Secarna Pharmaceuticals, a biopharmaceutical company specializing in antisense oligonucleotide (ASO) therapies, has published new preclinical data in The Journal of Immunology. The data demonstrates the potent therapeutic activity of their LNAplus(TM) ASO targeting NLRP3 in debilitating autoinflammatory diseases.
NLRP3 is a central component of the inflammasome pathway, which is overactive in many inflammatory diseases. Secarna's LNAplus™ ASOs effectively suppress the expression of NLRP3, providing potential treatment options for these conditions.
The study specifically focuses on Cryopyrin-associated periodic syndrome (CAPS), a group of rare inherited autoinflammatory diseases caused by overactive NLRP3. By using NLRP3-specific ASOs, the researchers were able to interrupt inflammatory downstream effects in immune cells and achieve significant symptom relief and survival prolongation in a CAPS disease model.