Gene therapy ‘viable’ long-term treatment for haemophilia A

An Australian and international study has shown gene therapy can help reduce bleeding episodes in haemophilia A patients for up to four years, despite previous studies suggesting otherwise. The phase 1–2 trial, funded by vector manufacturer Roche Group-member Spark Therapeutics, assessed adeno-associated viral (AAV) vector SPK-8011’s ability to support safe and durable hepatocyte expression of ...

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