Published finding — does the expert body still believe it?
The HOPE-B (Hemgenix / etranacogene) trial's stated primary conclusion — AAV5 FIX-Padua gene therapy produces sustained Factor IX activity, reducing bleeds in severe hemophilia B. — replicates in independent cohorts.
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Evidence stream
1 event · 0 snapshots
Registry data
Apr 18, 2026
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Source publication
Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B
Semantically related
Nearest claims in the expert-corpus vector space. Ordered by cosine distance — lower is closer.
0.1069
Recent follow-up analyses of HOPE-B (Hemgenix / etranacogene) are confirming the original effect size in real-world data.
0.1640
By 2028, one-time AAV gene therapy for severe hemophilia B will show ≥5-year FIX expression durability in >70% of treated patients.
0.1711
The HGB-206 (lovo-cel) trial's stated primary conclusion — Lentiviral β-globin gene therapy reduces or eliminates VOC events in severe sickle cell disease. — replicates in independent cohorts.
0.1923
The LUXTURNA (voretigene) trial's stated primary conclusion — Subretinal AAV2-hRPE65 gene therapy improves functional vision in biallelic RPE65-associated inherited retinal dystrophy. — replicates in independent cohorts.
0.2066
The CLIMB-121 (exa-cel, sickle cell) trial's stated primary conclusion — CRISPR-Cas9 BCL11A editing (exa-cel) eliminates vaso-occlusive crises in severe sickle cell disease over multi-year follow-up. — replicates in independent cohorts.
0.2079
Recent follow-up analyses of STR1VE (onasemnogene abeparvovec / Zolgensma) are confirming the original effect size in real-world data.