ANB-004

ANB-004 is an **adeno-associated virus serotype 9 (AAV9)**-based gene therapy developed to deliver a functional copy of the human **survival motor neuron 1 (SMN1) gene**. It is administered as a single intravenous infusion and is designed to address the genetic root cause of **5q spinal muscular atrophy (SMA)** by restoring SMN protein expression. The therapy is being tested primarily in infants and neonates with SMA who have a homozygous or heterozygous deletion in the SMN1 gene and 2–3 copies of the SMN2 gene. ANB-004 is under investigation in open-label, non-comparative, dose-escalation clinical trials and aims to halt disease progression by enabling endogenous production of the SMN protein.[1][2][3][4][9]

Other names

ANB-4ANB4ANB 4