sickle

Voxelotor (GBT 440) is a novel hemoglobin S (HbS) polymerization inhibitor primarily used for the treatment of sickle cell disease (SCD) and a cytochrome P450 3A4 inhibitor. Voxelotor targets and covalently binds to the N-terminal valine of the HbS alpha chain, thereby stabilizing sickle cell hemoglobin (HbS).

RN-1 dihydrochloride is an effective and selective irreversible inhibitor of lysine-specific demethylase 1 (LSD1, IC50 = 70 nM).

PF-04447943 (Edelinontrine) is a potent and selective phosphodiesterase 9A inhibitor with an IC50 of 12 nM, which is 78-fold more selective than that used for other PDE family members (IC50>1000 nM).PF-04447943 exhibits anti-inflammatory activity, attenuates inflammatory responses by inhibiting oxidative stress, inflammation, and modulating T-cell polarization, and may be useful for research on sickle cell anemia.

Velaresol (BW 12C79) is used for the study of tumors, sickle cell anemia and digestive disorders.

MS8511, a selective covalent irreversible inhibitor of G9a/GLP, targets a cysteine residue at the substrate binding site, displaying IC50 values of 100 nM (G9a) and 140 nM (GLP), alongside Kd values of 44 nM (G9a) and 46 nM (GLP). This compound effectively reduces cellular H3K9me2 levels and boosts antiproliferation activity, making it applicable in cancer research involving brain, breast, ovarian, lung, bladder, melanoma, and colorectal cancers, as well as studies on Alzheimer's disease (AD), sickle cell disease, and Prader−Willi syndrome (PWS) [1].

(Rac)-Etavopivat ((Rac)-FT-4202), an isomer of Etavopivat, is an orally active erythrocyte pyruvate kinase-R (PKR) activator used in sickle cell disease and haemoglobinopathy research [1].

Glutathione oxidized (L(-) -glutathione) is the oxidized form of Glutathione, which is a major antioxidant and antidote in cells. Glutathione oxidized can be used to study sickle cells and red blood cells.

D-fructose-1,6-diphosphate (D-fructose-1,6-diphosphate), also known as Fosfructose, is a cytoprotective natural sugar phosphate. It functions by promoting anaerobic glycolysis, which generates adenosine triphosphate (ATP) under ischemic conditions. This compound holds potential for research in cardiovascular ischemia, sickle cell anemia, and asthma.

Esafosfan trisodium (FDP trisodium) is a cytoprotective natural sugar phosphate for the potential treatment of sickle cell anemia, cardiovascular ischemia and asthma. It acts by stimulating anaerobic glycolysis which generates adenosine triphosphate under ischemic conditions.

Ferroportin-IN-1 is an inhibitor of the iron transport protein Ferroportin, which regulates cellular iron efflux and metabolism to study diseases caused by iron-modulating deficiencies or disorders of iron metabolism, used in metabolism studies such as thalassemia, sickle cell disease, and hemochromatosis.

Iso-SLR 080811 is an isomer of SLR 080811, suitable for relevant research in the life sciences. SLR080811 is a selective SphK2 inhibitor (Ki=1.3 μM) that elevates blood S1P levels in wild-type mice, reduces S1P levels in sphk1 knockout mice, and leaves S1P levels unchanged in SphK2 knockout mice.

dWIZ-1 ((rac)-dWIZ-1) is a potent WIZ molecular gel degrader that recruits WIZ (ZF7) to the cereblon thereby inducing degradation and thus induction of haemoglobin fetalis (HbF) in erythroblasts, which can be used to study sickle cell disease (SCD).

BMS-986470 is a CRBN E3 ubiquitin ligase modulator (CELMoD) that activates fetal hemoglobin (HbF). It is an orally available molecular glue degradator targeting ZBTB7A and WIZ protein degradation, thereby inducing gamma globin synthesis and activating HbF. It is applicable for studying sickle cell disease (SCD).

ASP8731 is a selective inhibitor of BACH1 that prevents inflammation and vascular occlusion, and induces fetal hemoglobin in sickle cell disease.

MS8511 hydrochloride is a selective G9a/GLP inhibitor with IC50 values of 100 nM and 140 nM respectively, exhibiting covalent and irreversible characteristics. MS8511 reduces intracellular H3K9me2 levels and enhances anti-proliferative activity, making it suitable for investigating multiple cancers.

Varespladib (LY315920), a specific and effective human non-pancreatic secretory phospholipase A2 (hnsPLA) inhibitor(IC50=7 nM), has been investigated for the treatment and prevention of sickle cell disease, vaso-occlusive crisis, and acute coronary syndrome.

WIZ degrader 8 (compound 10) is a potent and selective molecular glue degrader of the transcription factor WIZ, effectively inducing HbF expression. It promotes WIZ degradation and HbF induction, suggesting its potential use as an inhibitor for sickle cell disease.

Methoxyurea (Compound 3) is a potential modulator of nitric oxide (NO) donors, interacting with hemoglobin forms such as oxyhemoglobin (OxyHb) and methemoglobin (MetHb). It holds promise for use in research on sickle cell disease (SCD).

G9a-IN-3 (compound 16g) is a potent G9a inhibitor with an IC50 of 0.002 μM. It is applicable for research in sickle cell disease.

Enprofylline (Enprofilina), a bronchodilator, acts primarily as a competitive nonselective phosphodiesterase inhibitor. Enprofylline is used in asthma, chronic obstructive pulmonary disease, and in the management of sickle cell disease, diabetic neuropathy, and cerebrovascular insufficiency.

WIZ degrader 8 (TFA) is the trifluoroacetic acid salt of WIZ degrader 8. WIZ degrader 8 (compound 10) is a potent and selective molecular glue degrader of the transcription factor WIZ, effectively inducing HbF expression. It leads to WIZ degradation and HbF expression, showing potential as a sickle cell disease inhibitor.

Rivipansel is a glycomimetic compound. It acts as a pan-selectin antagonist and reverses acute vascular occlusions in sickle cell mice.

N-Phenylacetylphenylalanine is a biaromatic agent with properties that make them suitable candidates for the treatment of sickle cell disease.

Senicapoc (ICA-17043) is a potent inhibitor of the Gardos channel that ameliorates RBC dehydration in the SAD mouse. It blocks Ca2+-induced rubidium flux from human RBCs with an IC50 value of 11 ± 2 nM (CLT IC50 = 100 ± 12 nM) and inhibits RBC dehydration with an IC50 of 30 ± 20 nM. Senicapoc is used in the treatment of Sickle Cell Disease and Sickle Cell Anemia.