hereditary

By Targets:	Apoptosis (1) Cuproptosis (2) Factor Xa (1) Histone Methyltransferase (1) HPPD (1) Progesterone Receptor (1) Reactive Oxygen Species (1) Ser/Thr Protease (6) Small Interfering RNA (siRNA) (2) Transthyretin (TTR) (3)
By Research Areas:	Cancer (2) Cardiovascular Disease (4) Endocrinology (1) Inflammation/Immunology (2) Metabolic Disease (4) Neurological Disease (4)
Click Chemistry:	Alkynes (1)

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-16735

Avoralstat 4 Publications Verification BCX4161	Ser/Thr Protease	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-143476

Plasma kallikrein-IN-2	Ser/Thr Protease	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-P99110

Lanadelumab SHP643; DX-2930	Ser/Thr Protease	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-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.

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-12040

Elesclomol Maximum Cited Publications 24 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-139787

Donidalorsen ISIS-721744 free acid; IONIS-PKK-LRX free acid	Others	Others
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	Others	Others
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).

HY-150052

Plasma kallikrein-IN-3	Ser/Thr Protease	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
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-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 BCX7353 dihydrochloride	Ser/Thr Protease	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-109127

Berotralstat BCX7353	Ser/Thr Protease	Others
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-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 Others	Cardiovascular Disease Metabolic Disease Inflammation/Immunology
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 .

Cat. No.	Product Name

HY-L011

Membrane Transporter/Ion Channel Compound Library	1392 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 1392 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

1392 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 1392 smal-molecule modulators that can be used for the research of Ion Channel and Membrane Transporter or high throughput screening (HTS) related drug discovery.

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	Ser/Thr Protease	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.	Compare	Product Name	Species	Source

HY-P702321

	HFE Protein, Mouse (Cell-Free, His) hereditary hemochromatosis protein homolog	Mouse	E. coli Cell-free
	HFE Protein is crucial, binding to transferrin receptor (TFR) and reducing its affinity for iron-loaded transferrin. The interaction, pH-dependent and mediated by HFE's extracellular domain, highlights intricate molecular mechanisms. By modulating TFR's affinity, HFE plays a pivotal role in regulating iron homeostasis, emphasizing its significance in cellular processes related to iron uptake and metabolism. HFE Protein, Mouse (Cell-Free, His) is the recombinant mouse-derived HFE protein, expressed by E. coli Cell-free , with N-10*His labeled tag. The total length of HFE Protein, Mouse (Cell-Free, His) is 335 a.a., with molecular weight of 39.5 kDa.


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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 .

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