RNA therapy is basically the technique of utilizing molecules based upon RNA for treating disease via modulating the respective signaling cascades. Its major efficacy has been observed during the COVID-19 pandemic while developing mRNA vaccines. It has also been found that RNA-based molecules can be used to cure neurodegenerative diseases, cancers, metabolic syndromes, and even rarely occurring genetic diseases. The structural and catalytic properties of RNA such as its use as a genetic template, scaffold, and enzyme and its regulatory role have proved to be major benefits for the diagnostics and therapeutics of several pathological conditions. Its utilization must be considered for its affinity and specificity for other biomolecules. The major breakthroughs in the field of medicine and biochemistry are the development of miRNAs, antisense oligonucleotides, RNA interference, and especially CRISPR-Cas9 technology. However, poor pathological properties, difficulty in intracellular delivery, and inflammatory toxicity are the problems to be curbed while applying this kind of therapy. RNA-based therapy is way more efficient than conventional drug delivery technologies, as its complementary nature saves time and labor both to treat the rarest of rare conditions. The current book chapter deals with several challenges related to RNA-based therapeutics and novel opportunities in terms of advancements in the field of RNA therapeutics to target and cure various types of human disorders while the respective cascade.