ANAT inhibitor-2 is a ANAT inhibitor for canavan disease, with an IC50 value of 20 μM.

(Z)-PUGNAc is a potent O-GlcNAcase inhibitor. (Z)-PUGNAc is a vastly more potent inhibitor of O-GlcNAcase than the E form.

(±)13-HpODE (13-hydroperoxylinoleic acid) is a racemic mixture of hydroperoxides, which is produced by the oxidation of linoleic acid by lipoxygenase.

3-Phenylpropyl isothiocyanate has a stronger inhibitory effect on N-nitrosomethyl-benzylamine (NMBA) tumorigenesis. 3-Phenylpropyl isothiocyanate has chemopreventive effects.

1,4,7-Triazonane (1,4,7-Triazacyclononane), an intermediate in the synthesis of 1,4,7-trifunctionalized derivatives, is a possible reagent for compleximetric titrations with high cation-binding selectivity.

2-Bromohexadecanoic acid (2-BP, 2-bromopalmitate) is a nonmetabolisable analogue of palmitate and acts as a palmitoylation inhibitor. 2-Bromohexadecanoic acid can directly and irreversibly inhibit the palmitoyltransferase activity of all DHHC (Asp-His-His-Cys) proteins.

Dideoxyadenosine is konwn as 2′,3′-Dideoxyadenosine (ddA), a specific adenylyl cyclase inhibitor, is useful in biological process and pathway studies involving adenylyl cyclase activity and cAMP pool modulation.

Ammonium chloride is an inorganic compound and can be used for establishing the rat model of renal calcium oxalate calculus.

Cobomarsen (MRG-106) is an oligonucleotide inhibitor of miR-155. Cobomarsen inhibits multiple gene pathways associated with cell survival (including JAK/STAT, MAPK/ERK and PI3K/AKT). Cobomarsen can be used for the research of B-cell lymphoma.

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.

Revusiran (ALN-TTRSC) is a 1st-generation short interfering RNA, which directed against transthyretin (TTR) mRNA. Revusiran can be used for transthyretin (TTR)-mediated amyloidosis research.

Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid (siRNA) agent. Vutrisiran can be used for transthyretin (TTR)-mediated amyloidosis research.

IONIS-MAPTRx (BIIB080) is the first Tau-lowering antisense oligonucleotide (ASO). IONIS-MAPTRx has the potential for the research of Alzheimer Disease.

Lexanersen (WVE-120102) is an antisense oligonucleotide used for the study of Huntington's disease.

Suvodirsen (WVE-210201) is a oligonucleotide. Suvodirsen has the potential for study Duchenne muscular dystrophy (DMD).

Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran can be used for the research of acute intermittent porphyria.

Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD).

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.

Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD).

Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD).

Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research.

Lumasiran sodium, an investigational RNA interference (RNAi) therapeutic agent, reduces hepatic oxalate production by targeting glycolate oxidase. Lumasiran sodium reduces urinary oxalate excretion, the cause of progressive kidney failure in primary hyperoxaluria type 1 (PH1) .

Tadnersen (BIIB078), an antisense oligonucleotide (ASO), selectively targets C9ORF72 transcript variants 1 and 3 that carry the expansion.

SRP-5051 is a next-generation antisense oligonucleotide of peptide phosphorodiamidate morpholino oligomer (PPMO). SRP-5051 targeting exon 51 skipping in Duchenne muscular dystrophy (DMD).

IONIS-DNM2-2.5Rx (DYM101) is an antisense agent targeting dynamin 2. IONIS-DNM2-2.5Rx has the potential for the research of centronuclear myopathy (CNM).

Simufilam (PTI-125) (hydrochloride) is a low toxicity, orally active filamin A (FLNA) activator. Simufilam (hydrochloride) preferentially binds altered FLNA and restores its native conformation, restores receptor and synaptic activities, reduces its a7nAChR/TLR4 associations and downstream pathologies. Simufilam (hydrochloride) can be used for the research of Alzheimer's disease.

PTPN22-IN-1 is a potent PTPN22 inhibitor (IC50=1.4 µM; Ki=0.50 µM). PTPN22-IN-1 exhibits >7-10 fold selectivity for PTPN22 over similar phosphatases. PTPN22-IN-1 augments antitumor immune responses. From WO2021007491A1 compound L-1.

Nonanal is a saturated fatty aldehyde with antidiarrhoeal activity.

Piperitone is as a powerful repellent and antiappetent agent. Piperitone is very toxic to Cymbopogon schoenanthus (C. schoenanthus) adults, newly laid eggs and to neonate larvae. Insecticidal activity.

Tyrosylleucine (Tyr-Leu, YL) TFA, an orally active dipeptide, exhibits a potent antidepressant-like activity.