Antisense Morpholino Drug May Be Effective Against Deadly Marburg Virus; Current Human Fatality Rate Is 90%

An experimental drug that protected monkeys from the deadly Marburg virus appears to have potential for treating people who have been exposed to the virus, according to a study published in the July 23, 2015 issue of The New England Journal of Medicine. The article is titled “AVI-7288 for Marburg Virus in Nonhuman Primates and Humans.” Marburg virus is closely related to Ebola virus and, as Ebola, also causes a severe hemorrhagic fever. The research was jointly conducted by the U.S. Army Medical Research Institute of Infectious Diseases (USAMRIID) and the biotechnology firm Sarepta Therapeutics, Inc., using a compound known as AVI-7288. Taken together, the results of efficacy testing conducted in nonhuman primates and safety testing performed in a Phase I clinical trial suggest that AVI-7288 has the potential to be used to treat Marburg virus infection in humans when administered post-exposure, according to the authors. Case fatality rates associated with Marburg virus have been reported to be nearly 90 percent and the virus is deemed a potential "Category A" bioterrorism agent by the Centers for Disease Control and Prevention. No licensed vaccine or therapy is currently available for Marburg virus infection. For over a decade, USAMRIID and Sarepta have been collaborating to develop and test a class of antisense compounds known as phosphorodiamidate morpholino oligomers (or PMOs), according to senior author and USAMRIID Science Director Sina Bavari, Ph.D. Antisense drugs are designed to enter cells and eliminate viruses by preventing their replication, Dr. Bavari explained. The drugs act by blocking the translation of critical viral genetic sequences, preventing a key viral protein from being made, and giving the infected host time to mount an immune response and clear the virus.
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