ABSTRACT

RNA therapeutics is coming off age. Just recently, Alnylam scored their second FDA approval for an RNA interference drug in the course of 16 months. With its momentum building and a cumulative annual growth rate of ~10% predicted for the next decade, the RNA therapeutics field is poised for great breakthroughs in the coming years. RNA nanotechnology is ideally suited to capitalize on this impetus and join the field with industry first therapeutic applications in the clinic. One challenge on the path to the clinic has been the large-scale production of therapeutic grade oligonucleotides and RNA nanoparticles. In this chapter, we review the current standards in laboratory to industrial-scale techniques for the purification of oligonucleotides as well as recent advances in the preparative purification of long oligonucleotides and RNA nanoparticles.