Halfway through the year 2023, BioMarin bagged the approval for an “adeno-associated virus serotype 5 (AAV5)” gene therapy by the United States Food and Drug Administration (FDA). The therapy is for adults with a life-threatening and rare genetic condition known as Haemophilia A. In this article we will discuss about the novel therapy and the studies leading to its approval. Haemophilia A, also referred to as an X-linked disorder affects 1 in 5,000 male births. The sex-linke