# AST-201

Source: https://onco.cc/drugs/ast-201/  
OnCo record `ast-201` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Treatment
- Status: phase-1
- Last checked: 2026-11-03
- Tags: pipeline; ctgov-ingest
- 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.

## Sources

- Aptamer Sciences: aptsci.com: https://www.aptsci.com/en/pipeline/pipeline.php
- Aptamer Sciences: aptsci.com: https://www.aptsci.com/en/pipeline/pipeline.php
- ClinicalTrials.gov: trials of AST-201: https://clinicaltrials.gov/search?intr=AST-201

## Connected records

- cancers: [Hepatocellular carcinoma](https://onco.cc/cancers/hcc/), [Metastatic cancer (cancer that has spread)](https://onco.cc/cancers/metastatic-cancer/)
- targets: [Glypican-3](https://onco.cc/targets/gpc3/)
- companies: [Aptamer Sciences](https://onco.cc/companies/aptamer-sciences/)
- trials: [A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors](https://onco.cc/trials/nct06687941/)

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JSON: https://onco.cc/api/v1/entities/ast-201.json