AT100-01: AAV8 NAb Seroprevalence in Late Onset Pompe Disease
Research type
Research Study
Full title
AT00-01: An AAV8 Neutralizing Antibody Seroprevalence Study in Subjects with Late Onset Pompe Disease
IRAS ID
283403
Contact name
Jorge Diaz-Manera
Contact email
Sponsor organisation
Audentes Therapeutics, Inc., An Astellas Company
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Research Summary
This study aims to increase the understanding of prevalence of neutralizing antibodies (NAbs) against adeno-associated virus serotype 8 (AAV8) in the Late Onset Pompe Disease patient population. A single blood draw, which will be tested for AAV8 NAbs, will be collected from each study participant.
Summary of Results
The primary purpose of this study was to understand the prevalence of pre-existing AAV8 antibodies against viral vectors. These viral vectors are being used to deliver targeted interventional treatments and pre-existing antibodies can limit the efficacy and safety of gene therapies. Five patients participated in the study at the site with a single blood draw for anti-AAV8 antibody testing. All five patient samples were negative for significant levels of AAV8 antibodies.
REC name
HSC REC A
REC reference
20/NI/0111
Date of REC Opinion
21 Sep 2020
REC opinion
Further Information Favourable Opinion