AAV9-CAG-hSGPL1

AAV9-CAG-hSGPL1 is an experimental gene therapy utilizing an adeno-associated virus serotype 9 (AAV9) vector to deliver the human SGPL1 gene under the control of a CAG promoter. The therapeutic mechanism involves augmenting sphingosine-1-phosphate lyase (SPL) activity in lung tissue, which reduces sphingosine-1-phosphate (S1P) levels and attenuates pro-fibrotic signaling. The treatment targets idiopathic pulmonary fibrosis (IPF) by decreasing fibroblast activation, reducing expression of pro-fibrotic cytokines (TNFα, IL-1β), and suppressing pro-fibrotic genes including fibronectin, transforming growth factor-beta 1 (TGF-β1), alpha-smooth muscle actin (ACTA2), and collagen genes. The single intravenous dose has demonstrated proof-of-concept efficacy in bleomycin-induced pulmonary fibrosis animal models.

Other names

AAV9-mediated overexpression of human SGPL1AAV-9-mediated overexpression of human SGPL1AAV 9-mediated overexpression of human SGPL1AAV9 sphingosine-1-phosphate lyase gene therapyAAV-9 sphingosine-1-phosphate lyase gene therapyAAV 9 sphingosine-1-phosphate lyase gene therapy