Forecast horizon — calibration-scored at resolution.
By 2028, one-time AAV gene therapy for severe hemophilia B will show ≥5-year FIX expression durability in >70% of treated patients.
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Evidence stream
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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.
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By 2028, exa-cel or a comparable ex vivo gene-edited therapy will have treated >500 SCD patients globally with a durable VOC-free rate >90% at 3 years.
0.1640
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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By 2028, long-term follow-up will show durability of Zolgensma motor benefit plateau at ≥5 years post-infusion in early-treated cohorts.
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By 2028, at least two sickle cell gene-therapy products will compete head-to-head on real-world VOC-free survival and cost-effectiveness.
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Recent follow-up analyses of HOPE-B (Hemgenix / etranacogene) are confirming the original effect size in real-world data.
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By 2028, universal newborn SMA screening + early nusinersen (or onasemnogene/risdiplam) will be standard across all US states and most EU countries.