APTA-12

APTA-12 is an aptamer–drug conjugate in which a guanine-rich G-quadruplex DNA aptamer targeting cell-surface nucleolin (derived from the AS1411 aptamer) is site-specifically modified by replacing the 14th nucleotide with gemcitabine monophosphate (dFdCMP), creating a “nucleotide analog drug conjugate” that delivers gemcitabine into nucleolin-overexpressing tumor cells.[2][10][12] This construct retains high affinity and specificity for nucleolin, is efficiently internalized by cancer cells, and releases gemcitabine intracellularly to inhibit DNA synthesis and induce apoptosis, showing markedly higher in vitro cytotoxicity than free gemcitabine or AS1411 and robust tumor growth inhibition in pancreatic cancer xenograft models with minimal systemic toxicity.[2][10][12] APTA-12 (also referred to as nuc-gemcitabine) is being developed by AptaBio Therapeutics, with licensing and co-development activities with Hope Biosciences, primarily for the treatment of solid tumors including pancreatic cancer.[4][7][14]

Brand names

nuc-Gemcitabine

Other names

nuc-gemcitabine