AVI-7288

AVI-7288 is a phosphorodiamidate morpholino oligomer (PMO) developed as an antisense therapeutic targeting the messenger RNA encoding the nucleoprotein of Marburg virus (MARV). It utilizes Sarepta's proprietary PMOplus technology, which introduces positive charges into the PMO backbone to enhance binding to its viral RNA target. By binding to MARV nucleoprotein mRNA, AVI-7288 inhibits translation and disrupts viral replication. The drug has demonstrated dose-dependent efficacy in nonhuman primate models of Marburg virus infection, with survival rates reaching 100% at higher doses. In Phase I clinical trials in healthy adults, AVI-7288 was well tolerated up to 16 mg/kg/day for 14 days with no serious adverse events reported. Its primary indication is postexposure prophylaxis and treatment of Marburg virus infection[1][5][6][7][8].