Gene mutation

By Targets:	Bacterial (1) Biochemical Assay Reagents (1) DNA/RNA Synthesis (1) EGFR (1) Estrogen Receptor/ERR (1) Mps1 (1) Neurotensin Receptor (1) PD-1/PD-L1 (1) Sodium Channel (2) Trk Receptor (1)
By Research Areas:	Cancer (8) Infection (2)
Oligonucleotides:	Antisense Oligonucleotides (6)

Targets Recommended: CGRP Receptor LAG-3 MALT1 NEKs

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-P99114

Sugemalimab	PD-1/PD-L1	Cancer
Sugemalimab is a fully human, full length, anti-programmed death ligand 1 (PD-L1) immunoglobulin G4 (IgG4) monoclonal antibody (mAb). Sugemalimab shows anticancer activities and can be used for non-small cell lung cancer research .

HY-P99688

Latozinemab AL001	Neurotensin Receptor	Cancer
Latozinemab (AL001) is a recombinant human anti-Sortilin monoclonal antibody. Latozinemab effectively binds Sortilin with a high affinity and blocks the interaction between progranulin protein (PGRN) and Sortilin receptor. Latozinemab has the potential for progranulin gene (GRN) mutations causative of Frontotemporal dementia (FTD) (FTD-GRN) research .

HY-126210

GJ072	Biochemical Assay Reagents	Others
GJ072 is a read-through compound (RTCs). GJ072 can induce “readthrough” .

HY-164503

Neptinib NEP010	EGFR	Cancer
Neptinib (NEP010) is an orally active derivative of Afatinib (HY-10261) that has stronger antitumor activity than Afatinib (HY-10261) by improving pharmacokinetics. Neptinib has a significant inhibitory effect on tumor growth in mouse non-small cell lung cancer models with different EGFR mutations. Neptinib has a certain inhibitory effect on the EGFR kinase family, with IC₅₀ values ??of 0.24 nM, 7.25 nM, 0.46 nM and 1.79 nM for EGFR ^wt, EGFR ^L858R/T790M, EGFR ^L858R and EGFR ^T790M, respectively .

HY-123359

RTC14	Others	Others
RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene .

HY-164516

ONCOII	Mps1	Cancer
ONCOII is an Mps1 inhibitor with an IC₅₀ of 10.8 nM. The inhibitory activity of ONCOII against Mps1 is affected by naturally occurring mutations in the Mps1 gene, and cells with Mps1 mutations exhibit increased resistance to ONCOII. ONCOII can be used in cancer research .

HY-139786

Cofirasersen ION-827359	Sodium Channel	Others
Cofirasersen is designed to reduce the expression of ENaC in the lung. ENaC is a sodium transport channel and believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene.

HY-139786A

Cofirasersen sodium ION-827359 sodium	Sodium Channel	Others
Cofirasersen sodium is designed to reduce the expression of ENaC in the lung. ENaC is a sodium transport channel and believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene.

HY-E70239

2-Methylbutyryl-CoA	Others	Others
2-Methylbutyryl-CoA is an intermediate of isoleucine metabolism. 2-Methylbutyryl-CoA dehydrogenase deficiency is a genetic disorder caused by mutations in the HADH2 gene .

HY-N12109

Albizziin	Others	Others
Albizziin is an amino acid analog with activity as a competitive inhibitor of asparagine synthetase. Albizziin has been used to isolate Chinese hamster ovary cell mutants with altered levels of the target enzyme. Several mutational classes of albizziin can be distinguished based on cross-resistance to β-aspartic hydroxamic acid. Studies on asparagine synthetase have shown that resistance to albizziin may be associated with altered regulation of asparagine synthetase, structural mutations of the enzyme, and gene amplification .

HY-B0013

Lavofloxacin lactate (-)-Ofloxacin lactate	Bacterial DNA/RNA Synthesis	Infection
Lavofloxacin lactate ((-)-ofloxacin lactate) is a class of broad-spectrum antimicrobials that can kill or inhibit a variety of bacteria. Lavofloxacin lactate binds to DNA rotase and topoisomerase IV, resulting in blocked DNA replication and repair, thus inhibiting bacterial growth. Lavofloxacin lactate can be used to study resistance mechanisms in bacteria, including studying resistance genes and mutations .

HY-150561

Trk-IN-20	Trk Receptor	Cancer
Trk-IN-20 is a kind of 3-vinylindazole derivatives. Trk-IN-20 suppresses Trk kinases functions by phosphorylation inhibition of TrkA/B/C with IC₅₀ values of 1.6 nM, 2.9 nM and 2.0 nM, respectively .

HY-W016819B

5-Fluoroorotic acid monohydrate 5-Fluorouracil-6-carboxylic acid Mono(hydrate); 5-FOA; 5-fluoro OA	Others	Infection
5-Fluoroorotic acid (monohydrate) (5-Fluorouracil-6-carboxylic acid Mono(hydrate); 5-FOA) is used to detect URA3 gene expression in yeast molecular genetic constructs. Yeast with an active URA3 gene (which encodes orotidine-5'-monophosphate decarboxylase) convert 5-FOA to fluorodeoxyuridine, which is toxic to cells. Resistant yeast strains carrying mutations in the URA3 gene will grow in the presence of 5-FOA if uracil is added to the culture medium.

HY-112611

H3B-5942	Estrogen Receptor/ERR	Cancer
H3B-5942 is a selective, irreversible and orally active estrogen receptor covalent antagonist, inactivates both wild-type and mutant ERα by targeting Cys530, with K_is of 1 nM and 0.41 nM, respectively. H3B-5942 reduces ERα target gene GREB1, shows potent antitumor activity both in multiple cell lines or animals bearing ERα ^WT or ERα mutations .

HY-167710

CRCD2	Others	Cancer
CRCD2 is a small molecule NT5C2 nucleotidase inhibitor with enhanced 6-mercaptopurine cytotoxic activity. CRCD2 can effectively reverse 6-mercaptopurine resistance caused by mutations in the NT5C2 gene or non-genetic activation mechanisms. CRCD2 combined with 6-mercaptopurine can enhance its cytotoxic activity in NT5C2 wild-type leukemia, showing its potential in the treatment of acute lymphoblastic leukemia .

HY-117051

STA-2842	Others	Cancer
STA-2842 is a small molecule agent that inhibits heat shock protein 90 (HSP90) to treat autosomal dominant polycystic kidney disease (ADPKD). The disease is caused by inherited mutations in the PKD1 or PKD2 genes that aberrantly activate multiple signaling proteins and pathways that regulate cell proliferation. Through network construction, we found that many HSP90 client proteins associated with ADPKD are regulated by HSP90. STA-2842 induced degradation of these clients in Pkd1?/? primary kidney cells and in vivo. In experiments using conditional Cre-mediated in vivo knockout of Pkd1 in mice, we found that weekly administration of STA-2842 for 10 weeks significantly reduced initial renal cyst formation and kidney growth in mice and slowed disease progression in mice with pre-existing cysts. These improved disease phenotypes were accompanied by improvements in renal function markers and reductions in the expression and activity of HSP90 clients and their effectors, with the extent of this inhibition correlating with the extent of cyst expansion in individual animals. Pharmacokinetic analysis showed that HSP90 was overexpressed in cystic kidney tissue and HSP90 inhibitors were selectively retained in cystic tissue, which is similar to the situation in solid tumors. These results provide a preliminary basis for evaluating HSP90 inhibitors as therapeutic agents for ADPKD.

Cat. No.	Product Name

HY-L015

PI3K/Akt/mTOR Compound Library	616 compounds
The PI3K/Akt/mTOR pathway controls many cellular processes that are important for the formation and progression of cancer, including apoptosis, transcription, translation, metabolism, angiogenesis, and cell cycle progression. Every major node of this signaling network is activated in a wide range of human tumors. Mechanisms for the pathway activation include activation of receptor tyrosine kinases (RTKs) upstream of PI3K, mutation or amplification of PIK3CA encoding p110α catalytic subunit of PI3K, mutation or loss of PTEN tumor suppressor gene, and mutation or amplification of Akt1. Once the pathway is activated, signaling through Akt can stimulate a series of substrates including mTOR which is involved in protein synthesis. Thus, inhibition of this pathway is an attractive concept for cancer prevention and/or therapy. Currently some mTOR inhibitors are approved for several indications, and there are several novel PI3K/Akt/mTOR inhibitors in clinical trials. MCE owns a unique collection of 616 compounds that can be used for PI3K/Akt/mTOR pathway research. PI3K/Akt/mTOR Compound Library also acts as a useful tool for anti-cancer drug discovery.

PI3K/Akt/mTOR Compound Library

616 compounds

The PI3K/Akt/mTOR pathway controls many cellular processes that are important for the formation and progression of cancer, including apoptosis, transcription, translation, metabolism, angiogenesis, and cell cycle progression. Every major node of this signaling network is activated in a wide range of human tumors. Mechanisms for the pathway activation include activation of receptor tyrosine kinases (RTKs) upstream of PI3K, mutation or amplification of PIK3CA encoding p110α catalytic subunit of PI3K, mutation or loss of PTEN tumor suppressor gene, and mutation or amplification of Akt1. Once the pathway is activated, signaling through Akt can stimulate a series of substrates including mTOR which is involved in protein synthesis. Thus, inhibition of this pathway is an attractive concept for cancer prevention and/or therapy. Currently some mTOR inhibitors are approved for several indications, and there are several novel PI3K/Akt/mTOR inhibitors in clinical trials.

MCE owns a unique collection of 616 compounds that can be used for PI3K/Akt/mTOR pathway research. PI3K/Akt/mTOR Compound Library also acts as a useful tool for anti-cancer drug discovery.

HY-L004

Cell Cycle/DNA Damage Compound Library	2,197 compounds
DNA is prone to numerous forms of damage that can injure cells and impair fitness. Cells have developed an array of mechanisms to repair these injuries. Proliferating cells are especially vulnerable to DNA damage due to the added demands of cellular growth and division. Cell cycle checkpoints represent integral components of DNA repair that coordinate cooperation between the machinery of the cell cycle and several biochemical pathways that respond to damage and restore DNA structure. By delaying progression through the cell cycle, checkpoints provide more time for repair before the critical phases of DNA replication, when the genome is replicated, and of mitosis, when the genome is segregated. Loss or attenuation of checkpoint function may increase spontaneous and induced gene mutations and chromosomal aberrations by reducing the efficiency of DNA repair. MCE owns a unique collection of 2,197 cell cycle/DNA damage-related compounds which can be used in the research of the same.

Cell Cycle/DNA Damage Compound Library

2,197 compounds

DNA is prone to numerous forms of damage that can injure cells and impair fitness. Cells have developed an array of mechanisms to repair these injuries. Proliferating cells are especially vulnerable to DNA damage due to the added demands of cellular growth and division. Cell cycle checkpoints represent integral components of DNA repair that coordinate cooperation between the machinery of the cell cycle and several biochemical pathways that respond to damage and restore DNA structure. By delaying progression through the cell cycle, checkpoints provide more time for repair before the critical phases of DNA replication, when the genome is replicated, and of mitosis, when the genome is segregated. Loss or attenuation of checkpoint function may increase spontaneous and induced gene mutations and chromosomal aberrations by reducing the efficiency of DNA repair.

MCE owns a unique collection of 2,197 cell cycle/DNA damage-related compounds which can be used in the research of the same.

HY-L011

Membrane Transporter/Ion Channel Compound Library	1,518 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 1,518 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

1,518 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 1,518 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-L101

Anti-Liver Cancer Compound Library	1,969 compounds
Liver cancer is one of the leading malignancies which occupies the second position in cancer deaths worldwide, becoming serious threat to human health. Hepatocellular carcinoma (HCC), also known as hepatoma is the most common type accounting for approximately 90% of all liver cancers. Current evidence indicates that during hepatocarcinogenesis, two main pathogenic mechanisms prevail: (1) cirrhosis associated with hepatic regeneration after tissue damage caused by hepatitis infection, toxins or metabolic influences, and (2) mutations occurring in single or multiple oncogenes or tumor suppressor genes. Both mechanisms have been linked with alterations in several important cellular signaling pathways. These include the RAF/MEK/ERK pathway, PI3K/AKT/mTOR pathway, WNT/b-catenin pathway, insulin-like growth factor pathway, c-MET/HGFR pathway , etc. MCE offers a unique collection of 1,969 compounds with identified and potential anti-liver cancer activity. MCE anti-liver cancer compound library is a useful tool for anti-liver cancer drugs screening and other related research.

Anti-Liver Cancer Compound Library

1,969 compounds

Liver cancer is one of the leading malignancies which occupies the second position in cancer deaths worldwide, becoming serious threat to human health. Hepatocellular carcinoma (HCC), also known as hepatoma is the most common type accounting for approximately 90% of all liver cancers.

Current evidence indicates that during hepatocarcinogenesis, two main pathogenic mechanisms prevail: (1) cirrhosis associated with hepatic regeneration after tissue damage caused by hepatitis infection, toxins or metabolic influences, and (2) mutations occurring in single or multiple oncogenes or tumor suppressor genes. Both mechanisms have been linked with alterations in several important cellular signaling pathways. These include the RAF/MEK/ERK pathway, PI3K/AKT/mTOR pathway, WNT/b-catenin pathway, insulin-like growth factor pathway, c-MET/HGFR pathway , etc.

MCE offers a unique collection of 1,969 compounds with identified and potential anti-liver cancer activity. MCE anti-liver cancer compound library is a useful tool for anti-liver cancer drugs screening and other related research.

HY-L083

Anti-Cancer Metabolism Compound Library	2,227 compounds
Mutations in oncogenes and tumor suppressor genes can modify multiple signaling pathways and in turn cell metabolism, which facilitates tumorigenesis. The paramount hallmark of tumor metabolism is “aerobic glycolysis” or the Warburg effect, coined by Otto Warburg in 1926, in which cancer cells produce most of energy from glycolysis pathway regardless of whether in aerobic or anaerobic condition. Usually, cancer cells are highly glycolytic (glucose addiction) and take up more glucose than do normal cells from outside. The increased uptake of glucose is facilitated by the overexpression of several isoforms of membrane glucose transporters (GLUTs). Likewise, the metabolic pathways of glutamine, amino acid and fat metabolism are also altered. Recent trends in anti-cancer drug discovery suggests that targeting the altered metabolic pathways of cancer cells result in energy crisis inside the cancer cells and can selectively inhibit cancer cell proliferation by delaying or suppressing tumor growth. MCE provides a unique collection of 2,227 compounds which cover various tumor metabolism-related signaling pathways. These compounds can be used for anti-cancer metabolism targets identification, validation as well anti-cancer drug discovery.

Anti-Cancer Metabolism Compound Library

2,227 compounds

Mutations in oncogenes and tumor suppressor genes can modify multiple signaling pathways and in turn cell metabolism, which facilitates tumorigenesis. The paramount hallmark of tumor metabolism is “aerobic glycolysis” or the Warburg effect, coined by Otto Warburg in 1926, in which cancer cells produce most of energy from glycolysis pathway regardless of whether in aerobic or anaerobic condition. Usually, cancer cells are highly glycolytic (glucose addiction) and take up more glucose than do normal cells from outside. The increased uptake of glucose is facilitated by the overexpression of several isoforms of membrane glucose transporters (GLUTs). Likewise, the metabolic pathways of glutamine, amino acid and fat metabolism are also altered. Recent trends in anti-cancer drug discovery suggests that targeting the altered metabolic pathways of cancer cells result in energy crisis inside the cancer cells and can selectively inhibit cancer cell proliferation by delaying or suppressing tumor growth.

MCE provides a unique collection of 2,227 compounds which cover various tumor metabolism-related signaling pathways. These compounds can be used for anti-cancer metabolism targets identification, validation as well anti-cancer drug discovery.

Cat. No.	Product Name	Target	Research Area

HY-P5369

[Arg6]-β-Amyloid (1-42), england mutation	Peptides	Others
[Arg6]-β-Amyloid (1-42), england mutation is a biological active peptide. (Several mutations in the beta amyloid precursor gene cause autosomal dominant Alzheimer's Disease in a number of kindreds.Tthe English (H6R) mutation will disrupt H6 interactions.)

HY-P5331

[Asn23]-beta-Amyloid (1-42), iowa mutation	Peptides	Others
[Asn23]-beta-Amyloid (1-42), iowa mutation is a biological active peptide. (Several mutations in the beta amyloid precursor gene cause autosomal dominant Alzheimer's Disease in a number of kindreds. The Iowa mutation, where Asp 23 is replaced with Asn, is associated with severe cerebral amyloid beta-protein angiopathy (CAA). The affected individuals share a missense mutation in APP at position 694. The mutated beta-amyloid peptide aggregates more rapidly and forms toxic fibrils.)

HY-P5365

[Asn23] β-Amyloid (1-40), Iowa mutation	Peptides	Others
[Asn23] β-Amyloid (1-40), Iowa mutation is a biological active peptide. (Several mutations in the beta amyloid precursor gene cause autosomal dominant Alzheimer's Disease in a number of kindreds. The Iowa mutation, where Asp 23 is replaced with Asn, is associated with severe cerebral amyloid beta-protein angiopathy (CAA). The affected individuals share a missense mutation in APP at position 694. The mutated beta-amyloid peptide aggregates more rapidly and forms toxic fibrils.)

HY-P5368

[Arg6]-β-Amyloid (1-40), england mutation	Peptides	Others
[Arg6]-β-Amyloid (1-40), england mutation is a biological active peptide. (Several mutations in the beta amyloid precursor gene cause autosomal dominant Alzheimer's Disease in a number of kindreds. Among them, the English mutation, with His at position 6 replaced with Arg, was reported to accelerate the kinetics of oligomers formation which act as fibril seeds and are more toxic to cultured neuronal cells.)

Cat. No.	Product Name	Target	Research Area

HY-P99114

Sugemalimab	PD-1/PD-L1	Cancer
Sugemalimab is a fully human, full length, anti-programmed death ligand 1 (PD-L1) immunoglobulin G4 (IgG4) monoclonal antibody (mAb). Sugemalimab shows anticancer activities and can be used for non-small cell lung cancer research .

HY-P99688

Latozinemab AL001	Neurotensin Receptor	Cancer
Latozinemab (AL001) is a recombinant human anti-Sortilin monoclonal antibody. Latozinemab effectively binds Sortilin with a high affinity and blocks the interaction between progranulin protein (PGRN) and Sortilin receptor. Latozinemab has the potential for progranulin gene (GRN) mutations causative of Frontotemporal dementia (FTD) (FTD-GRN) research .

Cat. No.	Product Name	Category	Target	Chemical Structure

HY-P99114

Sugemalimab	Classification of Application Fields Disease Research Fields Cancer	PD-1/PD-L1
Sugemalimab is a fully human, full length, anti-programmed death ligand 1 (PD-L1) immunoglobulin G4 (IgG4) monoclonal antibody (mAb). Sugemalimab shows anticancer activities and can be used for non-small cell lung cancer research .

HY-N12109

Albizziin	Structural Classification Microorganisms Ketones, Aldehydes, Acids Source classification	Others
Albizziin is an amino acid analog with activity as a competitive inhibitor of asparagine synthetase. Albizziin has been used to isolate Chinese hamster ovary cell mutants with altered levels of the target enzyme. Several mutational classes of albizziin can be distinguished based on cross-resistance to β-aspartic hydroxamic acid. Studies on asparagine synthetase have shown that resistance to albizziin may be associated with altered regulation of asparagine synthetase, structural mutations of the enzyme, and gene amplification .

Cat. No.	Product Name		Classification

HY-153496

Sepofarsen QR 110		Antisense Oligonucleotides
Sepofarsen (QR-110) is an RNA antisense oligonucleotide targeting to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene.

HY-153496A

Sepofarsen sodium QR 110 sodium		Antisense Oligonucleotides
Sepofarsen (QR-110) sodium is an RNA antisense oligonucleotide targeting to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene.

HY-139786

Cofirasersen ION-827359		Antisense Oligonucleotides
Cofirasersen is designed to reduce the expression of ENaC in the lung. ENaC is a sodium transport channel and believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene.

HY-139786A

Cofirasersen sodium ION-827359 sodium		Antisense Oligonucleotides
Cofirasersen sodium is designed to reduce the expression of ENaC in the lung. ENaC is a sodium transport channel and believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene.

HY-147412A

Ultevursen sodium QR-421a sodium		Antisense Oligonucleotides
Ultevursen sodium is a single-stranded RNA based oligonucleotide that is designed to skip exon 13 in the RNA with the aim to stop vision loss in people that have retinitis pigmentosa due to a mutation in exon 13 of the USH2A gene (encoding usherin).

HY-147412

Ultevursen QR-421a		Antisense Oligonucleotides
Ultevursen (QR-421a) is a single-stranded RNA based oligonucleotide that is designed to skip exon 13 in the RNA with the aim to stop vision loss in people that have retinitis pigmentosa due to a mutation in exon 13 of the USH2A gene (encoding usherin). Ultevursen sequence: (P-thio)[2′-O-(2-methoxyethyl)](A-G-m ⁵C-m ⁵U-m ⁵U-m ⁵C-G-G-A-G-A-A-A-m ⁵U-m ⁵U-m ⁵U-A-A-A-m ⁵U-m ⁵C) .

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Screening Libraries

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Oligonucleotides

Gene mutation

Inhibitors & Agonists

Screening Libraries

Peptides

Inhibitory Antibodies

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Oligonucleotides

Natural
Products