{
  "abstract": "Background Neoantigen-based cancer vaccines offer a promising approach for treating solid tumors, but clinical efficacy has been limited by two major challenges: low accuracy in neoantigen prediction, particularly for identifying neoantigens capable of boost pre-existing tumor-specific immune responses, and inefficient antigen delivery to antigen-presenting cells (APCs). Conventional prediction methods rely heavily on MHC-I binding affinity and low-sensitivity immunogenicity assays, often missing clinically relevant T cell targets. Additionally, most delivery platforms lack the ability to safely and systemically target APCs, which is key for overcoming immune tolerance and eliciting robust T cell responses. To address these limitations, we developed GT601, a next-generation neoantigen vaccine platform powered by NEOvigator™, an AI-guided discovery system that incorporates functional validation using autologous tumor-reactive T cells. This approach achieves over 40% precision in identifying immunogenic neoantigens across diverse tumor types. GT601 is formulated with ViLNP® Dakin, an APC-targeted lipid nanoparticle (LNP) system that enables efficient systemic delivery to APCs while minimizing off-target expression in parenchymal tissues, such as hepatocytes.Methods This is a single-arm, open-label Phase 1 study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of GT601 in patients with hepatocellular carcinoma (HCC) at high risk of recurrence following curative surgical resection. The study consists of an accelerated titration phase followed by a traditional 3+3 dose-escalation phase. Primary endpoints include safety and tolerability. Secondary endpoints include assessment of preliminary efficacy (per RECIST v1.1), pharmacokinetics, GT601 persistence, tumor-specific T cell reactivity, and cytokine release following administration. Key inclusion criteria are 1) histologically or pathologically confirmed HCC; 2) ECOG performance status of 0 or 1 with life expectancy > 12 weeks; 3) Child-Pugh class A to B7, without major vascular invasion (excluding Vp1/Vp2) or extrahepatic metastasis; 4) adequate organ function. Eligible patients will undergo GT601 product manufacturing based on the whole exome and RNA sequencing of surgically resected tumor tissues. GT601 will be administered intravenously between 8 to 12 weeks post-surgery, with timing determined by the investigator based on the participant’s recovery status. The trial is currently open and enrolling patients.",
  "authors": [
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China",
        "University of Southern California, Los Angeles, CA, USA"
      ],
      "name": "Pin Wang"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Liqing Ma"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Meng Zhao"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Zhenjiang Liu"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Yanbin Liu"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Jun Cui"
    },
    {
      "affiliations": [
        "Shanghai Vitalgen Biopharma, Shanghai, China"
      ],
      "name": "Xi Zhu"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Jingwei Sun"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Jing Yu"
    },
    {
      "affiliations": [
        "Grit Biotechnology Inc, Shanghai, China"
      ],
      "name": "Yarong Liu"
    }
  ],
  "title": "559 Trial in progress: first in human study of GT601, a next-generation neoantigen cancer vaccine for hepatocellular carcinoma",
  "uid": "9cd7e998-8878-5416-973a-47f76a4ae6da"
}