Gene Therapy

First Haemophilia Gene Therapy Licensed by EMA

First Haemophilia Gene Therapy licensed by European Medicines Agency On Wednesday of this week, August 24th, it was announced that the European Medicines Agency ( EMA) have granted a conditional marketing authorisation to the company Biomarin for their FVIII Gene Therapy Valoctocogene Roxaparvovec (brand name: Roctavian). A conditional marketing authorisation (CMA) recognises that the medicine …

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First person with haemophilia in Ireland treated with gene therapy

Uncategorised / By barry

The Irish Haemophilia Society has today announced that gene therapy has been used for the first time to treat a person with haemophilia in Ireland. The recipient has received gene therapy as part of a Phase 3 clinical trial. The therapy uses an adeno-associated viral vector to deliver the Factor IX gene therapy intravenously to …

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