Thelansis

  Kind Pharmaceutical, a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers, announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to AND017 for the treatment of Sickle Cell Disease (SCD). AND017 is being developed to treat various anemia indications associated with non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD). Publish Date: 25-10-2024   Source: Kind Pharmaceutical Sickle cell disease (SCD) results from homozygous and compound heterozygote inheritance of a mutation in the β-globin gene. This genetic anomaly leads to the formation of dense and rigid red blood cells (RBCs), particularly those containing HbS or HbS in combination with other abnormal β alleles. When exposed to a deoxygenated environment, these RBCs undergo polymerization, becoming ...

 Sickle cell disease (SCD) results from homozygous and compound heterozygote inheritance of a mutation in the β-globin gene. The dense rigid RBC’s lead to vaso-occlusion, tissue ischaemia, infarction as well as haemolysis. Red blood cells (RBCs) that contain HbS or HbS in combination with other abnormal β alleles, when exposed to deoxygenated environment, undergo polymerisation and become rigid. The rigid RBC’s are liable to haemolysis and due to increased density, may affect blood flow and endothelial vessel wall integrity. The dense rigid RBC’s lead to vaso-occlusion, tissue ischaemia, infarction as well as haemolysis. The consequence of haemolysis is a complex cascade of events, including nitric oxide consumption; haemolysis is linked nitric oxide dysregulation and endothelial dysfunction, which underlie complications such as leg ulceration, stroke, pulmonary hypertension and priapism. ·        The incidence of Sickle cell disease (SCD) ranges bet...