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
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