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.
Drugs & Drug Targets
DrugTargetType