Evaluation of Cellular Immune Response to Adeno-Associated Virus-Based Gene Therapy.
AAV
Adeno-associated virus
Cellular immune response
anti-AAV immunogenicity
Journal
The AAPS journal
ISSN: 1550-7416
Titre abrégé: AAPS J
Pays: United States
ID NLM: 101223209
Informations de publication
Date de publication:
26 04 2023
26 04 2023
Historique:
received:
09
02
2023
accepted:
14
04
2023
medline:
28
4
2023
pubmed:
27
4
2023
entrez:
26
4
2023
Statut:
epublish
Résumé
The number of approved or investigational late phase viral vector gene therapies (GTx) has been rapidly growing. The adeno-associated virus vector (AAV) technology continues to be the most used GTx platform of choice. The presence of pre-existing anti-AAV immunity has been firmly established and is broadly viewed as a potential deterrent for successful AAV transduction with a possibility of negative impact on clinical efficacy and a connection to adverse events. Recommendations for the evaluation of humoral, including neutralizing and total antibody based, anti-AAV immune response have been presented elsewhere. This manuscript aims to cover considerations related to the assessment of anti-AAV cellular immune response, including review of correlations between humoral and cellular responses, potential value of cellular immunogenicity assessment, and commonly used analytical methodologies and parameters critical for monitoring assay performance. This manuscript was authored by a group of scientists involved in GTx development who represent several pharma and contract research organizations. It is our intent to provide recommendations and guidance to the industry sponsors, academic laboratories, and regulatory agencies working on AAV-based GTx viral vector modalities with the goal of achieving a more consistent approach to anti-AAV cellular immune response assessment.
Identifiants
pubmed: 37101079
doi: 10.1208/s12248-023-00814-5
pii: 10.1208/s12248-023-00814-5
pmc: PMC10132926
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
47Informations de copyright
© 2023. The Author(s), under exclusive licence to American Association of Pharmaceutical Scientists.
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