Nature Medicine, Published online: 09 September 2026; doi:10.1038/s41591-026-04607-zFollowing preclinical development, a randomized first-in-human trial showed that treatment with an inhaled, lung-epithelium-targeted siRNA directed against the receptor for advanced glycation end-products (RAGE) was safe and well tolerated and reduced RAGE levels in serum and bronchoalveolar lavage fluid.

Nature Medicine, Published online: 07 April 2026; doi:10.1038/s41591-026-04307-8In a phase 1 basket trial, the small interfering RNA zodasiran, targeting ANGPTL3, lowered triglycerides in patients with severe hypertriglyceridemia and lowered both triglycerides and low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia.