Work

AAV serotype IVDs for patient selection in gene therapy development: Technical and regulatory considerations by ARDAT consortium

Year: 2026

Type: review

Abstract: diagnostic (IVD) assay, an "AAV serotype IVD," with pragmatic analytical validations for single AAV serotypes. This approach would allow sponsors to establish clinically meaningful cutoffs through adaptive clinical trials that progressively include patients with escalating pre-existing AAV Ab levels by implementing multiple cutoffs during clinical development. These measures could expedite AAV GT development while expanding access for patients with elevated pre-existing AAV Ab levels.

Source: Molecular Therapy Advances

DOI: 10.1016/j.omta.2026.201862

Authors: Manuela Braun, Christel Ravesteijn-Verrijt, Philipp Schatz, Yanmei Lu, Sarra Seninet, Jon Giblin, John Millholland, Gurunadh Chichili, Fraser McBlane, Johannes Stanta, Mimoun Azzouz, Christopher Mann

Institutions: Bayer (Germany), Astellas Pharma (Netherlands), Sangamo BioSciences (United States), GenoSafe, Bristol-Myers Squibb (United States), Astellas Pharma (United States), Novartis (Switzerland), Celerion (United Kingdom), University of Sheffield

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Funders: Pfizer, European Federation of Pharmaceutical Industries and Associations, Innovative Health Initiative, European Commission

OpenAlex ID: W7220905784

FWCI:

Cites: 21

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Related to: 10