Inherited Metabolic Disorders
Clinical research includes Crigler-Najjar syndrome and severe Crigler-Najjar syndrome in patients requiring phototherapy, with a focus on restoring bilirubin metabolism and addressing persistent hyperbilirubinemia.
- Crigler-Najjar syndrome
- Severe Crigler-Najjar syndrome
- Phototherapy-dependent disease
Work in this area centers on liver-directed gene therapy and long-term clinical assessment of metabolic correction.
Neuromuscular Disease
Research activity extends to Duchenne Muscular Dystrophy, with attention to muscle function, mobility preservation, and therapeutic strategies relevant to boys who remain ambulatory.
- Duchenne Muscular Dystrophy
- Microdystrophin-based therapy
- Ambulatory function
The therapeutic emphasis is on neuromuscular gene transfer and functional outcomes in progressive muscle degeneration.
Primary Immunodeficiency
The sponsor supports studies in Wiskott-Aldrich syndrome, focusing on immune reconstitution, hematopoietic stem cell correction, and durable control of immune dysfunction.
- Wiskott-Aldrich syndrome
- Autologous CD34+ cell therapy
- Long-term immune recovery
This area reflects interest in ex vivo gene-modified cell therapy for inherited disorders affecting platelets and adaptive immunity.
Chronic Granulomatous Disease
Clinical development also covers X-linked Chronic Granulomatous Disease, with a therapeutic focus on correcting phagocyte function and reducing vulnerability to recurrent infection.
- X-linked Chronic Granulomatous Disease
- Autologous lentiviral cell therapy
- Innate immune correction
These studies are directed toward immune system restoration in patients with inherited defects in oxidative burst activity.
