Organization Overview
Alternative names
autologous hematopoietic stem cells genetically modified with globe lentiviral vector encoding for the human beta-globin gene (1 trial)
beta-Thalassemia (Phase 2)
Blood Platelet Disorders (Phase 2)
Cystic Fibrosis (Phase 3)
Fibrosis (Phase 3)
Granulomatous Disease, Chronic (Phase 2)
Immunologic Deficiency Syndromes (Phase 2)
Leukodystrophy, Metachromatic (Phase 2)
Lysosomal Storage Diseases (Phase 2)
Mucopolysaccharidoses (Phase 2)
Mucopolysaccharidosis I (Phase 2)
Mucopolysaccharidosis VI (Phase 2)
Osteoporosis (Phase 3)
Severe Combined Immunodeficiency (Phase 2)
Thalassemia (Phase 2)
Thrombocytopenia (Phase 2)