By Shiksha Mantri, Ph.D.
Nucleic acid therapy using RNA and DNA as the active pharmaceutical ingredient (API) has the potential to cause a paradigm shift in the way diseases are addressed. This innovative therapy targets the source of the disease at the genetic level and can be used to modulate the expression of one or more proteins simultaneously – a unique advantage over conventional biologics or small molecules. Given this mode of action, RNA and DNA therapeutics are a powerful means to treat, and in some cases cure, diseases that could not be addressed by other approaches. 1 Recognizing the potential of RNA-based therapeutics, this whitepaper focuses on lipid-based RNA therapeutic development.