PMID- 37101079
OWN - NLM
STAT- MEDLINE
DCOM- 20230428
LR  - 20230522
IS  - 1550-7416 (Electronic)
IS  - 1550-7416 (Linking)
VI  - 25
IP  - 3
DP  - 2023 Apr 26
TI  - Evaluation of Cellular Immune Response to Adeno-Associated Virus-Based Gene 
      Therapy.
PG  - 47
LID - 10.1208/s12248-023-00814-5 [doi]
LID - 47
AB  - 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.
CI  - (c) 2023. The Author(s), under exclusive licence to American Association of 
      Pharmaceutical Scientists.
FAU - Gorovits, Boris
AU  - Gorovits B
AUID- ORCID: 0000-0001-9405-7718
AD  - Sana Biotechnology, Cambridge, Massachusetts, USA. boris.gorovits@sana.com.
FAU - Azadeh, Mitra
AU  - Azadeh M
AD  - Ultragenyx Pharmaceutical Inc, Novato, California, USA.
FAU - Buchlis, George
AU  - Buchlis G
AD  - University of Pennsylvania, Philadelphia, Pennsylvania, USA.
FAU - Fiscella, Michele
AU  - Fiscella M
AD  - Regenxbio Inc, Rockville, Maryland, USA.
FAU - Harrison, Travis
AU  - Harrison T
AD  - Precision for Medicine, Inc, Redwood City, California, USA.
FAU - Havert, Mike
AU  - Havert M
AD  - Gene Therapy Partners, San Diego, California, USA.
FAU - Janetzki, Sylvia
AU  - Janetzki S
AD  - ZellNet Consulting, Inc, Fort Lee, New Jersey, USA.
FAU - Jawa, Vibha
AU  - Jawa V
AD  - Bristol Myers Squibb Pharmaceutical, Princeton, New Jersey, USA.
FAU - Long, Brian
AU  - Long B
AD  - BioMarin Pharmaceutical Inc, Novato, California, USA.
FAU - Mahnke, Yolanda D
AU  - Mahnke YD
AD  - FlowKnowHow LLC, Brooklyn, New York, USA.
FAU - McDermott, Andrew
AU  - McDermott A
AD  - Labcorp Early Development Laboratories Inc, Indianapolis, Indiana, USA.
FAU - Milton, Mark
AU  - Milton M
AD  - Lake Boon Pharmaceutical Consulting LLC, Hudson, New York, USA.
FAU - Nelson, Robert
AU  - Nelson R
AD  - BioAgilytix Europe GmbH, Hamburg, Germany.
FAU - Vettermann, Christian
AU  - Vettermann C
AD  - BioMarin Pharmaceutical Inc, Novato, California, USA.
FAU - Wu, Bonnie
AU  - Wu B
AD  - Janssen Pharmaceuticals, Raritan, New Jersey, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20230426
PL  - United States
TA  - AAPS J
JT  - The AAPS journal
JID - 101223209
SB  - IM
MH  - *Dependovirus/genetics
MH  - *Genetic Therapy/methods
MH  - Immunity, Cellular
MH  - Genetic Vectors
PMC - PMC10132926
OTO - NOTNLM
OT  - AAV
OT  - Adeno-associated virus
OT  - Cellular immune response
OT  - anti-AAV immunogenicity
COIS- The authors are employed by and receive compensation from companies that are 
      involved in development of gene therapy modality therapeutics and are listed on 
      the title page of the manuscript. The authors have no other relevant affiliations 
      or financial involvements with any organization or entity with a financial 
      interest in or financial conflict with the subject matter or materials discussed 
      in the manuscript apart from those disclosed. No writing assistance was utilized 
      in the production of this manuscript.
EDAT- 2023/04/27 00:42
MHDA- 2023/04/28 06:42
PMCR- 2023/04/26
CRDT- 2023/04/26 23:32
PHST- 2023/02/09 00:00 [received]
PHST- 2023/04/14 00:00 [accepted]
PHST- 2023/04/28 06:42 [medline]
PHST- 2023/04/27 00:42 [pubmed]
PHST- 2023/04/26 23:32 [entrez]
PHST- 2023/04/26 00:00 [pmc-release]
AID - 10.1208/s12248-023-00814-5 [pii]
AID - 814 [pii]
AID - 10.1208/s12248-023-00814-5 [doi]
PST - epublish
SO  - AAPS J. 2023 Apr 26;25(3):47. doi: 10.1208/s12248-023-00814-5.