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

PI Steven Pipe, MD

Primary endpoint

Annualized Bleeding Rate (ABR) for All Bleeding Episodes

Population

Hemophilia B; n=67

Historical

Current

Future

3 noemes

Historical

1 noeme

Published findings from this trial. Vote whether the expert body still believes each noeme stands given current evidence.

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.

gene-therapy

Current

1 noeme

Noemes tracking active replication, subgroup analysis, or long-term follow-up. Vote on the likelihood the original effect holds.

Recent follow-up analyses of HOPE-B (Hemgenix / etranacogene) are confirming the original effect size in real-world data.

gene-therapy

Future

1 noeme

Open forecast horizons tied to this trial. Vote your probability that each resolves TRUE by the declared horizon.

By 2028, one-time AAV gene therapy for severe hemophilia B will show ≥5-year FIX expression durability in >70% of treated patients.

gene-therapy

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