AstraZeneca's GPC3 CAR-T shows 44% response rate in liver cancer, Nature reports
In 36 heavily pretreated advanced hepatocellular carcinoma patients, C-CAR031/AZD7003 produced tumor shrinkage in most patients but a 4.2-month median progression-free survival.
Executive Summary
- A Phase 1 first-in-human study of a glypican-3-targeted, armored CAR-T therapy reported mature results in advanced hepatocellular carcinoma, showing tumor shrinkage in most treated patients and a response rate near 44%, but with responses that did not persist much beyond four months for most patients who responded.
- The therapy, now known as AZD7003, was co-developed in China before AstraZeneca took sole global control of its development, making this liver cancer dataset a benchmark for how far the company's GPC3 CAR-T franchise can travel.
- A high rate of tumor shrinkage and a double-digit-month median survival sit alongside a progression-free survival and duration of response each measured in a few months, a split that keeps the result short of a settled efficacy verdict.
- The safety profile read as consistent with the known cytokine-release and non-hematologic toxicity pattern for CAR-T therapies, without an unexpected new signal.
The publication
AbelZeta Pharma announced that Nature published mature clinical results from an investigator-initiated, first-in-human study of C-CAR031, an autologous glypican-3 (GPC3)-targeted CAR-T therapy armored with AstraZeneca's dominant-negative TGF-beta receptor II technology, designed to blunt the immunosuppressive tumor environment. The study, run by Dr. Tingbo Liang and Dr. Qi Zhang at The First Affiliated Hospital, Zhejiang University School of Medicine, enrolled 36 patients with advanced hepatocellular carcinoma who had received a median of four prior lines of therapy. The publication updates preliminary data the companies presented at the 2024 American Society of Clinical Oncology meeting. AstraZeneca now solely leads global development of the asset, renamed AZD7003, after acquiring AbelZeta's remaining China rights in January 2026. AbelZetaAbelZeta announces publication in Nature journal highlighting clinical findings for an armored autologous GPC3 CAR-T therapy in patients with advanced hepatocellular carcinomaJul 23, 2026
How it was done
The trial registered under NCT05155189 is a Phase 1, open-label, non-randomized study running in China, with an anticipated enrollment of 44 patients across three arms and a primary outcome measure of adverse events of special interest. The Nature publication reports on 36 patients treated and analyzed for efficacy and safety, short of the trial's target enrollment. Eligibility required GPC3-expressing hepatocellular carcinoma that had relapsed or progressed after at least one prior systemic therapy. NCT05155189+1A Study to Evaluate Safety and Efficacy of Armored CAR-T Cell Injection C-CAR031 in Advanced Hepatocellular CarcinomaNCT05155189AbelZeta announces publication in Nature journal highlighting clinical findings for an armored autologous GPC3 CAR-T therapy in patients with advanced hepatocellular carcinomaJul 23, 2026
The results
Tumor regression occurred in 32 of 36 patients, with a median best target-lesion reduction of 41.6%. The objective response rate was 44.4%, with a median duration of response of 4.4 months (95% CI 2.9 to 7.4). Median progression-free survival was 4.2 months (95% CI 2.9 to 4.8), and median overall survival was 14.2 months (95% CI 10.1 to not evaluable). The gap between the tumor-shrinkage rate and the shorter progression-free and duration-of-response figures means that most patients who benefited saw that benefit fade within months even as overall survival extended further. AbelZetaAbelZeta announces publication in Nature journal highlighting clinical findings for an armored autologous GPC3 CAR-T therapy in patients with advanced hepatocellular carcinomaJul 23, 2026
Safety
Two patients had grade 3 cytokine release syndrome and nine patients had non-hematologic adverse events of grade 3 or higher. AbelZeta and the investigators characterized the safety profile as manageable, and the toxicity pattern read as consistent with known CAR-T class effects rather than a new signal. AbelZetaAbelZeta announces publication in Nature journal highlighting clinical findings for an armored autologous GPC3 CAR-T therapy in patients with advanced hepatocellular carcinomaJul 23, 2026
Trial history
The registry record shows the trial's primary completion date moved from April 2024 to May 2026 and its enrollment target was revised from 20 to 48 and then to 44 patients between February and April 2024. The trial has run since December 2021 and remains listed as recruiting. Those revisions reflect a multi-year, multi-arm dose-exploration study rather than a single fixed cohort, and they predate the mature results now reported. NCT05155189A Study to Evaluate Safety and Efficacy of Armored CAR-T Cell Injection C-CAR031 in Advanced Hepatocellular CarcinomaNCT05155189
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