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(20607)
Research Areas Research AreasChromosomal Disease
Genetic disorders arise from abnormalities in single genes (monogenic), multiple genes (polygenic), or chromosomal structures. Over 6,000 genetic diseases have been described, and new conditions continue to be identified. Advances in genomics, gene editing, and molecular diagnostics have transformed understanding of disease mechanisms. Ongoing research is fundamental for enabling precision diagnostics, carrier screening, and the development of gene- and cell-based therapies.
Tezacaftor
Cited
VX661
T22631152311-62-0
Tezacaftor (VX661) is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.
- $34
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CitedBomedemstat
IMG-7289, IMG7289
T96341990504-34-1
Bomedemstat (IMG-7289), an orally active and irreversible lysine-specific demethylase 1 (LSD1) inhibitor, shows activity in mouse myeloproliferative neoplasm (MPN) models. This compound exhibits antineoplastic effects and can be used for acute myeloid leukemia (AML) and myelofibrosis (MF) research.
- $67
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Pepinemab
VX 15/2503
T773922097151-87-4
Pepinemab(VX 15/2503) is a humanized IgG4 monoclonal antibody targeting sematone 4D. Pepinemab has anti-cancer activity and blocks the activity of SEMA4D, which is used to study degenerative diseases similar to Alzheimer's disease.
- $198
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Olinciguat
IW-1701
T163831628732-62-6
Olinciguat (IW-1701) is an oral guanylate cyclase (sGC) stimulator. It also has concentration-dependent stimulation of sGC in purified rat and human enzyme assays and a whole-cell assay.
- $188
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Ivacaftor Cited
VX-770, Ivacaftor (VX-770)
T2588873054-44-5
Ivacaftor (VX-770) (VX-770) is a potentiator of CFTR targeting G551D-CFTR (EC50: 100 nM) and F508del-CFTR (EC50: 25 nM) in Fisher rat thyroid cells, respectively.
- $32
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CitedSeliciclib Cited
R-roscovitine, Roscovitine, CYC202
T2095186692-46-6
Seliciclib (Roscovitine) is a potent inhibitor of Cdk2/cyclin E (IC50=0.1 µM). Seliciclib also inhibits Cdk7/cyclin H, Cdk5/p35 and Cdc2/cyclin B (IC50=0.49/0.16/0.65 µM). Seliciclib has antitumor activity.
- $40
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CitedBitopertin
RO4917838, RG1678, Paliflutine
T6788845614-11-1
Bitopertin (Paliflutine) (RG1678, RO-4917838) is a potent inhibitor of glycine transporter 1 (GlyT1), with Ki of 8.1 nM for human hGlyT1b and IC50 of 22-25 nM in Chinese hamster ovary cells.
- $35
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Garadacimab
CSL312
T770182162134-62-3
Garadacimab (CSL312) is a potent whole-human IgG4 monoclonal antibody targeting activation factor XII (FXIIa). Garadacimab can be used to study hereditary angioedema.
- $313
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Eliglustat Cited
GENZ-112638, Genz 99067
T3663491833-29-5
Eliglustat (Genz 99067) is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease.
- $52
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CitedTolvaptan Cited
OPC-41061
T2326150683-30-0
Tolvaptan (OPC-41061) is an orally bioavailable, selective, arginine vasopressin receptor 2 (V2, AVPR2) antagonist that can be used to treat hyponatremia.
- $32
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CitedL-Glutamine Cited
L-Glutamic acid 5-amide
T0326L56-85-9
L-Glutamine (L-Glutamic acid 5-amide) is a non-essential amino acid that exists in large quantities in the human body and participates in many metabolic processes. L-Glutamine provides a carbon source for oxidation in certain cells.
- $29
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CitedThymidine Cited
Thymidin, NSC 21548, DThyd, Doxribtimine, Deoxyribothymidine, 5-Methyldeoxyuridine
TWP291150-89-5
Thymidine (DThyd) is a cell synchronizing agent and a specific precursor of deoxyribonucleic acid. Thymidine inhibits DNA synthesis and can cause cell cycle arrest in the G1/S phase.
- $45
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CitedSelumetinib Cited
AZD6244, ARRY-142886
T6218606143-52-6
Selumetinib (AZD6244) is a MEK1/2 inhibitor that inhibits MEK1 (IC50=14 nM) with potent selectivity and is non-ATP-competitive. Selumetinib has antitumor activity and is used for the treatment of neurofibromatosis type 1 (NF1).
- $47
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CitedLumacaftor Cited
VX-809, VRT 826809
T2595936727-05-8
Lumacaftor (VRT 826809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein. It enhances F508del-CFTR protein maturation in FRT cells (EC50: 100 nM).
- $43
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CitedSutimlimab
T767022049079-64-1
Sutimlimab is a novel monoclonal antibody targeting complement protein C1. Sutimlimab is commonly used as a C1 inhibitor to treat cold agglutina-associated hemolysis.
- $208
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Arimoclomol
BRX-220 free base
T13553289893-25-0
Arimoclomol (BRX-220 free base), a co-inducer of heat shock proteins (HSP), is utilized in studies on the treatment of amyotrophic lateral sclerosis (ALS).
- $35
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Concizumab
NN 7415, mAb 2021
T773731312299-39-0
Concizumab is an anti-TFPI monoclonal antibody (IgG4 type) that selectively binds to the Kunitz protease inhibitor (KPI) 2 domain of TFPI, blocking its interaction with the FXa active site. Injections of Concizumab increase thrombin in blood, thereby preventing and treating hemophilia.
- $228
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Blu-782
ALK2-IN-1
T81082141955-96-4
Blu-782 (ALK2-IN-1) is a activin receptor-like kinase-2 (ALK2) inhibitor ( IC50 of <10 nM)
- $74
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Acebilustat
CTX-4430
T3715943764-99-6
Acebilustat (CTX-4430) (ZK322) is an effective and specific leukotriene B4 hydrolase inhibitor.
- $32
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Afamelanotide acetate
T75891566590-77-9
Afamelanotide acetate is a synthetic α-melanocyte stimulating hormone analogue and first-in-class melanocortin-1 receptor agonist, used to for Erythropoietic Protoporphyria.
- $67
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Bardoxolone Methyl Cited
TP-155, RTA 402, NSC 713200, CDDO Methyl ester
T6165218600-53-4
Bardoxolone Methyl (TP-155) is a synthetic triterpenoid that acts as an activator of the Nrf2 pathway and an inhibitor of the NF-κB pathway with potential anti-tumor and anti-inflammatory activities.
- $39
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CitedTiomolibdate diammonium Cited
NSC 286644, Coprexa, ATTM, Ammonium tetrathiomolybdate, Ammonium molybdenum sulfide
T1966815060-55-6
Tiomolibdate diammonium (NSC-286644) is an inhibitor of superoxide dismutase 1 (SOD1) exerting antiangiogenic and antitumor activities. Tiomolibdate diammonium inhibits the activities of cuproenzymes, including SOD1 and COX.
- $35
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Cited2-Aminoethanethiol
β-Mercaptoethylamine, β-MEA, Cysteamine, 2-Mercaptoethylamine
T108060-23-1
2-Aminoethanethiol (Cysteamine) is a Cystine Depleting Agent. The mechanism of action of cysteamine is as a Cystine Disulfide Reduction.
- $45
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Liarozole
R75251 dihydrochloride
T11847L115575-11-6
Liarozole (R75251 dihydrochloride) is a cytochrome P450 (CYP450) dependent inhibitor, orally active, it also a potent inhibitor of estrogen (via inhibition of aromatase) and testicular androgen synthesis (inhibition of 17 ,20-lyase).Liarozole dihydrochloride is an imidazole derivative; it is being investigated as a non-hormonal agent in prostate cancer and in the treatment of various other cancers and skin disorders.
- $52
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