ArticleSignal transduction and targeted therapy2026
Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity.
Article in Signal transduction and targeted therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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Who cites it
3 citing papers in PubMed.
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.Molecular therapy. Nucleic acids · 2026Review
- Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.Nature medicine · 2026Article
- The therapeutic landscape of inherited bleeding disorders in China.Research and practice in thrombosis and haemostasis · 2026Review
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Authors and funding
17 authors.
Funding
Abstract
This prospective phase 1 pilot study evaluated the safety and efficacy of GS001, an adeno-associated virus serotype 8 (AAV8)-based vector encoding B-domain-deleted factor VIII with exploratory assessment of prophylactic immunosuppression. Twelve adult severe hemophilia A participants were enrolled. Participants received GS001 at either 2×10¹² (low dose cohort) or 4×10¹² (high dose cohort) vg/kg following prophylactic treatment with prednisone alone or prednisone plus tacrolimus starting from one week before GS001 infusion. Median (range) follow-up was 156.0 (144.0-208.0) / 110.5 (104.0-130.0) weeks (2×10¹² / 4×10¹² vg/kg). Transgene expression peaked at 5-7 weeks post-infusion. In low dose cohort, at week 144.0, median (range) factor VIII activity was 5.6 (0.5-52.0) IU/dL (n = 6). In high dose cohort, at week 104.0, median (range) factor VIII activity was 42.7 (29.4-92.1) IU/dL (n = 6) and all six participants sustained factor VIII levels above 29 IU/dL. Both cohorts showed significant reductions in annualized bleeding rate and factor VIII consumption with grade 1-2 adverse events. Alanine aminotransferase elevations occurred in 3/6 and 5/6 low/high-dose participants and most were grade 1. The Single-cell RNA analysis demonstrated that the prednisone plus tacrolimus pretreatment effectively inhibited CD8
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