MTL-CEPBA is a small activating RNA targeting for upregulation of C/EBPα. MTL-CEPBA has anti-inflammatory and anti-cancer activity.

Lademirsen (SAR339375; RG-012) is a highly specific antisense oligonucleotide (ASO) targeting miR-21. Lademirsen has the potential for Alport nephropathy research.

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).

RG-101 is a hepatocyte targeted N-acetylgalactosamine conjugated oligonucleotide that antagonises miR-122. miR-122 is an important host factor for hepatitis C virus (HCV) replication.

Teprasiran (QPI-1002) is a small interfering RNA that temporarily inhibits p53-mediated cell death that underlies acute kidney injury (AKI).

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).

Fomivirsen sodium is an antisense 21 mer phosphorothioate oligonucleotide. Fomivirsen is an antiviral agent that is used cytomegalovirus retinitis (CMV) research, incluiding in AIDs. Fomivirsen sodium binds to and degrades the mRNAs encoding CMV immediate-early 2 protein (required for viral replication), thus providing bioactive effects for CMV retinitis by inhibition of virus proliferation.

Pegaptanib sodium is an RNA aptamer directed against vascular endothelial growth factor (VEGF)-165. Pegaptanib could be used for the study of neovascular age-related macular degeneration (AMD) .

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).

Tyrphostin 8 is a tyrosine kinase, with an IC50 of 560 μM for EGFR kinase. Tyrphostin 8 is also a GTPase inhibitor. Tyrphostin 8 can inhibit the protein serine/threonine phosphatase calcineurin (IC50=21 μM).

Ivaltinostat (CG-200745) formic is a potent HDAC inhibitor which has the hydroxamic acid moiety to bind zinc at the bottom of catalytic pocket. Ivaltinostat formic inhibits deacetylation of histone H3 and tubulin. Ivaltinostat formic induces the accumulation of p53, promotes p53-dependent transactivation, and enhances the expression of MDM2 and p21 (Waf1/Cip1) proteins. Ivaltinostat formic induces apoptosis.

RIP2 Kinase Inhibitor 4 is a potent and selective RIPK2 PROTAC. RIP2 Kinase Inhibitor 4 effectively degrades RIPK2 (pIC50 of 8) and inhibits the release of related TNF-α.

SKF83822 hydrobromide is a potent dopamine D1 receptor agonist. SKF83822 hydrobromide activates Gs/olf/adenylyl cyclase (AC)-coupled D1 receptors, but not phospholipase C (PLC)-coupled D1-like receptors.

ARD-61 is a highly potent, effective and specific PROTAC androgen receptor (AR) degrader. ARD-61 potently and effectively induces AR and progesterone receptors (PR) degradation in AR+ cancer cell lines. ARD-61 induces apoptosis and effectively induces tumor growth inhibition in the MDA-MB-453 xenograft model in mice.

MS67 is a potent and selective WD40 repeat domain protein 5 (WDR5) degrader with a Kd of 63 nM. MS67 is inactive against other protein methyltransferases, kinases, GPCRs, ion channels, and transporters. MS67 shows potent acticancer effects.

Zapnometinib (PD0184264), an active metabolite of CI-1040, is a MEK inhibitor, with an IC50 of 5.7 nM. Zapnometinib exhibits antiviral activity against influenza virus and antibacterial activities.

MRTX9768 hydrochloride is a potent, orally active PRMT5 inhibitor. MRTX9768 hydrochloride is a synthetic lethal-based inhibitor binding the PRMT5-MTA complex.

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.

Carbetocin acetate, an oxytocin (OT) analogue, is an oxytocin receptor agonist with a Ki of 7.1 nM. Carbetocin acetate has high affinity to chimeric N-terminus (E1) of the oxytocin receptor (Ki=1.17 μM). Carbetocin acetate has the potential for postpartum hemorrhage research. Carbetocin acetate can crosse the blood-brain barrier and produces antidepressant-like activity via activation of oxytocin receptors in the CNS.

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.

Co 101244 (PD 174494) hydrochloride is a NR2B-containing NMDA receptor antagonist.

BP 897 is a potent and partial dopamine D3 receptor agonist and a weak D2 receptor antagonist. BP 897 displays a high affinity at the dopamine D3 receptor (Ki=0.92 nM) and a 70 times lower affinity at the D2 receptor (Ki=61 nM). BP 897 exhibits selective inhibition of cocaine-seeking behavior.

Contezolid acefosamil sodium (MRX-4), a new and orally active oxazolidinone, is an antibiotic in study for complicated skin and soft tissue infections (cSSTI) caused by resistant Gram-positive bacteria. Contezolid acefosamil sodium (MRX-4) markedly reduces potential for myelosuppression and monoamine oxidase inhibition (MAOI).