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Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

2014; National Academy of Sciences; Volume: 111; Issue: 31 Linguagem: Inglês

10.1073/pnas.1411393111

1091-6490

Jonathan E. Zuckerman, Ismael Gritli, Anthony W. Tolcher, Jeremy D. Heidel, Dean Lim, Robert J. Morgan, Bartosz Chmielowski, Antoni Ribas, Mark E. Davis, Yun Yen,

Nanoparticle-Based Drug Delivery

Significance CALAA-01 is a targeted nanoparticle containing siRNA that is a first-in-class experimental therapeutic for cancer. To our knowledge, it is the first targeted, polymer-based nanoparticle-carrying siRNA to be systemically administered to humans. Results from a human phase Ia/Ib clinical trial are presented and correlated to preclinical animal data to provide an initial assessment of how this class of experimental therapeutics is translated from animals to humans.