Tekmira Pharmaceuticals Corporation (NASDAQ: TKMR) is riding high on the Ebola scare, up nearly 130% in 3 months. The company has published a series of peer reviewed articles showing the effectiveness of their experimental therapy in non-human primates. The therapy itself is a 2′-O-methyl modified small interfering RNA (siRNA) encapsulated in a lipid nanoparticle (LNP). The company’s LNP technology may be a solution for the delivery problem that has seemingly plagued the therapeutic RNA field. Recent advances in oligonucleotide synthesis have allowed manufacturers to supply nearly any desired modified oligonucleotide or lipid to pharmaceutical companies developing new therapies. According to Paola Vecino, Ph.D. (lipids/RNA interference expert), “Phospholipid-based LNPs enter the cell and release their siRNA content. Interfering RNA is designed to block the translation of the viral nucleoporin mRNA. This approach does not interfere in the viral cycle but thwarts the mechanism by which the virus escapes the innate immunological defenses of the host, blocking the production of interferon, thereby boosting its infectivity.”
Drug discovery scientists in Europe are encouraged to contact Paola and her colleagues at tebu-bio to discuss their therapeutic RNA projects.