ArticleMolecular therapy. Methods & clinical development2025
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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Who cites it
4 citing papers in PubMed.
- Hemophilia B gene therapy: a NAb-solute barrier overcome by adeno-associated virus serotype 5.Research and practice in thrombosis and haemostasis · 2026Article
- Etranacogene dezaparvovec in people with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: a 4-year subgroup analysis of the Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B) trial.Research and practice in thrombosis and haemostasis · 2026Article
- Etranacogene dezaparvovec in people with hemophilia B with preexisting adeno-associated virus 5 neutralizing antibodies: 4-year subgroup results from the HOPE-B trial.Research and practice in thrombosis and haemostasis · 2026Article
- Global seroprevalence of neutralizing and total antibodies against AAV vectors and their impact on the clinical gene therapy landscape: a scoping review.Frontiers in medicine · 2026Review
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13 authors.
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Abstract
Testing for binding or neutralizing antibodies (NAbs) to adeno-associated virus (AAV) is part of the laboratory assessment of people with hemophilia considering AAV-based gene therapy. We evaluated the natural history of NAb titers to AAV serotype 5 (AAV5) in adult males ≥18 years old with hemophilia B (factor IX ≤ 2%) during the lead-in period of a phase 3 trial prior to the infusion of etranacogene dezaparvovec to characterize NAb in addition to immunoglobulin G (IgG) and immunoglobulin M (IgM) anti-AAV5 binding antibody changes over time. At screening, 48% (32/67) of enrolled participants had detectable NAbs (NAb+) with a median titer of 58 (range: 9-3,440). Participant-specific lead-in periods differed and included discontinuers (median duration: 240 days; range: 1-360). The median intra-participant coefficient of variation of NAb titer over time was 25% (range: 2%-154%). NAb seropositivity was associated with older age (
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