Search Result

Results for "

hereditary

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Amyloid-β (1) Apoptosis (2) Bcr-Abl (1) Biochemical Assay Reagents (2) Cuproptosis (2) DNA Alkylator/Crosslinker (1) DNA/RNA Synthesis (1) Drug Metabolite (1) E1/E2/E3 Enzyme (1) Endogenous Metabolite (2) ERK (1) Factor Xa (3) Ferroportin (2) FGFR (1) GABA Receptor (1) HPPD (3) Isotope-Labeled Compounds (3) Kallikrein (9) Liposome (2) LYTACs (1) Phosphatase (1) Plasminogen/Plasmin (2) Progesterone Receptor (1) RAD51 (1) Reactive Oxygen Species (ROS) (2) Reference Standards (2) RNA MTase (1) RSV (1) Ser/Thr Protease (4) Small Interfering RNA (siRNA) (4) Thrombin (1) TMPRSS6 (2) Transthyretin (TTR) (6) γ-secretase (1)
By Research Areas:	Cancer (11) Cardiovascular Disease (17) Endocrinology (1) Infection (1) Inflammation/Immunology (4) Metabolic Disease (10) Neurological Disease (10)
Click Chemistry:	Alkynes (1)
Oligonucleotides:	Nucleotide Analogs (1) Cationic Lipids (1) siRNA drugs (7) Antisense Oligonucleotides (4) Adenine Nucleotide (1) siRNAs (7)

Inhibitors & Agonists

Screening Libraries

Biochemical Assay Reagents

Peptides

Inhibitory Antibodies

Natural
Products

Isotope-Labeled Compounds

Antibodies

Click Chemistry

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-12040

Elesclomol Maximum Cited Publications 115 Publications Verification STA-4783	Reactive Oxygen Species (ROS) Apoptosis Cuproptosis	Cancer
Elesclomol (STA-4783) is a potent copper ionophore and promotes copper-dependent cell death (cuproptosis). Elesclomol specifically binds ferredoxin 1 (FDX1) α2/α3 helices and β5 strand. Elesclomol inhibits FDX1-mediated Fe-S cluster biosynthesis. Elesclomol is an oxidative stress inducer that induces cancer cell apoptosis. Elesclomol is a reactive oxygen species (ROS) inducer. Elesclomol can be used for Menkes and associated disorders of hereditary copper deficiency research .

HY-B0607

Nitisinone 2 Publications Verification NTBC; Nitisone; SC0735	HPPD	Metabolic Disease
Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

HY-132609

Patisiran sodium 1 Publications Verification	Small Interfering RNA (siRNA) Transthyretin (TTR)	Neurological Disease
Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis .

HY-P10272

Rusfertide PTG-300	Ferroportin	Others
Rusfertide is a peptide mimetic of natural hepcidin, which targets and degrades ferroportin, reduces serum iron and transferrin-saturation, and thus regulates the production of red blood cells. Rusfertide ameliorates the polycythemia vera, β-thalassemia and hereditary hemochromatosis .

HY-16735

Avoralstat 4 Publications Verification BCX4161	Kallikrein	Others
Avoralstat (BCX4161), a potent and orally active plasma kallikrein (PKK) inhibitor, is used for hereditary angioedema research .

HY-P99631

Garadacimab 1 Publications Verification CSL312	Factor Xa	Cardiovascular Disease
Garadacimab (CSL312) is a first-in-class, fully human IgG4 monoclonal antibody targeting activated factor XII (FXIIa). Garadacimab has the potential for hereditary angioedema research .

HY-132608

Inotersen sodium ISIS-420915 sodium	Transthyretin (TTR)	Neurological Disease
Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide. Inotersen sodium inhibits the production of transthyretin (TTR) protein by targeting the TTR RNA transcript and reduces the levels of the TTR transcript. Inotersen sodium can be used for the research of hereditary TTR amyloidosis polyneuropathy .

HY-112974

Inotersen GSK-2998728; ISIS-420915	Transthyretin (TTR)	Cardiovascular Disease Neurological Disease
Inotersen (GSK-2998728; ISIS-420915) is a 2'-O-methoxyethyl-modified antisense oligonucleotide and transthyretin (TTR) inhibitor with low genotoxicity. Inotersen triggers RNase H1-mediated degradation by binding to TTR mRNA, thereby effectively reducing the production of both mutant and wild-type transthyretin in the liver. Inotersen significantly reduces amyloid fiber deposition, yet specific toxicities such as inflammation or tumors are observed at high doses in some animal models. Inotersen is used in studies of hereditary transthyretin amyloidosis and the associated polyneuropathy and cardiomyopathy .

HY-109127

Berotralstat 1 Publications Verification BCX7353	Kallikrein	Cardiovascular Disease Cancer
Berotralstat (BCX7353) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema .

HY-173189A

2-5A pentasodium solution (100 mM) 2′,5′-ApApA pentasodium; 2′,5′-trioligoadenylate pentasodium; 5'-O-Triphosphoryladenylyl-(2'→5')-adenylyl-(2'→5')-adenosine pentasodium	RSV DNA/RNA Synthesis	Infection Cancer
2-5A (2′,5′-ApApA) pentasodium solution 100 mM is a high-affinity (K_a=0.04 nM) RNase L binder. 2-5A pentasodium solution 100 mM activates the nuclease activity of latent RNase L to cleave single-stranded RNA by inducing conformational changes and dimerization of latent RNase L, thereby mediating critical antiviral responses and enhancing interferon effects. 2-5A pentasodium solution 100 mM not only effectively inhibits Rauscher murine leukemia virus replication, reduces viremia and splenomegaly, but also enhances antileukemic efficacy when combined with amphotericin B and exhibits favorable high-dose tolerance. 2-5A pentasodium solution 100 mM is an important tool molecule for investigating the pathological mechanisms of viral infection, hereditary prostate cancer and leukemia .

HY-150229

306-N16B	Liposome	Cancer
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

HY-P99110

Lanadelumab SHP643; DX-2930; X124-G01	Kallikrein	Inflammation/Immunology
Lanadelumab (SHP643) is a human IgG1 monoclonal antibody against plasma kallikrein (pKal) with an K_i value of 0.12 nM. Lanadelumab inhibits both free and HMWK (high molecular weight kininogen)-bound pKal. Lanadelumab has the potential for the research of hereditary angioedema .

HY-126301

DMT1 blocker 1 3 Publications Verification	Ferroportin	Metabolic Disease
DMT1 blocker 1 is an orally active blocker of divalent metal transporter 1 (DMT1) with an IC₅₀ of 0.64 μM. DMT1 blocker 1 inhibits intestinal cell absorption of non-heme iron, thereby alleviating iron overload by blocking the DMT1 transporter. DMT1 blocker 1 demonstrates significant efficacy in rodent models of acute iron hyperabsorption. DMT1 blocker 1 is useful for studying iron overload disorders such as hereditary hemochromatosis and thalassemia .

HY-150151A

GalNac-siRNA negative control (21nt)	LYTACs	Cardiovascular Disease
GalNac-siRNA negative control (21nt) is the negative control form of GalNac-siRNA. GalNac-siRNA is an Asialoglycoprotein receptor (ASGPR)-targeted inhibitor conjugate. GalNac-siRNA is promising for research of hereditary transthyretin amyloidosis, acute hepatic porphyria, hemophilia and hypercholesterolemia .

HY-139787

Donidalorsen ISIS-721744 free acid; IONIS-PKK-LRX free acid	Kallikrein	Cardiovascular Disease Inflammation/Immunology
Donidalorsen (ISIS-721744; IONIS-PKK-LRX free acid) is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen can be used in the research of hereditary angioedema .

HY-139787A

Donidalorsen sodium ISIS-721744; IONIS-PKK-LRX	Kallikrein	Cardiovascular Disease Inflammation/Immunology
Donidalorsen (ISIS-721744; IONIS-PKK-LRX) sodium is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen sodium inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen sodium can be used in the research of hereditary angioedema .

HY-153609

AS-Patisiran sodium	Transthyretin (TTR) Small Interfering RNA (siRNA)	Neurological Disease
AS-Patisiran sodium is an antisense strand of Patisiran. Patisiran is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran can be used for the research of hereditary TTR amyloidosis .

HY-160928

Smurf1 modulator-1	E1/E2/E3 Enzyme	Cardiovascular Disease
Smurf1 modulator-1 (Compound 20) is a selective inhibitor for Smad ubiquitination regulatory factor 1 (Smurf1), with IC₅₀ of 180 nM .

HY-132590A

Revusiran sodium ALN-TTRSC sodium	Small Interfering RNA (siRNA) Transthyretin (TTR)	Neurological Disease
Revusiran (ALN-TTRSC) sodium is an RNA interference agent targeting the mRNA of transthyretin (Transthyretin, TTR). Revusiran sodium mediates sequence-specific degradation of TTR mRNA through RNA interference, reduces the synthesis of TTR protein, binds to GalNAc ligands, and is taken up by hepatocytes via the asialoglycoprotein receptor. Revusiran sodium exhibits favorable nonclinical safety profiles. Revusiran sodium can be used in studies related to transthyretin-mediated amyloidosis .

HY-109127A

Berotralstat dihydrochloride 1 Publications Verification BCX7353 dihydrochloride	Kallikrein	Cardiovascular Disease Cancer
Berotralstat dihydrochloride (BCX7353 dihydrochloride) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema .

HY-P991629

C1 Esterase Inhibitor (Human)	Ser/Thr Protease	Inflammation/Immunology
C1 Esterase Inhibitor (Human) is a C1 Esterase inhibitor derived from human plasma. C1 Esterase Inhibitor (Human), a glycoprotein, is a serum protease inhibitor (serpin) that binds covalently and inactivates C1r, C1s, and mannan-binding protein-associated proteases (MASPs). C1 Esterase Inhibitor (Human) has anti-inflammatory effects. C1 Esterase Inhibitor (Human) can be used to prevent angioedema attacks associated with hereditary angioedema .

HY-147278

Manusiran Divesiran; SLN124	Ser/Thr Protease Small Interfering RNA (siRNA) TMPRSS6	Cardiovascular Disease
Manusiran (Divesiran) is a GalNac-siRNA targeting liver and transmembrane serine protease 6 (Serine protease 6). Manusiran increases hepatic Hepcidin synthesis and plasma levels by silencing TMPRSS6, a negative regulator of hepcidin production, and limits the availability of iron required for erythropoiesis. Combined use of Manusiran with Deferiprone (HY-B0568) reduces ineffective erythropoiesis and hepatic iron overload in a mouse model of β-thalassemia. Manusiran can be used for research on polycythemia vera, type 1 hereditary hemochromatosis, and β-thalassemia .

HY-111302

Norgestrienone	Progesterone Receptor	Endocrinology
Norgestrienone is a progestin receptor agonist. Norgestrienone is commonly used as a progesterone compound in birth control pills and can be combined with ethinylestradiol. Norgestrienone is a click chemical. It contains Alkyne groups and can undergo copper-catalyzed azide-alkyne cycloaddition reactions (CuAAc) with molecules containing Azide groups. Norgestrienone can be used in the study of hereditary angioneurotic edema .

HY-N2093

Vicine 2 Publications Verification	Drug Metabolite	Cardiovascular Disease Metabolic Disease
Vicine, an alkaloid glycoside found mainly in fava beans, is toxic in individuals who have a hereditary loss of the enzyme glucose-6-phosphate dehydrogenase and may cause haemolytic anaemia.Vicine is an inactive compound that is hydrolysed by the intestinal microflora to a highly reactive free radical generating compound, the aglycone divicine when Vicine enters the body through food .

HY-B0607R

Nitisinone (Standard) 2 Publications Verification NTBC (Standard); Nitisone (Standard); SC0735 (Standard)	HPPD Reference Standards	Metabolic Disease
Nitisinone (Standard) is the analytical standard of Nitisinone. This product is intended for research and analytical applications. Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

HY-150052

Plasma kallikrein-IN-3	Kallikrein	Cardiovascular Disease Metabolic Disease
Plasma kallikrein-IN-3 is a plasma kallikrein inhibitor (IC₅₀: 0.15 μM). Plasma kallikrein-IN-3 can be used for hereditary angioedema, diabetic macular edema, and diabetic retinopathy research .

HY-122583

D-G23	RAD51	Cancer
D-G23 is a selective RAD52 inhibitor. D-G23 disrupts RAD52-mediated DNA repair pathways and suppresses the growth of BRCA1- and BRCA2-deficient cancer cells. D-G23 is promising for research of homologous recombination-related cancers, such as hereditary breast cancer and ovarian cancer caused by BRCA1/2 mutations .

HY-P11260

MD5	Ser/Thr Protease Thrombin	Metabolic Disease
MD5 is a selective TMPRSS6 mimetic peptide inhibitor with an IC₅₀ for recombinant human TMPRSS6 protein of 22 nM and a K_i of 3.4 nM. MD5 exhibits the significantly reduced inhibitory effect on Matriptase, with an IC₅₀ of 352 nM and a K_i of 99.2 nM. MD5 exhibits good target specificity and only has a weak inhibitory effect on thrombin (Thrombin), with K_i of 120 nM. MD5 demonstrates good initial drugability and can be used for the study of iron overload diseases (such as hereditary hemochromatosis, β-thalassemia) .

HY-143476

Plasma kallikrein-IN-2	Kallikrein	Cardiovascular Disease Metabolic Disease
Plasma kallikrein-IN-2 (Compound 198) is a potent plasma kallikrein (PKal) inhibitor with an IC₅₀ of 0.1 nM. Plasma kallikrein-IN-2 can be used for hereditary angioedema, diabetic macular edema, and diabetic retinopathy research .

HY-124044

ML 086	Phosphatase	Cardiovascular Disease
ML 086 is a highly selective PHOSPHO1 phosphatase inhibitor (IC₅₀=0.14 μM). ML 086 is promising for research of hereditary vascular calcification disorders (e.g., generalized arterial calcification of infancy) and spinal ligament ossification .

HY-101207

NCS-382	GABA Receptor	Neurological Disease
NCS-382 is a potent GABA receptor antagonist and also a GHBR receptor antagonist. NCS-382 has anticonvulsant and antisedative activity. NCS-382 is used in the related research of hereditary nervous system diseases .

HY-153141

MP-1	RNA MTase	Cancer
MP-1 is a potent Fumarate hydratase-dependent hit. MP-1 engages an array of functional cysteines, including one lying in the Zn-finger domain of the tRNA methyltransferase enzyme TRMT1. MP-1 causes fumarate hydratase-dependent synthetic lethality in a metastatic hereditary leiomyomatosis and renal cell carcinoma cell line .

HY-12040S

Elesclomol-d2 STA-4783-d₂	Isotope-Labeled Compounds Reactive Oxygen Species (ROS) Apoptosis Cuproptosis	Cancer
Elesclomol-d₂ (STA-4783-d₂) is a deuterium labeled Elesclomol (HY-12040). Elesclomol (STA-4783) is a potent copper ionophore and promotes copper-dependent cell death (cuproptosis). Elesclomol specifically binds ferredoxin 1 (FDX1) α2/α3 helices and β5 strand. Elesclomol inhibits FDX1-mediated Fe-S cluster biosynthesis. Elesclomol is an oxidative stress inducer that induces cancer cell apoptosis. Elesclomol is a reactive oxygen species (ROS) inducer. Elesclomol can be used for Menkes and associated disorders of hereditary copper deficiency research .

HY-163464

Plasma kallikrein-IN-5	Kallikrein	Cardiovascular Disease
Plasma kallikrein-IN-5 (Compound 20) is a potent covalent inhibitor of plasma potassium kinin peptide (Pka) with IC₅₀ values of 66 nM and 70 pM at 1 minute and 24 hours, respectively. Plasma kallikrein-IN-5 can be used IN the study of hereditary angioedema (HAE) .

HY-163329

BCH-HSP-C01	Biochemical Assay Reagents	Neurological Disease
BCH-HSP-C01 is a lead compound that restores AP-4-dependent protein trafficking in neuronal models of adapter protein complex 4 (AP-4)-associated hereditary spastic paraplegia. BCH-HSP-C01 can be used for the research of autophagy-associated diseases .

HY-109127R

Berotralstat (Standard) BCX7353 (Standard)	Reference Standards Ser/Thr Protease	Cardiovascular Disease Cancer
Berotralstat (Standard) is the analytical standard of Berotralstat. This product is intended for research and analytical applications. Berotralstat (BCX7353) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema.

HY-W414498

1-Deoxysphingosine	Endogenous Metabolite	Neurological Disease
1-Deoxysphingosine is elevated in lymphoblasts of individuals with hereditary sensory neuropathy type 1 (HSAN1) disorder; it features a (4E) double bond in its structure and is produced through the catabolism of 1-deoxyceramide catalyzed by the ceramidase enzyme, classifying it as an unsaturated deoxy-sphingoid base.

HY-145343A

(9R)-RO7185876	γ-secretase Amyloid-β	Neurological Disease
(9R)-RO7185876 (Compound example 16) is a γ-secretase inhibitor. (9R)-RO7185876 inhibits Αβ42 secretion. (9R)-RO7185876 can be used for the researches of Alzheimer's disease, cerebral amyloid angiopathy, hereditary cerebral hemorrhage with amyloidosis, multi-infarct dementia, dementia pugilistica or Down syndrome .

HY-B0607S

Nitisinone-d3 NTBC-d₃; Nitisone-d₃; SC0735-d₃	Isotope-Labeled Compounds HPPD	Metabolic Disease
Nitisinone-d₃ (NTBC-d₃) is deuterium labeled Nitisinone. Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

HY-W777526

Nitisinone-13C6	Isotope-Labeled Compounds	Others
Nitisinone- ¹³C₆ is a ¹³C-labeled Nitisinone (HY-B0607). Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

HY-186173

MD-3-A45	ERK FGFR	Metabolic Disease
MD-3-A45 is an inhibitor of fibroblast growth factor 23 (FGF-23). MD-3-A45 blocks the activation of the FGFRs/α-klotho signaling pathway by FGF-23 and inhibits FGF-23-induced ERK activation. MD-3-A45 can be used in the research of chronic kidney disease, hereditary hypophosphatemic rickets and acquired hypophosphatemic rickets .

HY-185319

Pexamsiran	Plasminogen/Plasmin	Others
Pexamsiran is a siRNA that inhibits plasminogen synthesis, and can be use for the study of hereditary haemorrhagic telangiectasia.

HY-185319A

Pexamsiran sodium	Plasminogen/Plasmin	Others
Pexamsiran sodium is a siRNA that inhibits plasminogen synthesis reducer, and can be use for the study of hereditary haemorrhagic telangiectasia.

HY-118523

AL-321	Endogenous Metabolite	Metabolic Disease
AL-321 is an insulin sensitizer. AL-321 exhibits significant hypoglycemic and hypolipidemic activities in insulin resistance models. AL-321 can be used in the research of hereditary obesity-related diabetes .

HY-141489

DSPE-PEG2000-Carboxy NHS sodium	Biochemical Assay Reagents Liposome	Neurological Disease
DSPE-PEG2000-Carboxy NHS sodium is a linker. DSPE-PEG2000-Carboxy NHS sodium enables conjugation of targeting peptides to lipid nanoparticles after their preparation. DSPE-PEG2000-Carboxy NHS (sodium) is applicable to the research of hereditary retinal degeneration .

HY-P991994

REGN9533	Factor Xa	Cardiovascular Disease
REGN9533 is a human-derived monoclonal antibody against FXII/FXIIa with high affinity for FXII, FXIIa and β-FXIIa. REGN9533 selectively inhibits the intrinsic coagulation pathway and FXIIa-driven plasma kallikrein activity. REGN9533 is applicable to FXIIa-related diseases such as thrombosis and hereditary angioedema .

HY-P11698

Guanidino-G-Clamp-PNA	DNA Alkylator/Crosslinker Transthyretin (TTR)	Cancer
Guanidino-G-Clamp-PNA is a highly efficient sequence-specific RNA binder and gene silencer. Guanidino-G-Clamp-PNA precisely targets such targets as miR-155 or transthyretin (TTR) mRNA through base pairing: the former regulates tumor-related signaling pathways by reducing microRNA activity, while the latter inhibits the translation of harmful proteins via steric hindrance. Guanidino-G-Clamp-PNA effectively stabilizes DNA/RNA duplexes, induces cancer cell apoptosis, and suppresses tumor growth. In addition, Guanidino-G-Clamp-PNA can be conjugated with targeting ligands to improve tissue-specific delivery and reduce in vivo adverse reactions, and it can also enhance the splicing regulation efficacy of other oligonucleotide platforms (such as PMO) when integrated into them. Guanidino-G-Clamp-PNA is applicable to the research of various diseases including diffuse large B-cell lymphoma and hereditary transthyretin-related amyloidosis .

HY-180217

WGU55	TMPRSS6 Factor Xa	Cardiovascular Disease
WGU55 is a selective and potent reversible type II transmembrane serine protease TMPRSS6 inhibitor with a K_i of 12.15 nM. WGU55 inhibits TMPRSS6 activity with an IC₅₀ of 138 nMin KEK293 cells. WGU55 has a K_i of 3510 nM (SI = 289) against the homologous protease matriptase and a K_i of 5.2 μM against the coagulation key protease Factor Xa. WGU55 can be used for the research of iron overload related diseases, such as hereditary hemochromatosis .

HY-182792

Embibatinib TERN-701	Bcr-Abl	Cancer
Embibatinib (TERN-701) (Compound 1) is a highly selective, orally effective BCR-ABL allosteric inhibitor. Embibatinib is applicable for cancer research, particularly leukemia .

Cat. No.	Product Name

HY-L011

Membrane Transporter/Ion Channel Compound Library

2,174 compounds

Most of molecules enter or leave cells mainly via membrane transport proteins, which play important roles in several cellular functions, including cell metabolism, ion homeostasis, signal transduction, the recognition process in the immune system, energy transduction, etc. There are three major types of transport proteins, ATP-powered pumps, channel proteins and transporters. Transport proteins such as channels and transporters play important roles in the maintenance of intracellular homeostasis, and mutations in these transport protein genes have been identified in the pathogenesis of a number of hereditary diseases. In the central nervous system, ion channels have been linked to, but not limited to, many diseases such asataxias, paralyses, epilepsies, and deafness. This indicates the roles of ion channels in the initiation and coordination of movement, sensory perception, and encoding and processing of information. Ion channels are a major class of drug targets in drug development.

MCE designs a unique collection of 2,174 smal-molecule modulators that can be used for the research of Ion Channel and Membrane Transporter or high throughput screening (HTS) related drug discovery.

HY-L204

Lactic Acid Metabolic Compound Library

535 compounds

Lactic acid metabolism is one of the key metabolic pathways within living organisms. It plays a crucial role not only in cellular energy conversion but is also closely related to a variety of physiological and pathological processes. The production and clearance of lactic acid are important indicators of cellular metabolic balance, and its abnormal regulation may lead to conditions such as lactic acidosis, muscle fatigue, and hereditary metabolic diseases. Moreover, lactic acid is closely related to the malignancy of tumors and is considered a biomarker for malignant tumors and poor prognosis. Lactic acid can serve as a metabolic substrate to support the metabolic needs of tumor cells under hypoxic conditions, and it can also cause acidification of the tumor microenvironment, suppress immune cell function to promote immune evasion, and induce drug resistance in tumor cells. Currently, targeting lactic acid-lactylation and its related metabolic pathways has become a new research avenue for cancer treatment. In-depth exploration of the molecular mechanisms of lactic acid metabolism can help in screening lead compounds that regulate the lactic acid metabolism.

MCE contains 535 small molecule compounds targeting enzymes involved in lactic acid metabolism. This library is of significant value for researching the role of lactate metabolism in the mechanisms of diseases.

Cat. No.	Product Name	Type

HY-150229

306-N16B

Biochemical Assay Reagents

306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

Cat. No.	Product Name	Target	Research Area

HY-P10272

Rusfertide PTG-300	Ferroportin	Others
Rusfertide is a peptide mimetic of natural hepcidin, which targets and degrades ferroportin, reduces serum iron and transferrin-saturation, and thus regulates the production of red blood cells. Rusfertide ameliorates the polycythemia vera, β-thalassemia and hereditary hemochromatosis .

HY-P11260

MD5	Ser/Thr Protease Thrombin	Metabolic Disease
MD5 is a selective TMPRSS6 mimetic peptide inhibitor with an IC₅₀ for recombinant human TMPRSS6 protein of 22 nM and a K_i of 3.4 nM. MD5 exhibits the significantly reduced inhibitory effect on Matriptase, with an IC₅₀ of 352 nM and a K_i of 99.2 nM. MD5 exhibits good target specificity and only has a weak inhibitory effect on thrombin (Thrombin), with K_i of 120 nM. MD5 demonstrates good initial drugability and can be used for the study of iron overload diseases (such as hereditary hemochromatosis, β-thalassemia) .

HY-P11698

Guanidino-G-Clamp-PNA	DNA Alkylator/Crosslinker Transthyretin (TTR)	Cancer
Guanidino-G-Clamp-PNA is a highly efficient sequence-specific RNA binder and gene silencer. Guanidino-G-Clamp-PNA precisely targets such targets as miR-155 or transthyretin (TTR) mRNA through base pairing: the former regulates tumor-related signaling pathways by reducing microRNA activity, while the latter inhibits the translation of harmful proteins via steric hindrance. Guanidino-G-Clamp-PNA effectively stabilizes DNA/RNA duplexes, induces cancer cell apoptosis, and suppresses tumor growth. In addition, Guanidino-G-Clamp-PNA can be conjugated with targeting ligands to improve tissue-specific delivery and reduce in vivo adverse reactions, and it can also enhance the splicing regulation efficacy of other oligonucleotide platforms (such as PMO) when integrated into them. Guanidino-G-Clamp-PNA is applicable to the research of various diseases including diffuse large B-cell lymphoma and hereditary transthyretin-related amyloidosis .

Cat. No.	Product Name	Target	Research Area	Image

HY-P99631

Garadacimab 1 Publications Verification CSL312	Factor Xa	Cardiovascular Disease
Garadacimab (CSL312) is a first-in-class, fully human IgG4 monoclonal antibody targeting activated factor XII (FXIIa). Garadacimab has the potential for hereditary angioedema research .

HY-P99110

Lanadelumab SHP643; DX-2930; X124-G01	Kallikrein	Inflammation/Immunology
Lanadelumab (SHP643) is a human IgG1 monoclonal antibody against plasma kallikrein (pKal) with an K_i value of 0.12 nM. Lanadelumab inhibits both free and HMWK (high molecular weight kininogen)-bound pKal. Lanadelumab has the potential for the research of hereditary angioedema .

HY-P991629

C1 Esterase Inhibitor (Human)	Ser/Thr Protease	Inflammation/Immunology
C1 Esterase Inhibitor (Human) is a C1 Esterase inhibitor derived from human plasma. C1 Esterase Inhibitor (Human), a glycoprotein, is a serum protease inhibitor (serpin) that binds covalently and inactivates C1r, C1s, and mannan-binding protein-associated proteases (MASPs). C1 Esterase Inhibitor (Human) has anti-inflammatory effects. C1 Esterase Inhibitor (Human) can be used to prevent angioedema attacks associated with hereditary angioedema .

HY-P991994

REGN9533	Factor Xa	Cardiovascular Disease
REGN9533 is a human-derived monoclonal antibody against FXII/FXIIa with high affinity for FXII, FXIIa and β-FXIIa. REGN9533 selectively inhibits the intrinsic coagulation pathway and FXIIa-driven plasma kallikrein activity. REGN9533 is applicable to FXIIa-related diseases such as thrombosis and hereditary angioedema .

Cat. No.	Product Name	Category	Target	Chemical Structure

HY-N2093

Vicine 2 Publications Verification	Alkaloids Classification of Application Fields Leguminosae Other Alkaloids Metabolic Disease Plants Vicia faba L. Disease Research Fields Source Classification	Drug Metabolite
Vicine, an alkaloid glycoside found mainly in fava beans, is toxic in individuals who have a hereditary loss of the enzyme glucose-6-phosphate dehydrogenase and may cause haemolytic anaemia.Vicine is an inactive compound that is hydrolysed by the intestinal microflora to a highly reactive free radical generating compound, the aglycone divicine when Vicine enters the body through food .

Cat. No.	Product Name	Chemical Structure

HY-12040S

Elesclomol-d2

Elesclomol-d₂ (STA-4783-d₂) is a deuterium labeled Elesclomol (HY-12040). Elesclomol (STA-4783) is a potent copper ionophore and promotes copper-dependent cell death (cuproptosis). Elesclomol specifically binds ferredoxin 1 (FDX1) α2/α3 helices and β5 strand. Elesclomol inhibits FDX1-mediated Fe-S cluster biosynthesis. Elesclomol is an oxidative stress inducer that induces cancer cell apoptosis. Elesclomol is a reactive oxygen species (ROS) inducer. Elesclomol can be used for Menkes and associated disorders of hereditary copper deficiency research .

HY-B0607S

Nitisinone-d3

Nitisinone-d₃ (NTBC-d₃) is deuterium labeled Nitisinone. Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

HY-W777526

Nitisinone-13C6
Nitisinone- ¹³C₆ is a ¹³C-labeled Nitisinone (HY-B0607). Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC₅₀ of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .

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HY-P85199

	HFE Antibody (YA4891) HFE; HLAH; hereditary hemochromatosis protein; HLA-H	WB, ICC/IF, ELISA	Human
	HFE Antibody (YA4891) is a Mouse-derived and non-conjugated monoclonal antibody, targeting to HFE.

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Cat. No.	Product Name		Classification

HY-111302

Norgestrienone		Alkynes
Norgestrienone is a progestin receptor agonist. Norgestrienone is commonly used as a progesterone compound in birth control pills and can be combined with ethinylestradiol. Norgestrienone is a click chemical. It contains Alkyne groups and can undergo copper-catalyzed azide-alkyne cycloaddition reactions (CuAAc) with molecules containing Azide groups. Norgestrienone can be used in the study of hereditary angioneurotic edema .

Cat. No.	Product Name		Classification

HY-132609

Patisiran sodium 1 Publications Verification		siRNAs siRNA drugs
Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis .

HY-132608

Inotersen sodium ISIS-420915 sodium		Antisense Oligonucleotides
Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide. Inotersen sodium inhibits the production of transthyretin (TTR) protein by targeting the TTR RNA transcript and reduces the levels of the TTR transcript. Inotersen sodium can be used for the research of hereditary TTR amyloidosis polyneuropathy .

HY-112974

Inotersen GSK-2998728; ISIS-420915		Antisense Oligonucleotides
Inotersen (GSK-2998728; ISIS-420915) is a 2'-O-methoxyethyl-modified antisense oligonucleotide and transthyretin (TTR) inhibitor with low genotoxicity. Inotersen triggers RNase H1-mediated degradation by binding to TTR mRNA, thereby effectively reducing the production of both mutant and wild-type transthyretin in the liver. Inotersen significantly reduces amyloid fiber deposition, yet specific toxicities such as inflammation or tumors are observed at high doses in some animal models. Inotersen is used in studies of hereditary transthyretin amyloidosis and the associated polyneuropathy and cardiomyopathy .

HY-173189A

2-5A pentasodium solution (100 mM) 2′,5′-ApApA pentasodium; 2′,5′-trioligoadenylate pentasodium; 5'-O-Triphosphoryladenylyl-(2'→5')-adenylyl-(2'→5')-adenosine pentasodium		Nucleotide Analogs Adenine Nucleotide
2-5A (2′,5′-ApApA) pentasodium solution 100 mM is a high-affinity (K_a=0.04 nM) RNase L binder. 2-5A pentasodium solution 100 mM activates the nuclease activity of latent RNase L to cleave single-stranded RNA by inducing conformational changes and dimerization of latent RNase L, thereby mediating critical antiviral responses and enhancing interferon effects. 2-5A pentasodium solution 100 mM not only effectively inhibits Rauscher murine leukemia virus replication, reduces viremia and splenomegaly, but also enhances antileukemic efficacy when combined with amphotericin B and exhibits favorable high-dose tolerance. 2-5A pentasodium solution 100 mM is an important tool molecule for investigating the pathological mechanisms of viral infection, hereditary prostate cancer and leukemia .

HY-150229

306-N16B		Cationic Lipids
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .

HY-150151A

GalNac-siRNA negative control (21nt)		siRNAs siRNA drugs
GalNac-siRNA negative control (21nt) is the negative control form of GalNac-siRNA. GalNac-siRNA is an Asialoglycoprotein receptor (ASGPR)-targeted inhibitor conjugate. GalNac-siRNA is promising for research of hereditary transthyretin amyloidosis, acute hepatic porphyria, hemophilia and hypercholesterolemia .

HY-139787

Donidalorsen ISIS-721744 free acid; IONIS-PKK-LRX free acid		Antisense Oligonucleotides
Donidalorsen (ISIS-721744; IONIS-PKK-LRX free acid) is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen can be used in the research of hereditary angioedema .

HY-139787A

Donidalorsen sodium ISIS-721744; IONIS-PKK-LRX		Antisense Oligonucleotides
Donidalorsen (ISIS-721744; IONIS-PKK-LRX) sodium is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen sodium inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen sodium can be used in the research of hereditary angioedema .

HY-153609

AS-Patisiran sodium		siRNAs siRNA drugs
AS-Patisiran sodium is an antisense strand of Patisiran. Patisiran is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran can be used for the research of hereditary TTR amyloidosis .

HY-132590A

Revusiran sodium ALN-TTRSC sodium		siRNAs siRNA drugs
Revusiran (ALN-TTRSC) sodium is an RNA interference agent targeting the mRNA of transthyretin (Transthyretin, TTR). Revusiran sodium mediates sequence-specific degradation of TTR mRNA through RNA interference, reduces the synthesis of TTR protein, binds to GalNAc ligands, and is taken up by hepatocytes via the asialoglycoprotein receptor. Revusiran sodium exhibits favorable nonclinical safety profiles. Revusiran sodium can be used in studies related to transthyretin-mediated amyloidosis .

HY-147278

Manusiran Divesiran; SLN124		siRNAs siRNA drugs
Manusiran (Divesiran) is a GalNac-siRNA targeting liver and transmembrane serine protease 6 (Serine protease 6). Manusiran increases hepatic Hepcidin synthesis and plasma levels by silencing TMPRSS6, a negative regulator of hepcidin production, and limits the availability of iron required for erythropoiesis. Combined use of Manusiran with Deferiprone (HY-B0568) reduces ineffective erythropoiesis and hepatic iron overload in a mouse model of β-thalassemia. Manusiran can be used for research on polycythemia vera, type 1 hereditary hemochromatosis, and β-thalassemia .

HY-185319

Pexamsiran		siRNAs siRNA drugs
Pexamsiran is a siRNA that inhibits plasminogen synthesis, and can be use for the study of hereditary haemorrhagic telangiectasia.

HY-185319A

Pexamsiran sodium		siRNAs siRNA drugs
Pexamsiran sodium is a siRNA that inhibits plasminogen synthesis reducer, and can be use for the study of hereditary haemorrhagic telangiectasia.

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