Browse small molecules associated with genetic disease areas for mutation-linked pathway research, mechanism studies, and discovery screening. This category helps organize compounds relevant to inherited disorders, rare disease biology, and target-focused follow-up work.
Genetic diseases
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- BQ-123, Antagonist of ET A receptorCAS: 136553-81-6 Formula: C31H42N6O7 Molecular Weight: 610.7Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: B275090View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
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- SMILES
- CC(C)CC1C(=O)NC(C(=O)NC(C(=O)N2CCCC2C(=O)NC(C(=O)N1)C(C)C)CC(=O)O)CC3=CNC4=CC=CC=C43
- InChIKey
- VYCMAAOURFJIHD-PJNXIOHISA-N
- InChI
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- Synonyms
- 2-((3R,6S,9R,12R,17aS)-9-((1H-indol-3-yl)methyl)-6-isobutyl-3-isopropyl-1,4,7,10,13-pentaoxohexadecahydro-1H-pyrrolo[...
- GSK-3 Inhibitor XIII, Inhibitor of aurora kinase A;Inhibitor of glycogen synthase kinase 3 beta;Inhibitor of SRC proto-oncogene; non-receptor tyrosine kinaseCAS: 404828-08-6 Formula: C18H19N5 Molecular Weight: 305.3Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%◐ .Made to order · 8–12 wks Item #: G338805View ProductPricing & Pack Sizes
Technical Identifiers
- Synonyms
- (5-Methyl-1H-pyrazol-3-yl)-(2-phenylquinazolin-4-yl)amine
- GW 590735, Peroxisome proliferator-activated receptor alpha agonistCAS: 622402-22-6 Formula: C23H21F3N2O4S Molecular Weight: 478.48● In Stock — US Item #: G275270View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- 2-methyl-2-[4-[[[4-methyl-2-[4-(trifluoromethyl)phenyl]-1,3-thiazole-5-carbonyl]amino]methyl]phenoxy]propanoic acid
- SMILES
- CC1=C(SC(=N1)C2=CC=C(C=C2)C(F)(F)F)C(=O)NCC3=CC=C(C=C3)OC(C)(C)C(=O)O
- InChIKey
- ILUPZUOBHCUBKB-UHFFFAOYSA-N
- InChI
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- Synonyms
- HMS3650D07 | DB07215 | QKY617BBX5 | EX-A5892 | ILUPZUOBHCUBKB-UHFFFAOYSA-N | 2-methyl-2-{4-[({4-methyl-2-[4-(trifluor...
- GSK-1016790A, Activator of TRPV4CAS: 942206-85-1 Formula: C28H32Cl2N4O6S2 Molecular Weight: 655.61Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: G275374View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- N-[(2S)-1-[4-[(2S)-2-[(2,4-dichlorophenyl)sulfonylamino]-3-hydroxypropanoyl]piperazin-1-yl]-4-methyl-1-oxopentan-2-yl]-1-benzothiophene-2-carboxamide
- SMILES
- CC(C)CC(C(=O)N1CCN(CC1)C(=O)C(CO)NS(=O)(=O)C2=C(C=C(C=C2)Cl)Cl)NC(=O)C3=CC4=CC=CC=C4S3
- InChIKey
- IVYQPSHHYIAUFO-VXKWHMMOSA-N
- InChI
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- Synonyms
- GSK101 | HY-19608 | GSK 1016790A | N-[(2S)-1-[4-[(2S)-2-[(2,4-dichlorophenyl)sulfonylamino]-3-hydroxypropanoyl]pipera...
- GW 1929 hydrochloride◐ .Made to order · 8–12 wks Item #: G286659View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- (2S)-2-(2-benzoylanilino)-3-[4-[2-[methyl(pyridin-2-yl)amino]ethoxy]phenyl]propanoic acid;hydrochloride
- SMILES
- CN(CCOC1=CC=C(C=C1)CC(C(=O)O)NC2=CC=CC=C2C(=O)C3=CC=CC=C3)C4=CC=CC=N4.Cl
- InChIKey
- KXNKIKXTGRMLEY-YCBFMBTMSA-N
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- Synonyms
- N-(2-Benzoylphenyl)-O-[2-(methyl-2-pyridinylamino)ethyl]-L-tyrosine hydrochloride
- GSK1904529ACAS: 1089283-49-7 Formula: C44H47F2N9O5S Molecular Weight: 851.96Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: G127237View ProductPricing & Pack Sizes
Technical Identifiers
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- SMILES
- CCC1=CC(=C(C=C1N2CCC(CC2)N3CCN(CC3)S(=O)(=O)C)OC)NC4=NC=CC(=N4)C5=C(N=C6N5C=CC=C6)C7=CC(=C(C=C7)OC)C(=O)NC8=C(C=CC=C8F)F
- InChIKey
- MOSKATHMXWSZTQ-UHFFFAOYSA-N
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- Synonyms
- MOSKATHMXWSZTQ-UHFFFAOYSA-N | SB18897 | Trifluralin [ANSI:BSI:ISO] | AMY24202 | A24618 | GSK 4529 | IGF-1R inhibitor ...
- GSK690693, Protein kinase C (PKC) inhibitorMoligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: G127527View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- 4-[2-(4-amino-1,2,5-oxadiazol-3-yl)-1-ethyl-7-[[(3S)-piperidin-3-yl]methoxy]imidazo[4,5-c]pyridin-4-yl]-2-methylbut-3-yn-2-ol
- SMILES
- CCN1C2=C(C(=NC=C2OCC3CCCNC3)C#CC(C)(C)O)N=C1C4=NON=C4N
- InChIKey
- KGPGFQWBCSZGEL-ZDUSSCGKSA-N
- InChI
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- Synonyms
- (R)-4-(2-(4-Amino-1,2,5-oxadiazol-3-yl)-1-ethyl-7-(piperidin-3-yl-MeO)-1H-imidazo[4,5-c]pyridin-4-yl)-2-Me-but-3-yn-2...
- GSK872CAS: 1346546-69-7 Formula: C19H17N3O2S2 Molecular Weight: 383.48Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: G276439View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- N-(6-propan-2-ylsulfonylquinolin-4-yl)-1,3-benzothiazol-5-amine
- SMILES
- CC(C)S(=O)(=O)C1=CC2=C(C=CN=C2C=C1)NC3=CC4=C(C=C3)SC=N4
- InChIKey
- ZCDBTQNFAPKACC-UHFFFAOYSA-N
- InChI
- 1S/C19H17N3O2S2/c1-12(2)26(23,24)14-4-5-16-15(10-14)17(7-8-20-16)22-13-3-6-19-18(9-13)21-11-25-19/h3-12H,1-2H3,(H,20,22)
- Synonyms
- GSK′872 | N-(6-(Isopropylsulfonyl)quinolin-4-yl)benzo[d]thiazol-5-amine
- Luzindole, Antagonist of MT 1 receptor;Antagonist of MT 2 receptorCAS: 117946-91-5 Formula: C19H20N2O Molecular Weight: 292.37Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥96%● In Stock — US Item #: L132758View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- N-[2-(2-benzyl-1H-indol-3-yl)ethyl]acetamide
- SMILES
- CC(=O)NCCC1=C(NC2=CC=CC=C21)CC3=CC=CC=C3
- InChIKey
- WVVXBPKOIZGVNS-UHFFFAOYSA-N
- InChI
- 1S/C19H20N2O/c1-14(22)20-12-11-17-16-9-5-6-10-18(16)21-19(17)13-15-7-3-2-4-8-15/h2-10,21H,11-13H2,1H3,(H,20,22)
- Synonyms
- 2-Benzyl-N-acetyltryptamine | HMS3411B10 | N-(2-(2-(PHENYLMETHYL)-1H-INDOL-3-YL)ETHYL)ETHANAMIDE | AM20020263 | Q6706...
- Nelfinavir Mesylate● In Stock — US Item #: N137745View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
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- SMILES
- CC1=C(C=CC=C1O)C(=O)NC(CSC2=CC=CC=C2)C(CN3CC4CCCCC4CC3C(=O)NC(C)(C)C)O.CS(=O)(=O)O
- InChIKey
- NQHXCOAXSHGTIA-SKXNDZRYSA-N
- InChI
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- Synonyms
- (3S,4aS,8aS)-3-(tert-butylcarbamoyl)-2-[(2R,3R)-2-hydroxy-3-[(3-hydroxy-2-methylbenzoyl)amino]-4-(phenylsulfanyl)buty...
- ACHPCAS: 406208-42-2 Formula: C21H24N4O2 Molecular Weight: 364.44● In Stock — US Item #: A288136View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- 2-amino-6-[2-(cyclopropylmethoxy)-6-hydroxyphenyl]-4-piperidin-4-ylpyridine-3-carbonitrile
- SMILES
- C1CC1COC2=CC=CC(=C2C3=NC(=C(C(=C3)C4CCNCC4)C#N)N)O
- InChIKey
- DYVFBWXIOCLHPP-UHFFFAOYSA-N
- InChI
- 1S/C21H24N4O2/c22-11-16-15(14-6-8-24-9-7-14)10-17(25-21(16)23)20-18(26)2-1-3-19(20)27-12-13-4-5-13/h1-3,10,13-14,24,26H,4-9,12H2,(H2,23,25)
- Synonyms
- 2-amino-6-(2-(cyclopropylmethoxy)-6-hydroxyphenyl)-4-(4-piperidinyl)-3-pyridine-carbonitrile | 2-amino-6-[2-(cyclopro...
- Nimodipine, Gating inhibitor of Ca v1.1;Gating inhibitor of Ca v1.2;Gating inhibitor of Ca v1.3;Gating inhibitor of Ca v1.4;Activator of CFTR;Antagonist of Mineralocorticoid receptor;Allosteric modulator of P2X4Solid Moligand™ ? Moligand™ — Aladdin's line of ligands and bioactive small molecules. Use for receptor, pathway, and binding studies needing defined small-molecule tools. ≥98%● In Stock — US Item #: N129506View ProductPricing & Pack Sizes
Technical Identifiers
- IUPAC Name
- 3-O-(2-methoxyethyl) 5-O-propan-2-yl 2,6-dimethyl-4-(3-nitrophenyl)-1,4-dihydropyridine-3,5-dicarboxylate
- SMILES
- CC1=C(C(C(=C(N1)C)C(=O)OC(C)C)C2=CC(=CC=C2)[N+](=O)[O-])C(=O)OCCOC
- InChIKey
- UIAGMCDKSXEBJQ-UHFFFAOYSA-N
- InChI
- 1S/C21H26N2O7/c1-12(2)30-21(25)18-14(4)22-13(3)17(20(24)29-10-9-28-5)19(18)15-7-6-8-16(11-15)23(26)27/h6-8,11-12,19,22H,9-10H2,1-5H3
- Synonyms
- Nimodipine ap | SPECTRUM1503600 | 2-methoxyethyl propan-2-yl 2,6-dimethyl-4-(3-nitrophenyl)-1,4-dihydropyridine-3,5-d...
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Frequently Asked Questions
What is included under Genetic diseases?
This listing collects compounds and tool molecules annotated to targets and pathways associated with Genetic diseases. It is an entry point for scoping a focused research set, not a therapeutic recommendation.
What does Genetic diseases cover?
Genetic diseases is a disease-annotated listing within By Disease (DOID), drawing together compounds reported in connection with this area across several target families.
How do I select compounds for Genetic diseases research?
For Genetic diseases, start from the target or pathway hypothesis rather than the disease label, then check that the reported activity was measured in a model relevant to your system. Confirm potency, selectivity, and solubility before committing to a set. All products are supplied for professional research and development use only, and are not for use in humans, animals, diagnostics, or therapy.
How do I find a specific compound under Genetic diseases?
Search Genetic diseases by compound name, CAS number, or SMILES, or filter by the underlying target. For unlisted compounds or bulk quantities, contact [email protected]. We supply verified businesses, research institutions, universities, and government agencies.