Therapeutic Focus
Glanzmann Thrombasthenia
GT
Glanzmann Thrombasthenia (GT) is an inherited bleeding disorder where platelets fail to properly aggregate. Patients experience frequent, disruptive, and potentially life-threatening bleeds. Scientific literature notes nosebleeds as common, while patient experiences reveal these often last days or weeks despite treatment, causing anemia and irreversible damage that may require surgery.
Von Willebrand Disease
VWD
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder. Yet, it’s often overlooked—especially in women and those whose symptoms aren’t considered severe enough to receive specialized care. VWD is caused by lack of an essential protein called von Willebrand factor (vWF). This deficiency in vWF results in recurrent bleeding events.
FVII Deficiency
FVIID
Factor VII Deficiency (FVIID) is a rare inherited bleeding disorder caused by low levels of factor VII, a protein essential for initiating blood clotting. Without it, clot formation is delayed, leading to unpredictable and sometimes life-threatening bleeding. People with FVIID may experience frequent nosebleeds, easy bruising, prolonged bleeding after injury, and, in severe cases, joint, gastrointestinal, or intracranial hemorrhage.
Sutacimig for Glanzmann Thrombasthenia (NCT06211634)
The goal of this Phase 1/2 study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of sutacimig in participants with Glanzmann Thrombasthenia. The study has three parts to allow for a methodological progression from safety and tolerability to establish the dose level(s) and dosing interval(s) for efficacy.
VELORA Discover Screening Study for Von Willebrand Disease (NCT06610201)
VELORA Discover is a screening study of bleeding and treatment in participants with Von Willebrand Disease (VWD). The purpose of this study is to understand bleeds by gathering data on frequency, severity and treatment for bleeding events, assess patients’ quality of life and establish baseline data to create a foundation for bleeding and treatment rates.
VELORA Pioneer— HMB-002 for Von Willebrand Disease (NCT06754852)
VELORA Pioneer is a Phase 1/2 study to evaluate the impact of HMB-002 in participants with Von Willebrand Disease (VWD). HMB-002 is being developed as a prophylactic treatment to reduce the number and severity of bleeding events in people with VWD using a convenient subcutaneous treatment regimen.