hereditary — TargetMol Chemicals

Cat No.	Product Name	Synonyms	Targets
T14358	Avoralstat	BCX4161	Others
	Avoralstat (BCX4161) is an oral plasma kallikrein (PKK) inhibitor. Which is used for the treatment of hereditary angioedema.		Others
T6170	Elesclomol	STA-4783	Apoptosis , HSP , Reactive Oxygen Species
	Elesclomol (STA-4783) is an oxidative stress inducer and a highly lipophilic copper ion carrier. Elesclomol induces apoptosis in tumor cells and is used in copper death related studies. Elesclomol also inhibits FDX1-medi...		Apoptosis , HSP , Reactive Oxygen Species
T19655	N-Acetyl-D-mannosamine	DEXM74,ManNAc,DEX M 74,DEX M-74,N-Acetylmannosamine hydrate,DEX-M-74,N-Acetylmannosamine	Antibacterial
	N-Acetyl-D-mannosamine (ManNAc) is a drug potentially for the treatment of hereditary inclusion body myopathy.		Antibacterial
T22344	Icatibant Acetate		Bradykinin Receptor
	Icatibant Acetate, an antagonist of the bradykinin B2 receptor, is used in the treatment of hereditary angioedema.		Bradykinin Receptor
T25276	Crilvastatin	PMD387,PMD 387,PMD-387	HMG-CoA Reductase
	Crilvastatin (PMD 387) is a novel non-competitive hydroxymethylglutaryl coenzyme A reductase inhibitor with cholesterol-lowering activity that inhibits cholesterol uptake in rats with hereditary hypercholesterolemia.		HMG-CoA Reductase
T77018	Garadacimab	CSL312	Factor Xa
	Garadacimab (CSL312) is a potent whole-human IgG4 monoclonal antibody targeting activation factor XII (FXIIa). Garadacimab can be used to study hereditary angioedema.		Factor Xa
T4994	Tocofersolan		Others
	Tocofersolan (TPGS) is a polyethylene glycol derivative of α-tocopherol and synthetic water-soluble version of Tocopherol. Tocofersolan is an oral treatment of vitamin E deficiency due to digestive malabsorption in pedia...		Others
T8644	Elamipretide Triacetate	Elamipretide TriTFA	Others
	Elamipretide Triacetate (Elamipretide TriTFA) is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane where it associates with cardiolipin...		Others
TP2487	Oligopeptide-68 Acetate	Oligopeptide-68 Acetate(1206525-47-4 Free base)	Others
	Oligopeptide-68 Acetate is a peptide. It is a novel whitening ingredient that mimics the binding of TGF-β to cell surface receptors to reduce induced and hereditary pigmentation by inhibiting MITF intramolecular channels...		Others
T75319	Taurodeoxycholic acid	Taurodeoxychloic acid	Apoptosis , Calcium Channel , Caspase
	Taurodeoxycholic acid (Taurodeoxychloic acid) is a bile acid taurine conjugate of deoxycholic acid, a human metabolite that stabilizes mitochondrial membranes and reduces the formation of free radicals.Taurodeoxycholic a...		Apoptosis , Calcium Channel , Caspase
T0755	Carglumic Acid	Carbaglu,N-Carbamylglutamate,N-Carbamyl-L-glutamic acid,Carbamylglutamic acid,Carbamino-L-glutamic acid,Ureidoglutaric acid	Others
	Carglumic Acid (Ureidoglutaric acid) is an orphan drug used to treat hyperammonemia in patients with N-acetyl glutamate synthase absence. This rare hereditary disease results in elevated blood levels of ammonia, which ca...		Others
T33725	Norgestrienone	Planor,A-301,A301,Ogyline,A 301
	Nogotrienone is an anabolic steroid with fertility and contraceptive activity that has been studied as a therapeutic agent for hereditary angioneurotic edema.
T38909	Inotersen sodium	ISIS-420915 sodium
	Inotersen sodium, also known as ISIS-420915 sodium, is a 2′-O-methoxyethyl-modified antisense oligonucleotide compound. It effectively inhibits transthyretin (TTR) protein production by targeting the TTR RNA transcript, ...
T39809	PRMT5-IN-14	PRMT5-IN-14
	PRMT5-IN-14 is a PRMT5 inhibitor to treat cancer, sickle cell, and hereditary persistence of foetal hemoglobin (HPFH) mutations.
T61403	Plasma kallikrein-IN-3
	Plasma kallikrein-IN-3, a potent inhibitor ( IC 50 : 0.15 μM) of plasma kallikrein, finds application in research related to hereditary angioedema, diabetic macular edema, and diabetic retinopathy [1].
T76710	Lanadelumab
	Lanadelumab (SHP643), a humanized IgG1 monoclonal antibody, targets plasma kallikrein (pKal) with a K i value of 0.12 nM, effectively inhibiting both its free form and its interaction with high molecular weight kininogen...
T38804	Patisiran sodium
	Patisiran sodium is a double-stranded small interfering RNA (siRNA) that selectively targets a specific sequence within the transthyretin (TTR) messenger RNA (mRNA). By doing so, it effectively blocks the hepatic synthes...
T72337	Plasma kallikrein-IN-2
	Plasma kallikrein-IN-2, a potent inhibitor of plasma kallikrein (PKal) with an inhibitory concentration (IC) 50 value of 0.1 nM, is utilized in research targeting hereditary angioedema, diabetic macular edema, and diabet...
T35869	Berotralstat
	Berotralstat (BCX7353) is a second-generation, synthetic, and orally active plasma kallikrein inhibitor with low toxicity. It is highly specific and effectively used in the research of hereditary angioedema (HAE) attacks...
T36055	Nitisinone-13C6	Nitisinone-13C6
	Nitisinone-13C6is intended for use as an internal standard for the quantification of nitisinone by GC- or LC-MS. Nitisinone is an inhibitor of 4-hydroxyphenylpyruvate dioxygenase (HPPD), which converts 4-hydroxyphenylpyr...