2. Disease Areas
  3. Blood or Cardio-cerebrovascular Disease
  4. Blood Disease
  5. Sickle Cell Disease

Sickle Cell Disease

Sickle cell disease (SCD) is a group of inherited blood disorders characterized by the production of abnormal hemoglobin (Hb S), resulting in rigid, sickle-shaped red blood cells that cause chronic hemolytic anemia, vasoocclusive crises, and progressive organ damage. The disease arises from a mutation in the β-globin gene, most commonly as homozygous Hb S (sickle cell anemia), but can also occur in compound heterozygotes with Hb C or β-thalassemia. These deformed cells obstruct small blood vessels, impairing oxygen delivery and leading to recurrent pain episodes, increased risk of infections, stroke, pulmonary hypertension, acute chest syndrome, splenic sequestration, and delayed growth. Chronic hemolysis contributes to anemia, jaundice, and complications such as leg ulcers and elevated risk of vascular events. Symptoms typically manifest in early childhood, and while management focuses on symptom relief and prevention of complications, curative options such as hematopoietic stem cell transplantation and gene therapy are emerging.

Sickle Cell Disease (6):

Cat. No. Product Name CAS No. Purity Chemical Structure
  • HY-123606
    GSK484 1652629-23-6 99.14%
    GSK484 is a PAD4 inhibitor that effectively inhibits protein citrullination and the formation of neutrophil extracellular traps (NETs) by blocking the catalytic activity of PAD4. GSK484 suppresses the production of histone H3, MHC-I expression, CD8+ T cell activation, proliferation and inflammatory cytokine release. GSK484 reduces inflammation and bone destruction in collagen-induced rheumatoid arthritis, alleviates pain and mast cell activation in sickle cell disease, and improves myocardial ischemia-reperfusion injury and experimental colitis. In addition, GSK484 restores intestinal microbial homeostasis by reversing ferroptosis-induced dysbiosis. GSK484 can be used to study the disease mechanisms of rheumatoid arthritis, sickle cell disease, thrombosis, myocardial injury, colitis and other conditions.
    GSK484
  • HY-167874
    ASP-8731 2488255-42-9 98.42%
    ASP-8731 is an orally active BACH1 inhibitor. ASP-8731 activates antioxidant, anti-inflammatory and globin gene pathways by relieving the inhibitory effect of BACH1 on NRF2-mediated gene transcription. ASP-8731 significantly upregulates the expression of HMOX1, FTH1 and various globins (such as HGB, HBG, HBA), increases fetal hemoglobin (HbF) levels, and effectively induces F-cell production in hydroxyurea-unresponsive cells. Meanwhile, ASP-8731 reduces inflammatory responses and white blood cell counts by downregulating VCAM1, ICAM-1 and the phosphorylation level of NF-κB(p65), and blocks heme-induced glutathione depletion and microcirculatory stasis. ASP-8731 holds potential for inhibiting sickle cell disease and related hematological disorders.
    ASP-8731
  • HY-159098
    dWIZ-1
    dWIZ-1 is an orally active molecular glue and chemical probe targeting the WIZ transcription factor, which based on an IMiD backbone, binding to human WIZ with an affinity of 3.5 μM. dWIZ-1 recruits WIZ to the cereblon-DDB1 complex via its ZF7 domain, thereby triggering proteasome-dependent degradation of WIZ. dWIZ-1 significantly induces fetal hemoglobin expression in erythroblasts while reducing the level of inhibitory H3K9 dimethylation at WIZ binding sites such as the β-globin locus. Meanwhile, dWIZ-1 does not affect the proliferation and differentiation of erythroblasts, and no cytotoxicity is observed in in vitro cells or cynomolgus monkey models. dWIZ-1 serves as a critical tool molecule for investigating the mechanism and underlying pathways of sickle cell disease.
    dWIZ-1
  • HY-D2902
    SNOTRAP 2080332-70-1 99.02%
    SNOTRAP (S-nitrosylation trapping by triaryl phosphine) is a chemical probe for S-nitrosylated (SNO) proteins. SNOTRAP based proteomic pipeline can identify 1181 SNO proteins (1714 SNO sites) in the brain of mouse models. SNOTRAP can be used for nitrosative stress related diseases such as neurodegenerative disease, endotoxic shock, cancer, multiple sclerosis, and sickle cell disease.
    SNOTRAP
  • HY-12897
    (R)-FTY720-OMe 1382486-90-9
    (R)-FTY720-OMe is a selective SphK2 inhibitor with an IC50 of 16.5  μM. (R)-FTY720-OMe can be used for cancer, fibrosis, and Alzheimer’s and sickle cell diseases research.
    (R)-FTY720-OMe
  • HY-125475
    Rivipansel 927881-99-0
    Rivipansel is a small-molecule glycomimetic pan-selectin antagonist with inhibitory activity against E-selectin and P-selectin. Rivipansel binds tightly to the lectin domain of E-selectin, and selectively blocks the recognition of CD62L by E-selectin without affecting the binding of PSGL-1 to E-selectin. Rivipansel functionally inhibits the adhesion of hematopoietic cells to endothelial cells, and is applicable to research related to sickle cell disease.
    Rivipansel