Therapeutic Focus
Glanzmann Thrombasthenia
GT
Glanzmann Thrombasthenia (GT) is an inherited rare bleeding disorder where blood doesn’t clot properly because platelets don’t stick together as they should. Bleeds in GT are frequent, disruptive, and sometimes life-threatening.
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.
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.