AVXS-301

AVXS-301 is an investigational gene therapy based on an adeno-associated virus serotype 9 (AAV9) vector engineered to deliver a short hairpin RNA (shRNA) sequence targeting the human superoxide dismutase 1 (SOD1) gene[7][19][23]. Its purpose is to downregulate mutant SOD1 involved in familial amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, aiming to reduce neurotoxicity and slow disease progression. Preclinical studies in ALS mouse models and non-human primates demonstrated that one-time administration markedly prolongs survival and preserves motor function. This therapy is in preclinical development for SOD1-mutant familial ALS, showing safety and CNS distribution via intrathecal or intracerebroventricular administration[4][7].

Brand names

AVXS-301AVXS301AVXS 301

Other names

AAV9-SOD1-shRNAAAV-9-SOD1-shRNAAAV 9-SOD1-shRNAAAV9-GFP-shRNA-SOD1AAV-9-GFP-shRNA-SOD1AAV 9-GFP-shRNA-SOD1