For decades, many people living with hemophilia followed a regular routine that required infusing their therapy multiple times per week, sometimes even daily.
Hemophilia is a rare, genetic disease that results from the body being unable to create enough of one of two main blood factors that cause clotting in order to stop bleeding. There are two main forms of the condition: Hemophilia A, which results from too little Factor VIII activity, and Hemophilia B, which is caused by low levels of Factor IX activity.
The standard-of-care treatment for people with hemophilia is an infusion of clotting factor, a protein their bodies don’t make naturally that help their blood to clot. Without those factors, patients are at a greatly increased risk of serious health problems from spontaneous and prolonged bleeding, often into joints and soft tissue, like muscles. However, conventional factor therapy only stays in the body for a short period of time. The factor’s “half-life” determines how frequently a person with hemophilia must infuse. The shorter the half-life, the more often a patient will have to infuse their factor.
In the past five years, the advent of therapies, such as extended half-life therapies, have made great strides in reducing how often a patient needs an infusion– to twice a week or even longer intervals between dosing, depending on the type of factor they need.
Researchers at Sanofi are working not only on treatments that could extend time between infusions further, but also on entirely new potential treatment approaches, including one early investigational gene therapy program where patients may need to be treated just once.
“There is a lot of good news for the hemophilia community,” said Rob Peters, Head of Rare Blood Disorders Research at Sanofi, who has been researching hemophilia treatments for more than 15 years. “At Sanofi, we have a really deep understanding of hematology and a long legacy of working with the hemophilia community, which has enabled us to design new potential treatments that may overcome the limitations of existing therapies.”