Article

Efficacy and safety of Factor IX Padua gene transfer via BBM-H901 among Chinese patients with haemophilia B: A multicentre, single-arm, phase 1/2 and 3 study and long-term follow-up of a phase 1 pilot study

2025-03-12

Abstract excerpt

<title>Abstract</title> <p> Building on promising phase 1 pilot results demonstrating safety and efficacy of BBM-H901 (AAV-mediated FIX Padua gene therapy) in restoring factor IX activity (FIX:C) among 10 patients with haemophilia B, we conducted current studies to verify its efficacy and safety in larger Chinese haemophilia B patients. A multicentre, single-arm, open-label study (phase 1/2: dose-escalation, n =...

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Literature Corpus work: 7954bcb6-7176-54ff-9e10-ccf2ae05b1d5
DOI: 10.21203/rs.3.rs-6138866/v1

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Efficacy and safety of Factor IX Padua gene transfer via BBM-H901 among Chinese patients with haemophilia B: A multicentre, single-arm, phase 1/2 and 3 study and long-term follow-up of a phase 1 pilot studyDOI 10.21203/rs.3.rs-6138866/v1Read

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