Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements.

adeno-associated virus clinical decision limit companion diagnostic diagnostic assay strategy gene therapy in vitro diagnostic regulations patient selection preexisting antibodies reference limit risk-benefit assessment

Journal

Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857

Informations de publication

Date de publication:
14 Mar 2024
Historique:
medline: 18 3 2024
pubmed: 18 3 2024
entrez: 18 3 2024
Statut: epublish

Résumé

Recombinant adeno-associated virus (AAV) vectors are the leading delivery vehicle used for

Identifiants

pubmed: 38496304
doi: 10.1016/j.omtm.2024.101217
pii: S2329-0501(24)00033-0
pmc: PMC10944107
doi:

Types de publication

Journal Article Review

Langues

eng

Pagination

101217

Informations de copyright

© 2024 The Author(s).

Déclaration de conflit d'intérêts

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. The views and conclusion presented in this paper are those of the authors and do not necessarily reflect the representative affiliation or company’s position on the subject.

Auteurs

Manuela Braun (M)

Bayer AG, Pharmaceuticals R&D, 13342 Berlin, Germany.

Claudia Lange (C)

Bayer AG, Pharmaceuticals R&D, 13342 Berlin, Germany.

Philipp Schatz (P)

Bayer AG, Pharmaceuticals R&D, 13342 Berlin, Germany.

Brian Long (B)

BioMarin Pharmaceutical Inc, Novato, CA, USA.

Johannes Stanta (J)

Celerion Switzerland AG, 8320 Fehraltorf, Switzerland.

Boris Gorovits (B)

Sana Biotechnology, 100 Technology Square, Cambridge, MA 02139, USA.

Edit Tarcsa (E)

Abbvie Bioresearch Center, Worcester, MA 01605, USA.

Vibha Jawa (V)

Bristol Myers Squibb, Lawrence Township, NJ 08648, USA.

Tong-Yuan Yang (TY)

Janssen R&D LLC, Spring House, PA 19477, USA.

Wibke Lembke (W)

Integrated Biologix GmbH, 4051 Basel, Switzerland.

Nicole Miller (N)

Ultragenyx Pharmaceutical Inc, Novato, CA 94949, USA.

Fraser McBlane (F)

Novartis, Basel, Switzerland.

Louis Christodoulou (L)

UCB Pharma, 216 Bath Road, SL1 4EN Slough, UK.

Daisy Yuill (D)

AstraZeneca, 1 Francis Crick Avenue, CB2 0AA Cambridge, UK.

Mark Milton (M)

Lake Boon Pharmaceutical Consulting, LLC, Hudson, MA 01749, USA.

Classifications MeSH