B-AMAZE immunology study

  • Research type

    Research Study

  • Full title

    B-AMAZE immunology study - plasma proteome

  • IRAS ID

    306976

  • Contact name

    Rose Sheridan

  • Contact email

    rose.sheridan@freeline.life

  • Sponsor organisation

    Freeline Therapeutics

  • Clinicaltrials.gov Identifier

    N/A, N/A

  • Duration of Study in the UK

    0 years, 5 months, 30 days

  • Research summary

    Owing to their non-pathogenic nature and outstanding safety record, adeno-associated virus (AAV) vectors have become the gene therapy vector of choice. However, immune response against AAV is observed in clinical trials, which leads to loss of transgene expression and treatment efficacy. Regular assessment of patients and prophylactic or reactive treatment with immunosuppressive drugs to suppress the immune response are required to prevent loss of treatment efficacy. Understanding the mechanisms underlying the deleterious immune response leading to loss of transgene expression is of utmost importance to support the management of the immune response in the clinic and to create targeted solutions to eliminate the risk of losing treatment efficacy.
    In the proposed study, we will profile the immune response and its correlation to transgene expression in patients who received AAV gene therapy. We hope to identify potential biomarkers that can predict the risk of loss of transgene expression, to progress our understanding of the immune response to AAV gene therapy and to inform on potential mechanisms of immune-mediated loss of transgene expression.

  • REC name

    Wales REC 6

  • REC reference

    22/WA/0115

  • Date of REC Opinion

    19 Apr 2022

  • REC opinion

    Favourable Opinion