Clinical research study for haemophilia B
BEYOND-9 aims to see if an investigational gene therapy may be effective and well-tolerated in males with haemophilia B.
Living with haemophilia B? You’re not alone.
This website provides information about a clinical study, BEYOND-9, for males with haemophilia B. Haemophilia B is a rare genetic disorder characterised by abnormally low levels of functional Factor IX (Factor 9) protein (FIX), which is necessary for normal blood clotting. The FIX protein is produced by liver cells and secreted in the blood. FIX is expressed by a gene located on the X chromosome. As males have only one X chromosome and no second copy of the gene, severe haemophilia more often affects males.
About 4 in every 100,000 males have haemophilia B according to the World Federation of Hemophilia (WFH). Clinical symptoms of moderate and severe haemophilia B include spontaneous bleeding inside the body, pain and heavy bleeding after a cut or injury.
Clinical studies are essential to investigating potential treatments and are not possible without the people who take part. Everyone involved in this clinical study plays an important role in increasing our understanding of haemophilia B, which may help others with this condition in the future.
Living with haemophilia B may be challenging, and you may feel that you have enough to do without committing to a clinical study. However, through participation in BEYOND-9, you may play a key role in helping to develop an investigational CRISPR-based targeted gene insertion therapy that may be effective and well-tolerated in people with haemophilia B, including adults and children.
Every treatment that exists today is available because of study participants who actively took part in research.
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