HOPE-B (Hemgenix / etranacogene)
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
AAV5 FIX-Padua gene therapy produces sustained Factor IX activity, reducing bleeds in severe hemophilia B.
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
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Sponsor
CSL Behring
Principal investigator
Steven Pipe, MD
Population
Hemophilia B; n=67
Primary endpoint
Annualized Bleeding Rate (ABR) for All Bleeding Episodes
Methodology notes
Full methodology is under curation. This project aggregates 3 seeded claims across three temporal layers. The replication spec, pre-registered analysis, and version history will appear here as the project matures.
Funding progress
$2.6M / $3.1M
85%
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Temporal layers
Published findings under validation
1
Active replication in progress
1
Forecast horizons pending resolution
1