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Results for "

hereditary

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Apoptosis (1) Cuproptosis (2) E1/E2/E3 Enzyme (1) Factor Xa (1) Ferroportin (1) GABA Receptor (1) Histone Methyltransferase (1) HPPD (2) Kallikrein (7) Progesterone Receptor (1) Reactive Oxygen Species (1) Ser/Thr Protease (1) Small Interfering RNA (siRNA) (2) Transthyretin (TTR) (3)
By Research Areas:	Cancer (4) Cardiovascular Disease (6) Endocrinology (1) Inflammation/Immunology (2) Metabolic Disease (5) Neurological Disease (6)
Click Chemistry:	Alkynes (1)
Oligonucleotides:	Antisense Oligonucleotides (3) siRNAs (2)

Inhibitors & Agonists

Screening Libraries

Peptides

Inhibitory Antibodies

Click Chemistry

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

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-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-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-P99110

Lanadelumab SHP643; DX-2930	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-12040

Elesclomol Maximum Cited Publications 46 Publications Verification STA-4783	Reactive Oxygen Species 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-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-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-163329

BCH-HSP-C01	Others	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-141876

PRMT5-IN-14	Histone Methyltransferase	Cancer
PRMT5-IN-14 is a PRMT5 inhibitor to research cancer, sickle cell, and hereditary persistence of foetal hemoglobin (HPFH) mutations.

HY-109127

Berotralstat 1 Publications Verification BCX7353	Kallikrein	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-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-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-132609

Patisiran sodium	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-W414498

1-Deoxysphingosine	Others	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-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-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-109127A

Berotralstat dihydrochloride 1 Publications Verification BCX7353 dihydrochloride	Kallikrein	Others
Berotralstat dihydrochloride is a low toxicity, effective, highly specific, second-generation, synthetic and orally active plasma kallikrein inhibitor used for the research of hereditary angioedema (HAE) attacks. Berotralstat dihydrochloride works by blocking the enzymatic activity of plasma kallikrein in releasing bradykinin, the major biologic peptide that promotes swelling and pain associated with attacks of HAE .

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-128530

Tetrathiomolybdate	Cuproptosis	Cardiovascular Disease Metabolic Disease Inflammation/Immunology Cancer
Tetrathiomolybdate, an orally active anti-copper agent, reduces copper levels in the body. Tetrathiomolybdate has a protective effect on collagen-induced arthritis in mice. Tetrathiomolybdate also reduces blood sugar, but has no effect on mice with hereditary diabetes (db/db). Tetrathiomolybdate inhibit angiogenesis, also shows antiangiogenic effects in malignant pleural mesothelioma .

HY-B0607R

Nitisinone (Standard) NTBC (Standard); Nitisone (Standard); SC56735 (Standard)	HPPD	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-109127R

Berotralstat (Standard)	Ser/Thr Protease	Others
Berotralstat (Standard) is the analytical standard of Berotralstat. This product is intended for research and analytical applications. Berotralstat (BCX7353) is a low toxicity, effective, highly specific, second-generation, synthetic and orally active plasma kallikrein inhibitor used for the research of hereditary angioedema (HAE) attacks. Berotralstat works by blocking the enzymatic activity of plasma kallikrein in releasing bradykinin, the major biologic peptide that promotes swelling and pain associated with attacks of HAE .

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 .

Cat. No.	Product Name

HY-L011

Membrane Transporter/Ion Channel Compound Library	1,491 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 1,491 smal-molecule modulators that can be used for the research of Ion Channel and Membrane Transporter or high throughput screening (HTS) related drug discovery.

Membrane Transporter/Ion Channel Compound Library

1,491 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 1,491 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	330 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 330 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.

Lactic Acid Metabolic Compound Library

330 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 330 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	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 .

Cat. No.	Product Name	Target	Research Area

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	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 .

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-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-132609

Patisiran sodium		siRNAs
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-153609

AS-Patisiran sodium		siRNAs
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-139787

Donidalorsen ISIS-721744 free acid; IONIS-PKK-LRX free acid		Antisense Oligonucleotides
Donidalorsen is an antisense oligonucleotide designed to reduce the production of prekallikrein (PKK). PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of Hereditary angioedema (HAE).

HY-139787A

Donidalorsen sodium ISIS-721744; IONIS-PKK-LRX		Antisense Oligonucleotides
Donidalorsen (sodium) is an antisense oligonucleotide designed to reduce the production of prekallikrein (PKK). PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of Hereditary angioedema (HAE).

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