Asphalion experts Jon Giblin and Christopher Mann, together with experts from the ARDAT Consortium, have just published an article on Molecular Therapy.
“Currently, most clinical trials for adeno-associated virus (AAV)-based gene therapy (GT) exclude patients with pre-existing anti-AAV antibodies (AAV Abs) due to potential impact on treatment efficacy and safety.
This may prevent some patients from accessing potentially beneficial treatments. We advocate for more flexible validation approaches based on current understanding of antibody characteristics and the commonly used assay formats. We propose an AAV serotype-specific in vitro diagnostic (IVD) assay, an ‘AAV Serotype IVD,’ with pragmatic analytical validations for single AAV serotypes.
These measures could expedite AAV GT development while expanding access for patients with elevated pre-existing AAV Ab levels.”
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