The effective delivery of target-specific siRNA to the brain by exploiting the exosomes derived from dendritic cells renders the paradigm shift for the prospective use of nanosized exosomes as a delivery system. Although the in vivo targeting strategies by other nanovesicles like liposomes exist, still this novel exosome-based delivery approach holds an inclusive dominance of in vivo security and reduced immunogenicity. Achieving promising exosome-based delivery strategies warrants more desirable exploration of their biology. Over the years, the invention of novel production, characterization, targeting strategies, and cargo loading techniques of exosome improved its ability to reach clinics. Essentially, exosome-based delivery of therapeutics assures to conquer the major hurdles, like delivery of cargos across impermeable biological barriers, like the blood–brain barrier, biocompatibility, increased solubility, metabolic stability, improved circulation time, target specific delivery, and pharmacokinetics, and thereby enhanced the efficacy of loaded therapeutic agents. In this article, we cover the current status of exosome as a delivery vehicle for therapeutics and the challenges that need to be overcome, and we also discuss future perspectives of this exciting field of research to transform it from bench to clinical reality.