s6k1 siRNA

S6K1 siRNA is a preclinical-stage RNA interference (RNAi) therapeutic developed at the University of Massachusetts Chan Medical School. It is a fully chemically modified tetravalent small interfering RNA (siRNA) designed to target and silence the gene encoding ribosomal protein S6 kinase beta-1 (S6K1), a downstream effector of the mTORC1 signaling pathway. Administered via intravitreal injection, the therapeutic is engineered to enrich specifically in photoreceptor cells. In preclinical models of age-related macular degeneration (AMD), including mice, pigs, and non-human primates, S6K1 silencing has been shown to reverse AMD-associated phospholipid changes, restore lysosomal activity in retinal pigment epithelium (RPE) cells, reduce lipoprotein buildup at Bruch's membrane, and slow the growth of macular drusen. This long-lasting therapy is being developed as a potential treatment for both dry and wet AMD, including geographic atrophy, with the potential to reduce injection frequency to once or twice a year.

Other names

S6K1 small interfering RNAS-6K1 small interfering RNAS 6K1 small interfering RNAS6k1 siRNAS-6k1 siRNAS 6k1 siRNAtetravalent S6K1 siRNAtetravalent siRNA targeting S6K1