CALAA-01

CALAA-01 is an experimental, targeted nanoparticle therapeutic designed for the treatment of cancer. It was the first RNA interference (RNAi)-based drug to be administered to cancer patients in clinical trials. The drug consists of a four-component system that self-assembles into nanoparticles containing small interfering RNA (siRNA) targeting the M2 subunit of ribonucleotide reductase (RRM2), a clinically validated anticancer target. The delivery system uses a cyclodextrin-based polymer and includes human transferrin protein as a targeting ligand to engage transferrin receptors, which are upregulated on many tumor cells. Upon intravenous administration, these nanoparticles bind to transferrin receptors on tumor cells and enter via endocytosis; inside the cell, they release siRNA that silences RRM2 expression through RNA interference, thereby inhibiting DNA synthesis and tumor growth. Developed by Calando Pharmaceuticals (a subsidiary of Arrowhead Research Corporation), CALAA-01 advanced through phase 1 clinical trials in patients with solid tumors refractory to standard therapies but development has since been discontinued[1][2][3][4][5][6][7][8].

Other names

transferrin receptor-targeted anti-RRM2 siRNA CALAA-01