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.
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