There are about 10,000 known and registered genetic rare diseases affecting approximately 400 million people worldwide. Rare diseases represent a substantial unmet medical need for patients and development of new therapeutic tools to treat rare diseases are often not considered profitable and too expensive. The majority of current treatments are based on protein-targeting chemicals and small molecules, which frequently show limited efficiency because of their short half-life, poor target affinity, and ineffective cell penetration, which can result in drug resistance. Nucleic acid based treatments including RNA Editing may provide better therapeutic options that aim to fix the genetic problem at its source by modifying and repairing the disease causing gene’s transcript. RNA editing is inherently safer to develop since it is reversible and does not permanently alter the genetic code. We are excited about the RNA editing space and the potential for development of a new class therapy for multiple orphan rare diseases.