Fig. 5. Integrative nano-drug development.
(A) Amalgamation of cancer biology/genetics, bioengineering, i.e., the design and synthesis of nanotechnological platforms, and nanodrug development to enable the preclinical and clinical development of RNA-based nanoconjugates. (B) Validation scheme for the (pre-)clinical characterization of iCCG nano-conjugates. Initial studies aim to assess CCG expression in a panel of cancer cells to stratify lineages with high, low, or absent expression for subsequent functional studies to assess siCCG-nanoconjugates. Animal studies, together with toxicology, pharmacokinetic and –dynamic studies will enable early phase 0/I/II clinical studies in humans. Of note, model systems to validate iCCG nanoconstructs show different degrees of cost- and time effectiveness, and complexity.