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Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments

Year: 2025

Type: article

Abstract: study duration, emphasizing that shorter-term studies are generally sufficient for identifying relevant toxicities associated with AAV-based gene therapies. Embracing this approach can reduce animal use, accelerate development timelines, and support harmonized regulatory expectations for AAV gene therapy products.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2025.101628

Authors: Basel T. Assaf, David Edwards, Shawn Jay Berens, Brian Lauritzen, Christelle Pierrot, Francis Fonyuy Tukov, Cristina Bertinetti-Lapatki, Manuela Braun, Kai Schaefer, Jessica L. Lynch

Institutions: UCB Pharma (United Kingdom), Eli Lilly (United States), Novo Nordisk (Denmark), Astellas Pharma (Netherlands), Novartis (United States), Roche (Switzerland), Bayer (Germany), AbbVie (Germany)

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: gold

Estimated APC paid: $3,900

OpenAlex ID: W4416118987

FWCI: 1.656

Cites: 12

Cited by: 3

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