GLN-1062 (Memogain) is an inactive pro-drug of galantamine, the latter being a plant alkaloid approved for the treatment of mild to moderate Alzheimer's disease.

MM-3122 is a selective inhibitor of host cell serine protease TMPRSS2 (transmembrane protease serine 2) with IC50 of 0.34 nM against recombinant full-length TMPRSS2 protein.

WAY-296817 is casein kinase 1 (CK1) inhibitor and can be used for research on the treatment or prevention.

CHMFL-PI4K-127 (compound 15g) is an orally active, potent and high selective PfPI4K (Plasmodium falciparum PI4K kinase) inhibitor, with an IC50 of 0.9 nM. CHMFL-PI4K-127 exhibits potent activity against 3D7 Plasmodium falciparum, with an EC50 of 25.1 nM. CHMFL-PI4K-127 shows antimalaria efficacy.

Exarafenib (RAF/KIN_2787) is a potent, orally active pan-RAF inhibitor. Exarafenib has anticancer activity by suppression of downstream MAPK pathway signaling. Exarafenib can be used for cancer research.

Tinlorafenib (PF-07284890) (compound 10) is an orally active BRAF kinase inhibitor, with IC50s of 4.25 and 2.7 nM for BRAFV600E/V600K respectively. Tinlorafenib demonstrates CNS penetration and can be used in the research of BRAF-associated malignant and benign tumors of the CNS as well as extracranial malignancies[1].

MTB-9655 is a novel, potent and selective Acetyl-Coenzyme A Synthetase 2 (ACSS2) inhibitor, which is currently in Phase I clinical investigation on patients with tumors with high levels of ACSS2.

dTAGV-1-NEG is a diastereomer and as a heterobifunctional negative control of dTAGV-1. dTAGV-1 is an FKBP12F36V-selective degrader.

BMS-986339 is an orally active, potent FXR agonist. BMS-986339 forms H-bond with His298 and ASN287 residues.

BE1218 is a liver X receptor (LXR) inverse agonist with IC50 values of 9 nM and 7 nM against LXRα and LXRβ, respectively.

TriGalNAc CBz is a GalNAc derivative and tri-GalNAc is an asialoglycoprotein receptor (ASGPR) ligand. TriGalNAc CBz can be used for mRNA drug delivery as well as lysosomal targeted chimerism (LYTAC) studies.

Nivasorexant is a potent orexin receptor antagonist.

PSB-06126 is a selective nucleoside triphosphate diphosphohydrolase (NTPDase) inhibitor, with the Ki values of 0.33 μM for rat NTPDase 1, 19.1 μM for NTPDase 2 and 2.22 μM for NTPDase 3, respectively.

TDI-011536 is a potent Lats kinase inhibitor, interrupts Hippo-Yap signaling and initiates the proliferation of lesioned heartmuscle cells. TDI-011536 can be used in studies of organ conservation and regeneration.

MS4-8107 is a potent and selective positive allosteric modulator of G protein-coupled receptor 68 (GPR68). MS48107 is selective for GPR68 over the closely related proton GPCRs, neurotransmitter transporters, and hERG ion channels. MS 48107 can readily cross the blood-brain barrier (BBB) in mice[1].

E3 ligase Ligand 19 is a ligand for E3 ubiquitin ligase. E3 ligase Ligand 19 can be connected to the ligand for protein (e.g., SMARCA BD ligand) by a linker to form PROTACs (e.g., PROTAC 1). PROTAC 1 is a partial degrader of SMARCA2 and SMARCA4[1]

BRD4/CK2-IN-1 is the first highly effective and oral active dual-target inhibitor of BRD4/CK2 (bromodomain-containing protein 4/casein kinase 2), with IC50s of 180 nM and 230 nM for BRD4 and CK2, respectively. BRD4/CK2-IN-1 has strong anticancer activity without obvious toxicities. BRD4/CK2-IN-1 induces apoptosis and autophagy-associated cell death in triple-negative breast cancer (TNBC)[1]

L202 is a novel ionizable lipid for RNA delivery.

Ensifentrine (RPL-554) is an inhaled first-in-class dual inhibitor of phosphodiesterase 3 (PDE3) and PDE4 with IC50s of 0.4 nM and 1479 nM, respectively. Ensifentrine has bronchoprotective and anti-inflammatory activities. Ensifentrine can be used for chronic obstructive pulmonary disease (COPD) research[1][2].

N-0385 is a novel highly potent inhibitor of TMPRSS2-like proteases (IC50 = 1.9 nM) that blocks SARS-CoV-2 VOCs (B.1.1.7, P.1, B.1.351 and B.1.617.2) and is broadly protective against infection and mortality in mice. N-0385 demonstrates potent inhibition of SARS-CoV-2 infection in Calu-3 cells with EC50 of 2.8 nM and a SI of > 10E6.

IDP-410 is a novel therapeutic peptide that target directly N-MYC protein. IDP-410 peptide acts by reducing the interaction of N-MYC with its partner MAX and favoring the degradation of the protein.

BLU-945(BLU945,BLU 945) is a highly potent and selective EGFR+/T790M/C797S (IC50 of 0.5 nM) and EGFR+/T790M (IC50 of 0.4 nM) inhibitor with >900-fold selectivity over EGFR WT (IC50 of 736.3 nM).

GNE-064 (compound 5) is a selective, oral and highly soluble inhibitor of the SMARCA4, SMARCA2 and PBRM1 bromodomains 5. GNE-064 inhibits SMARCA4 with an IC50 of 0.035 μM and inhibits SMARCA2 with an EC50 of 0.10 μM. GNE-064 possess Kds with 0.01, 0.016, 0.018 and 0.049 μM for SMARCA4, SMARCA2, PBRM1 bromodomains 5 and PBRM1 bromodomains 2, repectively. GNE-064 can be used as a chemical probe for the research of drug synthesis.

CHF-6366 is a potent M3 muscarinic antagonist and β2-adrenergic receptors agonist with pKi values of 10.4 and 11.4, respectively. CHF-6366 is also a weak calcium channel inhibitor (IC50~50 μM). CHF-6366 inhibits bronchoconstriction in guinea pigs.

BSP16 is a potent, orally active stimulator of interferon genes (STING) agonist. BSP16 can selectively stimulate the STING pathway. BSP16 can be used for the research of cancer.

VVD-118313 (compound 5a) is a potent, selective JAK1 inhibitor. VVD-118313 targets an isoform-restricted allosteric cysteine to block JAK1-dependent trans-phosphorylation and cytokine signaling. VVD-118313 can be used for research of cancer.

5-Ethynyluridine (5-EU) is a potent cell-permeable nucleoside can be used to label newly synthesized RNA. 5-Ethynyluridine can be used for isolation and sequencing of nascent RNA from neuronal populations in vivo. 5-Ethynyluridine can be used to identify changes in transcription in vivo in nervous system disease models.

AZD5462 is a selective oral allosteric relaxin family peptide receptor 1 (RXFP1) agonist with pEC50 of 7.8. AZD5462 has better kinetic solubility and shows much improved metabolic stability compared to AZ7976 in vitro.

Astrophloxine is a fluorescent imaging probe capable of targeting antiparallel dimers. Astrophloxine can be used to detect aggregated Aβ in brain tissue and cerebrospinal fluid samples of Alzheimer's disease (AD) mice.

GSK-626616 is a dual-specificity tyrosine-regulated kinase (DYRK3) inhibitor with an IC50 of 0.7 nM which is a potential therapy for the treatment of anemia.

CC-99282 (Golcadomide) is an orally active cereblon (CRBN) E3 ligase modulator (CELMoD). Ikaros and Aiolos are degraded potently and specifically by CC-99282, which co-opts CRBN.

RPT193 is an orally active inhibitor of CCR4, blocks the recruitment of Th2 inflammatory immune cells into inflamed tissues. RPT193 can be used for allergic inflammation in atopic dermatitis, asthma, and other diseases research.

Chiauranib (CS2164) selectively inhibits multiple kinase targets aurora B kinase (AURKB), colony-stimulating factor 1 receptor (CSF1R), and vascular endothelial growth factor receptor (VEGFR)/platelet-derived growth factor receptor (PDGFR)/c-Kit , thereby inhibiting the rapid proliferation of tumor cells, enhancing the antitumor immunity, and inhibiting tumor angiogenesis, to achieve the anti-tumor efficacy.

QN523 exhibits potent cytotoxicity in pancreatic cancer cell lines (IC50 of 0.11 μM in MIA PaCa-2 cells) and significantly delays growth of MIA PaCa-2 xenograft in NOD/SCID mice without systemic toxicity.

ML901 is a specific reagent that hijacks a single aminoacyl transfer RNA (tRNA) synthetases (aaRSs) in the malaria parasite Plasmodium falciparum. ML901 inhibits the growth of the malaria parasite Plasmodium falciparum in vitro and in animals but was not toxic to human cells.

ERX-41 is a potent therapeutic agent targeting lysosomal acid lipase A (LIPA) and shows the mechanism of action (disruption of protein folding and induction of ER stress) that may have utility in treating patients with TNBC or other multiple solid tumors.

CID661578 is a potent inducer of β-cell regeneration, which boosts protein synthesis and regeneration by blocking MNK2 from binding eIF4G in the translation initiation complex at the mRNA cap.

NKTP-3 is a potent, selective, biostable, and cell-permeable cyclic d-peptide that targets NRP1 and KRASG12D with binding affinity of 116.28 nM and 50.34 nM, respectively. NKTP-3 shows good biostability and a strong cellular uptake ability and further demonstrated its antitumor effect on lung cancer with the KRASG12D mutation in vitro and in vivo.

IGUANA-1 is an isoform-selective guanidinyl antagonists of ALDHs, which exhibits proteome-wide target specificity and selectively blocks the growth of colon cancer spheroids and organoids.

S3-15 selectively interrupts soluble RANKL-RANK interaction and without interfering with the membrane RANKL-RANK interaction. S3-15 exhibits anti-osteoporotic effects without causing immunosuppression.

JZ128 is a potential, irreversible, selective inhibitor of PKN3 with IC50  of 120 nM and shows a narrow kinome inhibition spectrum. JZ128 is used as a tool compound to identify novel potential PKN3 substrates.

Compound 16 (UNC-CA94) is a reversible inhibitor of PKN3 with IC50 of 14 nM. Compound 16 (UNC-CA94) is a potential tool compound to study the cell biology of PKN3.

Jun9-62-2R (dichloroacetamide) is a selective covalent Mpro inhibitor with IC50 of 0.43 μM and viral replication with EC50 of 2.05 μM in Caco2-hACE2 cells. Jun9-62-2R (dichloroacetamide) is highly selective toward Mpro and does not inhibit the host calpain I, cathepsins B, K, L, caspase-3, and trypsin. The widely used antibiotic chloramphenicol contains the dichloroacetamide, suggesting Jun9-62-2R might be tolerated in vivo.

D11 is a potent and selective inhibitor of protein kinase CK2 with Ki of 5 nM.

Comopound X1 (Xist) binds the non-coding RNA prototype Xist, specifically the RepA motif, with Kd of 0.4 μM. X1 binding reduces the conformational space of RepA, displaces cognate interacting protein factors (PRC2 and SPEN), suppresses histone H3K27 trimethylation, and blocks initiation of X-chromosome inactivation.

WC36 is a novel nonlipidic inhibitor of the lipid-SH2 domain interaction. WC36 is effective against RAS-mutated HL-60 AML cells that shows de novo resistance to entospletinib.

Sitagliptin, also known as MK-0431, is a potent inhibitor of DPP4 with an IC50 of 19 nM in Caco-2 cell extracts. Sitagliptin is believed to exert its actions in patients with type 2 diabetes mellitus by slowing the inactivation of incretin hormones. By increasing and prolonging active incretin levels, sitagliptin increases insulin release and decreases glucagon levels in the circulation in a glucose-dependent manner. Sitagliptin demonstrates selectivity for DPP-4 and does not inhibit DPP-8 or DPP-9 activity in vitro at concentrations approximating those from therapeutic doses.

Tematropium(CDDD3602) is a soft anticholinergics.

CDC801 is a potent and orally active phosphodiesterase 4 (PDE4) and tumor necrosis factor-α (TNF-α) inhibitor with IC50 of 1.1 μM and 2.5 μM, respectively.

APOL1-IN-1 is a apolipoprotein L1 (APOL1) inhibitor extracted from patent WO2020131807A1 compound 87. APOL1-IN-1 can be used for the research of focal segmental glomerulosclerosis (FSGS) and non-diabetic kidney disease (NDKD)[1].

ART812 is a DNA polymerase Polθ inhibitor with an IC50 value of 7.6 nM. ART812 has an IC50 value of 240 nM for cell based microhomology-mediated end joining (MMEJ)[1][2].

CCG-203769 is a selective G protein signaling (RGS4) inhibitor, which blocks the RGS4-Gαo protein-protein interaction in vitro with an IC50 of 17 nM.

Bucolome is a CYP2C9 inhibitor, used as an uricosuric agent or anti-inflammatory agent.

Carbamic acid, N-[2,4-dimethyl-6-(4-morpholinyl)-3-pyridinyl]-, phenylmethyl ester is a new invention for treating, one or more disorders or conditions wherein the dopaminergic system is disrupted, such as one or more disorders or conditions independently selected from the group consisting of: schizophrenia and other psychotic states; mood disorders ADHD; aggression; movement disorders.

GN13 is a cell-permeable, nucleotide-active-state-selective inhibitor of Gαs, with high selectivity over other G protein subfamilies. GN13 binds to Gαs/GNP with KD of 0.19 μM.

GD20 is a cell-permeable, nucleotide-inactive-state-selective inhibitor of Gαs, with high selectivity over other G protein subfamilies. GD20 inhibits Gαs steady-state GTPase activity with IC50 of 1.15 μM.

(R)-69 is a 5-HT2AR partial agonists with EC50 of 41 nM, and shows 4.6-fold/29-fold selectivity over 5HT2BR/5HT2CR. (R)-69 has substantial brain permeability in mouse pharmacokinetic studies and dose not possess locomotor-stimulating or reinforcing activity.

(R)-70 is a 5-HT2AR partial agonists with EC50 of 110 nM, and shows 6.4-fold/51-fold selectivity over 5HT2BR/5HT2CR. (R)-70 has substantial brain permeability in mouse pharmacokinetic studies and dose not possess locomotor-stimulating or reinforcing activity.

Antitubercular agent-30 is an antibacterial agent against Mycobacterium tuberculosis (MIC=50 μg/mL). Antitubercular agent-30 has little cytotoxic effect on murine macrophage cells (LD85=~100 μg/mL).

SphK1&2-IN-1 is a SphK inhibitor targeting to SphK1 and SphK2. SphK1&2-IN-1 has thermal stability.

c-Met-IN-15 (compound S3) is a c-Met kinase inhibitor. c-Met-IN-15 inhibits c-Met kinase activity of 21.1% at the concentration of 10 μM.

WAY-641966 is a anti-prion agent.

WAY-320461 is a FAK inhibitor.

ESI-08 is a potent and selective EPAC antagonist, which can completely inhibit both EPAC1 and EPAC2.

5-HT3 antagonist 5 is a quinoxalin-2-carboxamide compound, a 5-HT3 receptor antagonist. 5-HT3 antagonist 5 exerts antagonism on 5-HT3 agonist and 2-methyl-5-HT, and shows anti-depressant effect in mice[1].

PKCiota-IN-2 is a potent PKCiota (PKC-ι) inhibitor with an IC50 of 2.8 nM. PKCiota-IN-2 also inhibits PKC-α and PKC-ε with IC50s of 71 nM and 350 nM, respectively.

TRPM4-IN-2 (NBA) is a potent transient receptor potential melastatin 4 (TRPM4) inhibitor with an IC50 value of 0.16 μM. TRPM4-IN-2 can be used for researching prostate cancer and colorectal cancer[1][2].

Lateritin is a potent inhibitor of acyl-CoA:cholesterol acyltransferase (ACAT), isolated from the mycelial cake of Gibberella lateritium IFO 7188[1]. Lateritin also inhibits the growth of a mini-panel of human cancer cell lines, gram-positive bacteria, and Candida albicans[2].

UT-34 is a potent, selective and orally active second-generation pan-androgen receptor (AR) antagonist and degrader with IC50s of 211.7 nM, 262.4 nM and 215.7 nM for wild-type, F876L and W741L AR, respectively. UT-34 binds to ligand-binding domain (LBD) and function-1 (AF-1) domains and requires ubiquitin proteasome pathway to degrade the AR. UT-34 has anti-prostate cancer efficacy[1][2].

TH-Z145, a lipophilic bisphosphonate, is a FPPS inhibitor (IC50: 210 nM).

UCB7362 is an orally active and potent antimalarial plasmepsin X (PMX) inhibitor, with an IC50 of 7 nM. UCB7362 inhibits parasite growth.

RP-6685 is a potent, selective and orally active DNA polymerase theta (Polθ) inhibitor with an IC50 value of 5.8 nM (PicoGreen assay). RP-6685 shows antitumor efficacy in mouse tumor xenograft model.

UNC1021 is a selective L3MBTL3 inhibitor with an IC50 of 0.048 μM.

NDI034858 is a TYK2 inhibitor, target TYK2 JH2 domain with binding constant Kd of <200 pM.

BI-0474 is a potent KRASG12C inhibitor with an IC50 value of 7.0 nM for the GDP-KRAS::SOS1 protein-protein interaction. BI-0474 exhibits good anti-proliferative activity against NCI-H358 cells carrying the G12C mutation. BI-0474 also shows good anti-tumour activity in non-small cell lung cancer xenograft models.

GDC-2394 is an orally active and selective NLRP3 inhibitor, and also inhibits IL-1β with IC50s of 0.4 μM (human IL-1β) and 0.1 μM (mouse IL-1β). GDC-2394 inhibits NLRP3-induced caspase-1 activity without inhibiting NLRC4-dependent inflammasome activation.

Tri-GalNAc(OAc)3 TFA is a tri-GalNAc ligand that can be used for the synthesis of GalNAc-LYTAC. GalNAc-LYTAC engages the asialoglycoprotein receptor for targeted protein degradation. tri-GalNAc: triantenerrary N-acetylgalactosamine; LYTAC: lysosome-targeting chimera.

4A3-SC8 is a novel Ionizable amino lipid for RNA delivery.The CRISPR-Cas9 gene editing system has been a hotspot in the field of gene therapy, especially the gene correction induced by homology-directed repair (HDR). However, its application has various obstacles, such as large molecular weight, poor stability, off-target risk, and the complexity of codeliver multiple genes. Farbiak et al. established a novel ionizable lipid library consisting of four distinct amine cores (3A3, 3A5, 4A1, 4A3) and nine peripheries with different alkyl chain lengths (SC5-SC14), and screened out a class of iLNPs with ability of encapsulating Cas9 mRNA, sgRNA and donor DNA simultaneously. The delivery efficiency (quantified by luciferase mRNA expression) and iLNPs toxicity were evaluated with three different cell lines (HEK293T, HeLa, and IGROV-1), indicating the formulation containing 4A3-SC8 was the best. 4A3-SC8 iLNPs successfully induced HDR in HEK293 cells by one-pot delivery of Cas9 mRNA, sgRNA, and the correct ssDNA template. Confocal microscopy imaging showed that a portion of blue fluorescence in cells was corrected to green fluorescence. Furthermore, the nucleic acid ratios of Cas9: sgRNA: donor DNA loading in iLNPs at a ratio of 2:1:3 could maximize the HDR efficiency with the editing efficiency up to 23%, which breaks through the current bottleneck of HDR efficiency of only 1–5%. This progress is undoubtedly an important advance in the gene therapy field to cure diseases caused by genetic mutations.

BAY-4931 is a potent covalent PPARγ inverse-agonist with IC50 of 0.17 nM and could be a novel tool for probing the in vitro biology of PPARγ inverse-agonism.

BAY-0069 is a potent covalent PPARγ inverse-agonist with IC50 of 0.22 nM and could be a novel tool for probing the in vitro biology of PPARγ inverse-agonism.

BAY-069 is a potent dual BCAT1/BCAT2 inhibitor with IC50 of 31 nM/153 nM. BAY-069 displays high cellular activity in U-87 MG and MDA-MB-231 cell lines.

ATX-001 is a novel ionizable cationic lipid compound for RNA delivery.

A12-Iso5-2DC18 is a novel amine containing lipid can be used for mRNA delivery, activate the stimulator of interferon genes (STING) pathway, and exhibit anti-tumor immunity.

Adaptaquin is an inhibitor of hypoxia-inducible factor prolyl hydroxylase 2 (HIF-PHD2), with an IC50 of 2 μM. Adaptaquin can inhibit lipid peroxidation and maintain mitochondrial function[1][2].

(E/Z)-ZINC09659342 is an inhibitor of Lbc-RhoA interaction[1].

Sigma‑1 receptor antagonist 1 (compound 137) is a potent and selective sigma-1 receptor (σ1R) antagonist, with a high binding affinity to σ1R receptor (Ki = 1.06 nM). Sigma‑1 receptor antagonist 1 exhibits antineuropathic pain activity and acts as a promising agent for the treatment of neuropathic pain[1].

Sigma-1 receptor antagonist 2 is a potent and selective sigma 1 receptor (σ1 R) antagonist with Kis of 3.88 and 1288 nM for σ1 and σ2 receptor, respectively[1].

GSK3-IN-1 (compound 11) is a GSK-3 inhibitor with an IC50 value of 12 μM. GSK3-IN-1 can be used in the research of diabetes[1].

MYF-03-176 is an orally active and potent anticancer agent. MYF-03-176 shows strong antitumor efficacy in MPM mouse xenograft model via oral administration[1].

Dopamine D2 receptor agonist-2 (compound 36) is a potent dopamine D2 receptor biased agonism ligand with an Ki value of 11.2 nM. Dopamine D2 receptor agonist-2 can be used to research antipsychosis[1].

WAY-622024(Compound 5j) is a GPCR GPR109b (HM74) agonist, with a pEC50 value of 6.51. GPCR agonist-2 can be used for research of lipid disorders.

Olvanil (NE-19550)is an agonist of transient receptor potential vanilloid type 1 (TRPV1) channels with an EC50 of 0.7 nM.Analgesic[1].

PQM130, a Feruloyl-Donepezil Hybrid compound with brain penatration, is a multitarget drug candidate against the neurotoxicity induced by Aβ1-42 oligomer (AβO) and shows anti-inflammatory activity. PQM130 acts as a neuroprotective compound for anti-AD drug development[1].

PKR-IN-C51(compound 51) is an ATP-competitive double-stranded RNA-activated protein kinase (PKR) inhibitor with an IC50 of 9 μM. PKR-IN-C51 inhibits intracellular PKR activation in a dose-dependent manner in primary mouse macrophages[1].

c-Fms-IN-13 (compound 14) is a potent FMS kinase inhibitor with an IC50 value of 17 nM. c-Fms-IN-13 can be used as an anti-inflammatory agent[1].

RLX-33 is a potent, selective and blood-brain barrier (BBB) penetrant relaxin family peptide 3 (RXFP3) antagonist, also blocks relaxin-3-induced ERK1/2 phosphorylation, with IC50 values of 2.36 μM for RXFP3, 7.82 and 13.86 μM for ERK1 and ERK2 phosphorylation, respectively. RLX-33 can block the stimulation of food intake induced by the RXFP3-selective agonist R3/I5 in rats. RLX-33 can be used for the research of metabolic syndrome[1].

ISR-IN-1 (Compound 48) is an inhibitor of the integrated stress response.

ISR-IN-2 (Compound 47) is an inhibitor of the integrated stress response .

Methoxy-X04 is a fluorescent dye that crosses the blood-brain barrier and selectively binds to beta-pleated sheets found in dense core amyloid Aβ plaques.

BRD-7586(BRD 7586) is a cell-permeable small molecule inhibitor of SpCas9 with EC50 of 6.2  µM and 5.7  µM in the eGFP-disruption and HiBiT-knock-in assays, respectively. BRD7586 specifically engages SpCas9 but not Cas12a in cells and enhances SpCas9 specificity at multiple loci.

Coelenterazine h is a derivative of Coelenterazine. Coelenterazine h is more sensitive to Ca2+ than is the native complex, thus providing a valuable tool for measuring small changes in Ca2+ concentrations.

Centanamycin alkylates the A-T-rich minor groove of the DNA and thereby blocks DNA replication. Centanamycin potently inhibits replication of human cytomegalovirus (HCMV), mouse CMV (MCMV), and herpes simplex virus-2 (HSV-2) in vitro. Centanamycin-attenuated whole virion MCMV elicits a robust serum neutralizing antibody response and protects the mice from live-virus challenge.

ATX-081 is a novel ionizable lipid for rna delivery.

ATX-083 is a novel ionizable lipid for rna delivery.

ATX-084 is an ionizable cationic lipids for RNA or Gene delivery.

ATX-087 is a novel ionizable lipid for rna delivery.

304O13 is a novel Biodegradable lipidoid for RNA delivery.

CSL3 is a novel cationic switchable lipid, pH-triggered molecular switch for siRNA delivery.In vivo factor VII silencing efficiency of the CSL3-based formulations was compared to that of the CSL4-based formulation (Fig. 5E). CSL3-based LNP were found to demonstrate a dose–response knockdown, resulting in significant silencing efficacy (∼50% silencing) at 1 mg kg−1.These results confirm the crucial role of the pH-sensitive conformational change of CSL3 in the in vivo transfection efficacy, in agreement with the in vitro results. The in vivo silencing efficacy of the CSL3 LNP may be further improved by lowering the molar percentage of DMG-PEG2000 in the formulation11,33 or by using combinatorial approaches to enhance the potency of the CSL3 lipid structure as well as the blood stability of the LNP (currently under investigation).

UT-143 is a selective androgen receptor irreversible covalent antagonist and inhibits AR transactivation with IC50 of 150 nM. UT-143 covalently and selectively binds to C406 and C327 in the AF-1 region and inhibits PCa cell proliferation and tumor growth.

A947 is a potent and selective SMARCA2 PROTAC with DC50 of 39 pM in W1573 cells. A947 shows 28-fold selectivity over SMARCA4 and notably exhibits no unexpected off-target effects at high concentration.

Fomivirsen (ISIS-2922 free base) is an antisense 21 mer phosphorothioate oligonucleotide. Fomivirsen is an antiviral agent that is used cytomegalovirus retinitis (CMV) research, incluiding in AIDs. Fomivirsen binds to and degrades the mRNAs encoding CMV immediate-early 2 protein, thus inhibiting virus proliferation.

DVR-01 is a HBV inhibitor with EC50 values of 1.7 and 1.6 μM in AML12HBV10 and HepDES19 cells, respectively. DVR-01 shows antiviral activity against drug-resistant HBV mutants with EC50s of 2.403-3.273 μM. DVR-01 can be used for the research of HBV infection and related diseases.

SpdSyn binder-1 is a weak binder, which binds in the active site of plasmodium falciparum spermidine synthase. SpdSyn binder-1 can be used for the research of malaria.

MMRi62, a ferroptosis inducer targeting MDM2-MDM4 (negative regulators of tumor suppressor p53). MMRi62 shows a P53-independent pro-apoptotic activity against pancreatic ductal adenocarcinoma (PDAC) cells and induce autophagy. MMRi62 inducesferroptosis, resulting in a increase of reactive oxygen and lysosomal degradation of ferritin heavy chain (FTH1). MMRi62 also leads to proteasomal degradation of mutant p53, also inhibits orthotopic xenograft PDAC mouse model in vivo with high frequency mutation characteristics of KRAS and TP53.12.

Tabernanthalog is a water-soluble, non-hallucinogenic and non-toxic analogue of ibogaine. Tabernanthalog is a 5-HT2A agonist. Tabernanthalog is found to promote structural neural plasticity, reduce alcohol- and heroin-seeking behaviour, and produce antidepressant-like effects in rodents.

Upidosin (Rec 15/2739) is an α-1 adrenoceptor (α-1 AR) antagonist. Upidosin shows moderate selectivity for the α-1A AR subtype. Upidosin shows uroselectivity in urethra and prostate with a Kb value of 2-3 nM higher than in ear artery and aorta with a Kb value of 20-100 nM. Upidosin inhibits [3H]prazosin binding to cloned human α-1A adrenergic receptor. Upidosin can be used for the research of urethral obstruction.

JNJ10191584 (VUF6002) is an orally active and selective histamine H4 receptor antagonist with a Ki value of 26 nM. JNJ10191584 shows 540-fold selectivity to H4 receptor over H3 receptor with a Ki value of 14.1 μM. JNJ10191584 inhibits chemotaxis of eosinophils and mast cells with IC50 values of 530 nM and 138 nM, respectively.

PF-07258669 is a melanocortin-4 receptor (MC4) antagonist. PF-07258669 can be used for the research of cachexia, anorexia, or anorexia nervosa.

CP-447697 is a lipophilic C5a receptor antagonist with an IC50 value of 31 nM. CP-447697 can be used for the research of inflammation.

BAY-390 is a selective, across species active and brain penetrating TRPA1 inhibitor. BAY-390 inhibits hTRPA1 FLIPR, hTRPA1 Ephys, rTRPA1 FLIPR and rDRG Ephys with IC50s of 16, 82, 63 and 35 nM, respectively.

TDI-11861 is second-generation soluble Adenylyl Cyclase (sAC, ADCY10) inhibitor with slow dissociation rates. TDI-11861 inhibits sAC cellular with an IC50 value of 5.5 nM.

Oct4 inducer-1 (compound OAC-3) is a potent Oct4 activator. Oct4 inducer-1 activates Oct4 and Nanog promoters and enhances induced pluripotent stem cells (iPSC) formation. Oct4 inducer-1 facilitates the reprogramming of cells by enhancing efficiency and shortening the reprogramming time.

DSPE-SPDP is a phospholipid molecule. DSPE-SPDP can orient itself to form lipid bilayer in water. DSPE-SPDP can be used for the research of various biochemical.

DSPE-MAL is a thiol reactive a phospholipid compound. DSPE-MAL has two saturated fatty acids and can self-assemble in water to form lipid bilayer. DSPE-MAL can be used to prepare liposomes as drug nanocarrier.

DSPE-N3 is a lipid. DSPE-N3 can be used for the research of various biochemical.

PBT 1033 (PBT 2) is an orally active copper/zinc ionophore. PBT 1033 restores cognition in mouse models of Alzheimer's disease (AD). PB 1033 also has antibacterial activity against Gram-positive bacteria.

ZINC00640089 is a specific Lipocalin-2 (LCN2) inhibitor. ZINC00640089 inhibits cell proliferation, cell viability and reduces AKT phosphorylation levels in SUM149 cells. ZINC00640089 has good potential for research in inflammatory breast cancer (IBC).

SRI 6409-94 is an orally active Ro 13-6298 analogue that is teratogenic. SRI 6409-94 serves as a molecular tool to study the effect of the three-dimensional configuration of retinol on teratogenic activity.

CFT7455 is an orally active zinc finger transcription factors (IKZF1) (Ikaros) and IKZF3 (Aiolos) degrader. It binds with high affinity to the cereblon E3 ligase (Kd of 0.9 nM). It is used in the treatment of multiple myeloma & non-Hodgkin’s lymphoma.

HBC620 is an analogue of HBC which is a GFP fluorophore-like synthetic dye that is nonfluorescent in solution, but strongly fluoresces upon forming tight complex with Pepper RNA aptamer. HBC-Pepper complex can be used to visualize RNA dynamics in live cells.

KCC-07 is a potent, selective and brain-penetrant inhibitor of MBD2 (methyl-CpG-binding domain protein 2) with anticancer activity. It prevents binding of MBD2 to methylated DNA and activates brain specific angiogenesis inhibitor 1 (BAI1) inducing anti-proliferative BAI1/p53/p21 signaling.

VT-1598 tosylate is an orally active and selective fungal inhibitor targeting CYP51. VT-1598 tosylate shows anti-fungal activity against C. auris.

FLX475 is a potent CCR4 antagonist that blocks regulatory T cells that interfere with effective antitumor immune responses and has antitumor activity.

ICeD-2 is a inducer of cell death, can induce HIV-1 infected cell kill. ICeD-2-mediated HIV-1 infected cell kill is dependent on HIV-1 protease activity. ICeD-2 potently blocks hydrolysis of Gly-Pro-AMC by dipeptidyl peptidase DPP8 and DPP9. ICeD-2 shows strong stabilization of DPP9 in PBMCs.

TUG-499 is a selective free fatty acid receptor 1 (FFAR1 or GPR40) (Free Fatty Acid Receptor) agonist with a pEC50 of 7.39. TUG-499 exhibits >100-fold selectivity over the related receptors FFA2, FFA3, and the nuclear receptor PPARγ and other diverse receptors, ion channels, and transporters. TUG-499 can be used for the research of type 2 diabetes.

MS8815 is a selective enhancer of zeste homolog 2 (EZH2) PROTAC degrader. MS8815 has inhibition activity for EZH2 with an IC50 value of 8.6 nM. MS8815 can be used for the research of triple-negative breast cancer (TNBC).

Tri-GalNAc-NHS ester is a LYsosome TArgeting Chimera (LYTAC) and a ligand of asialoglycoprotein receptor (ASGPR). ASGPR is a lysosomal targeting receptor specifically expressed on liver cells, for the degradation of extracellular proteins including membrane proteins. Tri-GalNAc-NHS ester can be used as a protein degrader and it can be used for the research of LYTAC.

BT5 is an irreversible inhibitor of SET domain of nuclear receptor-binding SET domain 1 (NSD1) histone methyltransferase.

GLPG3970 (compound 88) is a first-in-class SIK2/SIK3 inhibitor. GLPG3970 can be used for the research of inflammation and autoimmune disease.

DS69910557 is a potent, selective and orally activehuman parathyroid hormone receptor 1 (hPTHR1) antagonist. DS69910557 has antagonistic activity for PTHR1 with an IC50 value of 0.08 μM.

PF-249(PF06885249) is a potent, selective AMPK β1-containing complexes activator; increases the phosphorylation of the AMPK substrate ACC at S79 with EC50 of 296 nM, potently inhibits de novo lipogenesis (IC50=15 nM) in primary rat hepatocytes.

306-O12B-3 is an ionizable cationic lipidoid. It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of antisense oligonucleotides (ASOs). LNPs containing 306-O12B-3 localize to the liver after intravenous administration in mice. LNPs containing 306-O12B-3 in combination with other lipids encapsulating ASOs targeting the gene encoding proprotein convertase subtilisin kexin type 9 (PCSK9) decrease hepatic PCSK9 expression in mice. Intravenous administration of LNPs containing 306-O12B-3 and the cationic lipidoid NT1-O14B encapsulating ASOs against tau decreases tau expression and levels in mouse brain.

BI-7273 is a potent, selective, and cell-permeable BRD9 BD Inhibitor.

AA3-DLin is an ionizable cationic amino lipid (pKa = 5.8) that has been used in combination with other lipids in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA.LNPs containing AA3-DLin and encapsulating mRNA for the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) spike glycoprotein induce immunogenicity in mice.

PPZ-A10 is an ionizable cationic lipid.It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of siRNA and mRNA in vitro and in vivo. Intraperitoneal administration of LNPs containing PPZ-A10 and encapsulating an mRNA reporter preferentially accumulates in hepatic Kupffer cells and splenic macrophages in mice.

93-O17O is a chalcogen-containing ionizable cationic lipidoid. It has been used in the generation of lipid nanoparticles (LNPs). LNPs containing 93-O17O localize to the spleen after intravenous injection into mice.LNPs containing 93-O17O have been used for the delivery of Cre recombinase and ribonucleoproteins for genome editing in mice and for the intratumoral delivery of cGAMP to enhance cross-presentation of tumor antigens.

LIPID-10a-26 is an analogue of ALC-0315 with the head adjusted to imidazole. It is a novel cationic ionizable Lipid for RNA delivery.

BAMP-TK-12 is ROS‐degradable lipid used for gene/RNA delivery.

113-O16B is a disulfide bond-containing ionizable cationic lipidoid. It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA.

LIPID 14 is a novel ionizable lipid used for mRNA delivery.In 2021, Elia et al. used lipid 2 LNPs and lipid 14 LNPs to deliver mRNA encoding SARSCoV-2 human Fc-conjugated receptor binding domain (RBDhFc mRNA). While both lipid 274 LNP RBD-hFc mRNA and lipid 14 LNP RBD-hFc mRNA induced equal cellular and humoral responses in mice at an mRNA dose of 5 μg, only lipid 14 LNP RBD-hFc mRNA exhibited strong immunogenicity following intradermal administration. Both intradermal administration and intramuscular administration of lipid 14 LNPs could activate antigen presenting cells (APCs), thus inducing cellular responses.

PABK (para-azidobenzyloxycarbonyl lysine) is a site-specific small molecule-activated amino acids which could be installed into proteins in the zebrafish embryo and enable fast and tunable activation of the function of several enzymes.

Cephalofurimazine (CFz) is a NanoLuc substrate with improved brain performance and pairs with Antares luciferase produces greater than 20-fold more signal from the brain than the standard combination of D-luciferin with firefly luciferase.

UDP-GlcNAc Disodium Salt (UDP-α-D-N-Acetylglucosamine Disodium Salt) is a donor substrate of O-GlcNAc transferase (OGT).

UDP-GalNAc (UDP-N-acetyl-D-galactosamine) disodium is a sugar nucleotide and a substrate of EpsC115. EpsC115 is an exopolymeric substances (EPS) N-terminal deletion mutant with the residue 1-115 deletion. UDP-GalNAc UDP-GalNAc is the donor substrate of many N-acetylgalactosaminyltransferases, enzymes which transfer GalNAc from the nucleotide sugar to a saccharide or peptide acceptor.

IC8 is an ionizable cationic lipid. It has been used in combination with other lipids for the formation of lipid nanoparticles (LNPs). Immunization with severe acute respiratory coronavirus 2 (SARS-CoV-2) spike glycoprotein mRNA in IC8- and manganese-containing LNPs induces IgG responses to SARS-CoV-2 Delta and Omicron variants in mice.1 Administration of mRNA encoding B7-H3 X CD3 bispecific T cell engaging (BiTE) antibodies in IC8-containing LNPs reduces tumor growth in MV4-11 and A375 mouse xenograft models.

SSPalmM is an ionizable cationic disulfide-cleavable (SS-cleavable) proton-activated lipid-like material (SSPalm).

80-O16B is a disulfide bond-containing ionizable cationic lipidoid. It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of CRISPR complementary single-guide RNA (sgRNA) and Cas9 for genome editing in mice. LNPs containing 80-O16B conjugated to phenylboronic acid (PBA) and encapsulating an mRNA reporter increase luciferase reporter expression in HeLa cancer cells.2 LNPs containing 80-O16B conjugated to PBA and encapsulating p53 mRNA decrease the viability of DU145 prostate and SiHa and HeLa cervical cancer cells.

PL-SNS-032 conjugate 955 can potently degrade CDK9 in a ubiquitin-proteasome-dependent manner and is much more potent than SNS-032 against various tumor cells in vitro.

UDP-GlcNAz disodium is a substrate for UDP-GlcNAc:polypeptidyltransferase. UDP-GlcNAz serves as a sugar donor for the process catalyzed by the OGT enzyme and labels proteins through this process.

A066 is a lipid for RNA and vaccine delivery.A-066 is an analog of DODMA that contains a pyrrole head group. A-066 LNPs encapsulating TetR siRNA could induce significant antitumor efficacy in orthotopic hepatocellular carcinoma models.

DMAP-BLP is a lipid for RNA and vaccine delivery.DMAP-BLP exhibits optimized bilayer destabilizing and pKa properties leading to highly potent gene silencing in hepatocytes following IV administration that is similar to “gold standard” lipids such as DLinMC3-DMA.

Dimethyldioctadecylammonium bromide (DDAB) is a cationic surfactant that is used as an antimicrobial agent.

DiC14-amidine (amidine) is a nonphysiological, cationic lipid that forms stable liposomes under physiological pH and temperature.

DMRIE is a cationic lipid, suitable for transfecting DNA and RNA into eukaryotic cells, and is particularly effective for transfecting suspension cells (e.g., Jurkat) and other lymphoid-derived cell lines.

Dicetylamine is a biochemical

DlenDMA is a lipid for RNA and vaccine delivery. DLenDMA showed better siRNA transfection efficiency than DODMA.

GL 67 (N4-Spermine cholesteryl carbamate) is a cationic lipid. GL-67 can be used for nucleic acid agents and vaccines delivery, and gene transfection[1]. Genzyme Lipid (GL67), The cationic lipid N4 -cholesteryl-spermine is one of the most effective non-viral gene carriers comprising spermine as a headgroup that bond in a T-shape configuration to a cholesterol anchor.

BGTC is a non-amino acid cationic lipid. BGTC can be used for delivery of nucleic acids.