RNA interference; Novel strategy in treatment of cancer

CALAA-01

The siRNA nanoparticle is based on a polymer of cationically derivatized cyclodextrin complexed with the anionic siRNA payload. The nanoparticles are stabilized by binding of hydrophobically terminated PEG to the cyclodextrin rings, which occurs via the interaction of the hydrophobe (guest) within the core of the cyclodextrin (host). Delivery of cyclodextrin-bound-PEG further grafted with transferrin — a protein for which many tumour cells over-express a receptor — resulted in knockdown of target messenger RNA and the reduction of protein levels in tumours, opening the door for further clinical investigations focused on antitumour activity.

Reference: Hubbell, Jeffrey A., and Robert Langer. "Translating materials design to the clinic." Nature materials 12.11 (2013): 963-966.

The whole of science is nothing more than a refinement of everyday thinking; Albert Einstein