Davis, Mark E. (2009) The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic. Molecular Pharmaceutics, 6 (3). pp. 659-668. ISSN 1543-8384. doi:10.1021/mp900015y. https://resolver.caltech.edu/CaltechAUTHORS:20090820-150240761
Full text is not posted in this repository. Consult Related URLs below.
Use this Persistent URL to link to this item: https://resolver.caltech.edu/CaltechAUTHORS:20090820-150240761
Abstract
Experimental therapeutics developed to exploit RNA interference (RNAi) are now in clinical studies. Here, the translation from concept to clinic for the first experimental therapeutic to provide targeted delivery of synthetic, small interfering RNA (siRNA) in humans is described. This targeted, nanoparticle formulation of siRNA, denoted as CALAA-01, consists of a cyclodextrin-containing polymer (CDP), a polythethylene glycol (PEG) steric stabilization agent, and human transferrin (Tf) as a targeting ligand for binding to transferrin receptors (TfR) that are typically upregulated on cancer cells. The four component formulation is self-assembled into nanoparticles in the pharmacy and administered intravenously (iv) to patients. The designed features of this experimental therapeutic are described, and their functions illustrated
| Item Type: | Article | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Related URLs: |
| |||||||||
| ORCID: |
| |||||||||
| Additional Information: | © 2009 American Chemical Society. Publication Date (Web): March 6, 2009. Received: January 15, 2009. Accepted: March 6, 2009. I thank all my coauthors who are listed on the references provided. I especially thank Dr. Jeremy Heidel who provided the leadership in bringing CALAA-01 from the benchtop to the bedside. Lastly, I thank the patients who willingly participated in the clinical trial. M.E. Davis has been a consultant to and has financial interest in Calando Pharmaceuticals | |||||||||
| Subject Keywords: | CALAA-01; siRNA; targeted delivery; targeted nanoparticle; clinical trial | |||||||||
| Issue or Number: | 3 | |||||||||
| DOI: | 10.1021/mp900015y | |||||||||
| Record Number: | CaltechAUTHORS:20090820-150240761 | |||||||||
| Persistent URL: | https://resolver.caltech.edu/CaltechAUTHORS:20090820-150240761 | |||||||||
| Official Citation: | The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic Mark E. Davis Molecular Pharmaceutics 2009 6 (3), 659-668 | |||||||||
| Usage Policy: | No commercial reproduction, distribution, display or performance rights in this work are provided. | |||||||||
| ID Code: | 15195 | |||||||||
| Collection: | CaltechAUTHORS | |||||||||
| Deposited By: | Ruth Sustaita | |||||||||
| Deposited On: | 08 Sep 2009 21:53 | |||||||||
| Last Modified: | 08 Nov 2021 23:17 |
Repository Staff Only: item control page
