In a recent study published in Nucleic Acid Therapeutics, Isis scientists published the first study to comprehensively evaluate the activity and distribution of Isis’ Generation 2.5 antisense drugs. In order to evaluate the broad distribution and activity of Generation 2.5 drugs in multiple tissues, scientists selected MALAT1, a noncoding, highly expressed gene in multiple organs. The results from this study showed that systemic delivery of Isis’ Generation 2.5 antisense drugs effectively reduced MALAT1 RNA in many tissues, including the liver, kidney, lung, muscle, adipose, adrenal gland and peripheral nerves. Isis also evaluated the effect of Isis’ Generation 2.0 chemistry on the MALAT1 gene, confirming that Generation 2.5 antisense drugs are more potent and active in more tissues within the body than Isis’ Generation 2.0 drugs.
The journal article titled, “Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals” is now available in Nucleic Acid Therapeutics.
This is the first study to comprehensively evaluate antisense drug distribution and efficacy in an entire organism. The comparative data between Generation 2.0 and Generation 2.5 drugs demonstrate Isis’ continued success in improving the potency of antisense drugs and support a broad evaluation of both Generation 2.0 and Generation 2.5 antisense drugs to many different disease targets in many different tissues.
Hung, G. et al. (2013) Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals. Nucleic Acid Therapeutics. 23:369-378