 8 (AAV8)–based FIX gene therapy being investigated for the treatment of hemophilia B.
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
DrugTargetType
Betibeglogene autotemcelAlpha globintarget
Valoctocogene roxaparvovecCoagulation factor VIII (F8)target
Lovotibeglogene autotemcelHemoglobin subunit betatarget