Cyclodextrin-Containing Polymers: Versatile Platforms of Drug Delivery Materials
Figure 10
Interim data from a first-in-man, phase I clinical evaluation of CALAA-01 reveals RRM2 down regulation via an RNAi mechanism of action. (a) Measurements of RRM2 mRNA or protein levels in tumor biopsies from three patients (A, B, and C2) obtained before or after CALAA-01 treatment reveal significant reductions in target expression levels. (b) 5′-RLM-RACE analysis of RNA from one patient (C2) reveals evidence of the precise RRM2 mRNA cleavage product expected from RNAi-mediated down-regulation from the C05C siRNA contained within CALAA-01. This was the first such evidence of the RNAi mechanism of action in humans of any kind (from [26]).
