Active replication — ongoing trial, registry, or meta-analysis.
Recent follow-up analyses of HOPE-B (Hemgenix / etranacogene) are confirming the original effect size in real-world data.
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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
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.
0.1170
Recent follow-up analyses of STR1VE (onasemnogene abeparvovec / Zolgensma) are confirming the original effect size in real-world data.
0.1185
Recent follow-up analyses of HGB-206 (lovo-cel) are confirming the original effect size in real-world data.
0.1194
Recent follow-up analyses of LUXTURNA (voretigene) are confirming the original effect size in real-world data.
0.1229
Recent follow-up analyses of CLIMB-121 (exa-cel, sickle cell) are confirming the original effect size in real-world data.
0.1436
Recent follow-up analyses of NURTURE (nusinersen) are confirming the original effect size in real-world data.