{"entity":{"id":"ast-201","kind":"drug","name":"AST-201","aka":[],"tldr":"AST-201 is an experimental aptamer-drug conjugate from Aptamer Sciences, in phase 1 trials for hepatocellular carcinoma and GPC3-positive advanced solid tumours.","summary":"AST-201 is an aptamer-drug conjugate (ApDC) from Aptamer Sciences. A DNA aptamer that binds GPC3 with gemcitabine built into its sequence; once taken into the cell it releases the active gemcitabine form, designed to get round known gemcitabine resistance mechanisms. Its furthest stage is phase 1, for hepatocellular carcinoma and GPC3-positive advanced solid tumours. ClinicalTrials.gov registers it in one study linked below. Whether it works in people has not been established unless a trial below reports it.","status":"phase-1","asOf":"2026-11-03","links":[{"label":"Aptamer Sciences: aptsci.com","url":"https://www.aptsci.com/en/pipeline/pipeline.php"},{"label":"Aptamer Sciences: aptsci.com","url":"https://www.aptsci.com/en/pipeline/pipeline.php"},{"label":"ClinicalTrials.gov: trials of AST-201","url":"https://clinicaltrials.gov/search?intr=AST-201"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc","metastatic-cancer"],"sections":[],"technologies":[],"targets":["gpc3"],"drugs":[],"companies":["aptamer-sciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06687941"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo gap audit, class 5 (company pipeline pages and ClinicalTrials.gov v2)","editedOn":"2026-11-03"},"code":"AST-201","modality":"Aptamer-drug conjugate (ApDC)","mechanism":"A DNA aptamer that binds GPC3 with gemcitabine built into its sequence; once taken into the cell it releases the active gemcitabine form, designed to get round known gemcitabine resistance mechanisms.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/ast-201/","neighbours":{"cancer":[{"id":"hcc","kind":"cancer","name":"Hepatocellular carcinoma","route":"/cancers/hcc/"},{"id":"metastatic-cancer","kind":"cancer","name":"Metastatic cancer (cancer that has spread)","route":"/cancers/metastatic-cancer/"}],"target":[{"id":"gpc3","kind":"target","name":"Glypican-3","route":"/targets/gpc3/"}],"company":[{"id":"aptamer-sciences","kind":"company","name":"Aptamer Sciences","route":"/companies/aptamer-sciences/"}],"trial":[{"id":"nct06687941","kind":"trial","name":"A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors","route":"/trials/nct06687941/"}]}}