What is Hemophilia?
Hemophilia is a genetic disorder caused by a mutation on the X chromosome resulting in deficiency of certain blood clotting factors. The deficiency in these clotting factors leads to recurrent prolonged bleeding episodes. Hemophilia affects people from all races and ethnic groups. People with hemophilia B have insufficient levels of blood clotting factor IX (FIX). The estimated global prevalence is 3.8 cases per 100,000 males for all severities of hemophilia B.
How is Hemophilia B Treated?
Currently, there is no cure for hemophilia B. Standard of care for hemophilia B is regular long-term prophylaxis treatment with intravenous (IV) administration of FIX replacement to raise circulating FIX activity. The frequency of FIX replacement infusions can vary from daily to weekly, depending on individual needs and type of factor replacement. A gene therapy with adeno-associated virus (AAV) has recently been approved in the US and EU for adults only.
Despite the major advances in the treatment options, there remains an unmet need for people with hemophilia B. The continued need for repeated and frequent infusions, the lack of consistent, predictable bleed protection and the potential risk of bleeding events and long-term consequences, such as significant pain from irreversible joint damage, point to the unmet need for optimal hemophilia B management and an acceptable quality of life.
Shifting the Paradigm of Today’s Protein-Based Therapies to Create Freedom for People Living with Hemophilia B
At Be Bio, our goal is to create a new therapeutic option for people with hemophilia B which provides the freedom to live a normal life, free of the limitations due to hemophilia B. We are pioneering a new class of engineered B Cell Medicines (BCMs) designed with precision engineering to deliver continuous expression of proteins that address important limitations of existing therapies. Our lead program, BE-101, starts with the person’s own B cells which are then modified to produce predictable, persistent FIX activity levels to prevent bleeding and joint damage. Additionally, the medicine has the potential to be redosable if necessary. This program is advancing toward a clinical trial for the treatment of people with moderately severe to severe hemophilia B.