Haemophilia B and Gene Therapy
Research activity includes haemophilia B and Hemophilia B, with a strong emphasis on AAV-based gene therapy, factor IX expression, and long-term control of bleeding risk in adolescent and adult populations.
- Factor IX replacement
- Bleeding prevention
- AAV vector therapy
- Long-term safety follow-up
Clinical interest also extends to neutralizing antibodies and sustained treatment outcomes after gene-based intervention.
Hereditary Angioedema
The sponsor funds studies in hereditary angioedema, Hereditary Angioedema (HAE), and Hereditary angioedema (HAE), focusing on prophylaxis, attack prevention, and treatment switching across adult and pediatric groups.
- C1-inhibitor deficiency
- Angioedema prophylaxis
- Attack prevention
- Pediatric HAE care
Research includes both subcutaneous and intravenous approaches for controlling recurrent swelling episodes.
Sickle Cell Disease and Vaso-occlusive Crisis
Trials in sickle cell disease, Sickle Cell Disease, and sickle cell disease vaso-occlusive crisis examine symptom relief, crisis resolution, and safety in affected adults and adolescents.
- Vaso-occlusive crisis
- Hemolytic complications
- Acute symptom management
- Supportive biologic therapy
Additional work addresses anemia-related complications and disease burden during acute episodes.
Immune-Mediated and Inflammatory Disorders
The portfolio includes acute graft-versus-host disease, dermatomyositis, and related immune-mediated inflammatory disorders, reflecting interest in transplant-associated and systemic autoimmune conditions.
- Graft-versus-host disease
- Dermatomyositis
- Hematopoietic cell transplant
- Immune modulation
These studies focus on disease control in settings where immune dysregulation drives tissue injury and persistent symptoms.
Renal, Pulmonary, and Metabolic Conditions
Research also covers atherosclerotic cardiovascular disease in patients with ESKD, non-cystic fibrosis bronchiectasis, homeostatic iron regulator gene-related hereditary hemochromatosis, and post-COVID-19 postural orthostatic tachycardia syndrome.
- End-stage kidney disease
- Bronchiectasis
- Hereditary hemochromatosis
- Post-COVID dysautonomia
These areas include interest in cardiovascular inflammation, iron overload, and chronic respiratory or autonomic complications.
