A phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials.

ABSTRACT:

Introduction

Haemophilia A is an X-linked bleeding disorder resulting from a deficiency of factor VIII (FVIII). To date, multiple gene therapies have entered clinical trials with the goal of providing durable haemostatic protection from a single dose. TAK 754 (BAX 888) is an investigational AAV8-based gene therapy containing a FVIII transgene. Reduction in CpG motifs was performed to reduce immunogenicity based on prior observations. Here, we describe the results of the first two cohorts treated with TAK 754.

Aim

To report clinical and translational results of the TAK-754 phase 1/2 AAV gene therapy study for the treatment of haemophilia A.

Methods

A phase 1/2 single arm open-label dose escalation study of TAK-754 was performed in participants with severe haemophilia A

SUBMITTER: Chapin J 

PROVIDER: S-EPMC11780198 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature