ADAMDEC1 inhibitor (AAV-mediated knockdown)

ADAMDEC1 inhibitor (AAV-mediated knockdown) is an experimental adeno-associated virus (AAV)-based gene therapy designed to knock down the expression of ADAM-like decysin-1 (ADAMDEC1). Developed by researchers at Southern Medical University, this therapeutic candidate is being investigated for the treatment of rheumatoid arthritis (RA). ADAMDEC1 is a metalloprotease that is significantly upregulated in the fibroblast-like synoviocytes (FLS) of RA patients, where it binds to and activates matrix metalloproteinase 3 (MMP3), leading to extracellular matrix degradation and cartilage destruction. Additionally, ADAMDEC1 overexpression promotes FLS proliferation, migration, and the M1 polarization of synovial macrophages, exacerbating the inflammatory response. In preclinical models of RA, AAV-mediated knockdown of ADAMDEC1 has been shown to reduce immune cell aggregation, cartilage destruction, and bone resorption, thereby ameliorating disease progression.