Gene therapy for liver cancer- version 1

  • Research type

    Research Study

  • Full title

    Cell-specific expression of a potential RNA gene therapy for Hepatocellular Carcinoma (HCC).

  • IRAS ID

    356139

  • Contact name

    Carin Ingemarsdotter

  • Contact email

    carin.ingemarsdotter@spliceor.com

  • Sponsor organisation

    Spliceor Ltd

  • Duration of Study in the UK

    2 years, 0 months, 0 days

  • Research summary

    We have developed a novel gene therapy approach for cancer that selectively targets malignant cells while sparing normal tissues, with the goal of improving both efficacy and safety for patients. Our preliminary studies in cell lines have demonstrated proof-of-concept. The next step is to test this strategy in more physiologically relevant models by using primary cancer cells obtained directly from patients alongside primary normal liver cells. In this way, we will assess whether the therapy can discriminate between malignant and healthy cells in a system that better reflects the complexity of cancer. This pilot study is designed to provide the foundation for future clinical translation, with the ultimate aim of advancing toward clinical trials and improved outcomes for patients.

  • REC name

    West Midlands - Coventry & Warwickshire Research Ethics Committee

  • REC reference

    25/WM/0231

  • Date of REC Opinion

    27 Oct 2025

  • REC opinion

    Favourable Opinion