MDN-01

MDN-01 is an experimental RNA interference (RNAi) therapeutic developed by Marina Biotech (formerly MDRNA, Inc.) for the treatment of hypercholesterolemia, specifically targeting familial forms of the disease. The drug utilizes a proprietary "meroduplex" siRNA architecture, also known as mdRNA or UsiRNA, which features a double-stranded RNA molecule where the passenger (non-coding) strand is composed of two shorter, non-overlapping oligonucleotides. This "nicked" design is intended to enhance the thermodynamic stability and specificity of the guide strand's loading into the RNA-induced silencing complex (RISC), thereby minimizing off-target effects and improving potency. MDN-01 targets the messenger RNA (mRNA) encoding apolipoprotein B (ApoB), a vital structural protein required for the assembly and secretion of very-low-density lipoproteins (VLDL) and low-density lipoproteins (LDL) by the liver. By facilitating the catalytic degradation of ApoB mRNA, MDN-01 reduces the systemic levels of these atherogenic particles. Development of the program was discontinued following Marina Biotech's financial restructuring and a shift in corporate strategy.

Other names

apolipoprotein b meroduplex small interfering ribonucleic acidApoB-mdRNAApoB-UsiRNAApoB meroduplex siRNA