CD40 antisense inhibitor

CD40 antisense inhibitor refers to a Generation 2.5 antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) that targets CD40 mRNA to reduce the expression of the CD40 protein. This therapeutic utilizes 2'-4' constrained ethyl (cET) modified bicyclic nucleic acid technology, which enhances potency and stability compared to earlier ASO generations. The drug is designed to inhibit the CD40-CD40L costimulatory pathway, a critical driver of immune activation and chronic inflammation. In preclinical models of renal disease, such as doxorubicin-induced nephropathy and glomerulonephritis, the ASO demonstrated significant reduction in kidney inflammation and fibrosis. A key advantage of this ASO approach is its ability to provide kidney-specific targeting—specifically to proximal tubule epithelial cells and podocytes—while avoiding the systemic thromboembolic complications often associated with antibody-based CD40 inhibitors.

Other names

CD40 ASOCD-40 ASOCD 40 ASOCD40 antisense oligonucleotideCD-40 antisense oligonucleotideCD 40 antisense oligonucleotideGeneration 2.5 CD40 ASOGeneration2.5 CD40 ASOGeneration-2.5 CD40 ASO