scAAV9-VEGF-165

scAAV9-VEGF-165 is a gene therapy product that utilizes a self-complementary adeno-associated virus serotype 9 (scAAV9) vector to deliver the human vascular endothelial growth factor isoform 165 (VEGF-A_165) gene. The therapy is designed for intrathecal administration, enabling targeted delivery to the central nervous system and peripheral tissues. VEGF-A_165 is a potent angiogenic and neuroprotective factor that promotes blood vessel formation, modulates inflammation, and supports neuronal survival. In preclinical models of amyotrophic lateral sclerosis (ALS), intrathecal injection of scAAV9-VEGF-165 has been shown to reduce neuroinflammation by decreasing microglial activation in the CNS, inhibit macrophage invasion into peripheral nerves, activate pro-survival signaling pathways such as PI3K/Akt, increase anti-apoptotic Bcl‑2 levels, shift microglia toward an anti-inflammatory phenotype (M2), improve motor function, and prolong survival[1][6]. The primary indication under investigation is ALS.

Other names

self-complementary adeno-associated virus serotype 9 encoding vascular endothelial growth factor 165scAAV9-VEGFscAAV-9-VEGFscAAV 9-VEGF