AZ-8838 (AZ8838) is a potent, and selective PAR2 antagonist with Kd of 125 nM.

Novel potent inhibitor of the GRK2--​dependent bovine tubulin oxidation

Imidacloprid impurity 1 (IMP-2) is the impurity found during the synthesis of Ivermectin.

PD-089828 is an ATP competitive inhibitor of FGFR-1, PDGFR-β and EGFR (IC50s=0.15, 1.76, and 5.47 µM, respectively) and a noncompetitive inhibitor of c-Src tyrosine kinase (IC50=0.18 µM). PD-089828 also inhibits MAPK with an IC50 of 7.1 µM.

GSK-J1 is a potent and selective inhibitor of the H3K27 histone demethylases JMJD3 and UTX (IC50 = 60 nM for human JMJD3 in vitro).

PF9601N is a selective and potent monoamine oxidase B inhibitor that exhibit anti-Parkinsonian effects in several models of PD.

LDN192960 (LDN 192960) is a highly potent and selective DYRK2 inhibitor with IC50 of 13 nM toward DYRK2 at 50 uM ATP.LDN192960 suppresses Thr25 RPT3 phosphorylation in a dose-dependent manner in HEK293T cells transiently overexpressing DYRK2-FLAG, with maximal effects at 1-10 uM.LDN192960 does not inhibit any of the 130+ kinases including other CMGC kinase family members that are closely related to the DYRKs.LDN192960 binds to the ATP-binding pocket of DYRK2, also potently inhibits DYRK1A, DYRK3 and Pim with IC50 of <3, 122 and 10 nM, respectively.LDN192960 perturbs proteasome activity, induces cell death, and impedes proliferation and invasion on MDA-MB-468 cells.LDN192960 impedes MM progression and delays myeloma-mediated bone degeneration.LDN192960 induces cytotoxicity in bortezomib-resistant MM, both in cells and in vivo. significantly alleviates tumor burden in standard and PDX TNBC models.

CRANAD-2 is a difluoroboron-derivatized curcumins as near-infrared probe for in vivo detection of amyloid-beta deposits. Upon interacting with Abeta aggregates, CRANAD-2 undergoes a range of changes, which include a 70-fold fluorescence intensity increase, a 90 nm blue shift (from 805 to 715 nm), and a large increase in quantum yield. Moreover, this probe also shows a high affinity for Abeta aggregates (K(d) = 38.0 nM), a reasonable log P value (log P = 3), considerable stability in serum, and a weak interaction with albumin. After intravenous injection of this probe, 19-month-old Tg2576 mice exhibited significantly higher relative signal than that of the control mice over the same period of time.

A5-CE-C7-6 (3-tail analog) is a structural control compound derived from the standard 2-tail lipid(A5-CE-C7-6), with three carbonate alkyl chains linked to the A5 amine core.

LEE011 is an orally available cyclin-dependent kinase (CDK) inhibitor targeting cyclin D1/CDK4 and cyclin D3/CDK6 cell cycle pathway, with potential antineoplastic activity.

AO12 is a novel antioxidant ionizable lipid derived from SM-102 skeleton with para-hydroxyphenyl propionic acid side chains. Integrated into LNPs, it efficiently scavenges diverse reactive oxygen species including ·OH and ONOO⁻, shielding encapsulated mRNA from oxidative degradation. It retains fine LNP formulation features and cellular uptake capacity of conventional lipids, boosting in vivo mRNA translation. Applied for regenerative mRNA therapy and CRISPR gene editing against fibrosis and inflammatory disorders.

NEK7 degrader-1 is a NEK7 molecular glue degrader with a DC50 of 9  nM. NEK7 degrader-1 dose-dependently inhibits caspase-1 activity and IL-Ιβ release in macrophages in response to NLRP3 inflammasome activation. NEK7 degrader-1 can be used for autoinflammatory and autoimmune disorders like multiple sclerosis, neurodegeneraive diseases like Alzheimer's disease and cardiovascular and melabolic disorders like pericarditis and Type 2 diabetes research.

PL-40​​ is a ​​cardiolipin-mimetic ionizable lipid​​ engineered for high-efficiency, antibody-free mRNA delivery to T cells. PL 40 LNPs exhibit a mean particle size of ​​120 nm​​, zeta potential of ​​-5.19 mV​​, and >80% mRNA encapsulation efficiency, with excellent plasma stability (≤5% size change after 6h in serum). Cryo-TEM reveals ​​polyhedral nanoparticles​​ with phase-separated domains, while SAXS confirms tight mRNA packing (d-spacing: ​​~3 nm​​ vs. 6.64 nm in conventional LNPs). AFM demonstrates exceptional rigidity (high bending modulus), enabling T cell-selective uptake via actin-mediated endocytosis (>2× higher than ALC0315 LNPs).In primary human T cells, PL40 LNPs achieve ​​>90% transfection​​ at 0.5 μg mRNA dose and sustain >100× higher luciferase expression than benchmark lipids. When delivering circular RNA, they extend protein expression ​​>5 days​​ with superior spleen tropism (spleen:liver ratio = ​​2.63​​). Crucially, they reprogram T cells into functional CAR-Ts in vivo without antibody conjugation, evading exhaustion markers (no Tim-3/PD-1 upregulation). Therapeutically, PL40-based uPAR-targeted CAR mRNA reduces liver fibrosis (​​collagen↓50%​​, ALT↓50%) and rheumatoid arthritis severity (​​clinical scores↓60%​​) by clearing senescent cells. Humanized anti-uPAR CARs delivered via PL40 show near-complete cytotoxicity (>95%) against uPAR+ cells, underscoring clinical translatability.

MRTX-1719 is a potent first-in-class selective inhibitor of the PRMT5/MTA complex, with an IC50 of less than 10 nM in PRMT5/MTA MTAPDEL SDMA cells.

LLL12B is a selective STAT3 inhibitor that suppresses Th17 development, specifically inhibits STAT3 and suppresses Th17 differentiation and expansion.

Lipid 51 is a top-performing thioglycerol-based biodegradable ionizable lipid disclosed in PCT patent WO2026/147683 (Eli Lilly, filed Dec 16, 2025). Built with cleavable thioester linkages, it balances neutral surface charge at physiological pH and protonatable amines in acidic endosomes for efficient mRNA encapsulation and endosomal escape. Formulated into LNPs with DSPC, cholesterol and DMG-PEG2K, it exhibits favorable particle size, low PDI and high RNA loading efficiency. In intracerebroventricular (ICV) mouse tests targeting central nervous system (CNS), it delivers Cre mRNA to brain neurons far more potently than Lipid 1/2/3, with the highest tissue fluorescent signal among all tested candidates. It shows low cellular toxicity in vitro and robust CNS tropism, making it an optimal carrier for brain-targeted mRNA and CRISPR gene editing therapeutics.

DPNB-ABT594 is a nitrobenzyl-caged ABT594 and activates nAChRs containing the α4β2 subunits with good selectivity than the α7 subunit. DPNB-ABT594 can be used to map the distribution of nAChRs on neurons of the medial habenula (MHb) and helps to gain a deeper understanding of the nAChR‐mediated Ca2+ signalling in the MHb.

PPIL4 is a member of the cyclophilin family, characterized by its peptidyl-prolyl cis–trans isomerase activity, which facilitates the folding of target proteins through accelerated rotation around proline imidic peptide bonds. In addition to this isomerase function, PPIL4 harbors an RNA-recognition motif (RRM), implicating it in post-transcriptional processes. This dual-domain architecture suggests that PPIL4 not only participates in protein conformation quality control but also interfaces with the spliceosomal machinery and broader RNA metabolic pathways, potentially coupling protein folding events with co-transcriptional or post-transcriptional regulation.

AX6​​ is an ionizable lipid in the ​​F32 LNP​​ formulation, engineered by ReNAgade/Orna Therapeutics for targeted mRNA delivery to T cells. AX-6's unique ​​bridged bicyclic/polycyclic core​​ with a ​​tertiary amine group​​ enables pH-dependent protonation and endosomal escape, while ​​C14-C18 hydrophobic tails​​ (optionally branched/fluorinated) enhance bilayer stability and mRNA encapsulation. Demonstrating ​​exceptional T-cell tropism​​, AX6 achieves high transfection efficiency in CD4+/CD8+ T cells (validated in NHP/humanized models) with minimal toxicity. Compared to clinical benchmarks (SM-102, ALC-0315), its rigid core offers superior ​​serum stability​​ and ​​immune-cell specificity​​, positioning it as an ideal candidate for ​​CAR-T/NK therapies​​ and ​​next-gen vaccines​​. The F32 LNP system's proven efficacy (e.g., in vivo B-cell depletion) underscores AX 6's transformative potential for ​​cell engineering​​ and ​​immunotherapies​​.

Alogabat (example 8) is a GABAA α5 receptor positive allosteric modulators (PAMs) (extracted from patent WO2018104419A1).

MM0299 analog 13 (MM 0299 derivative 13) is a potent, selective, orally bioavailable, brain-penetrant inhibitor of lanosterol synthase (LSS, competition EC50=28.7 nM) with anti-proliferative activity.

CVL1 (C24)​​ is an ionizable lipid developed by CureVac for mRNA delivery, featuring a vitamin E (α-tocopherol) core linked via a thioether bridge to piperidine-based cationic headgroups. Its unique design enables pH-dependent charge switching (neutral at physiological pH, cationic in endosomes) for efficient mRNA encapsulation and endosomal escape. Formulated in lipid nanoparticles (LNPs) with DPhyPS and PMOZ4, CVL1 preferentially targets spleen and lymph node dendritic cells (DCs), enhancing antigen presentation and T-cell immunity. Key advantages include high mRNA encapsulation (>90%), stability under lyophilization, and reduced liver accumulation compared to PEGylated LNPs. In preclinical studies, CVL1-based LNPs induced robust CD8+/CD4+ T-cell responses and IgG2a-dominant antibody titers against tumor antigens (e.g., Trp2). With a particle size of 70–120 nm and low polydispersity (PDI <0.2), CVL1 balances delivery efficiency and biocompatibility, making it ideal for cancer and infectious disease vaccines requiring strong cellular immunity. Its degradable ester and thioether bonds further improve safety profiles.

KPT-6566 is a covalent PIN1 inhibitor selectively targets cancer cells by a dual mechanism of action.

L52715 is a novel ionizable lipid developed by Shanghai Vitalgen, used to deliver mRNA.

MIC2 is a set of multi-charged lipids with four tertiary amino nitrogen atoms (4N4T) which could be constructed and applied to form novel lipid nanoparticles. 4N4T-LNPs based on MIC2 exhibit much higher mRNA translation efficiency than the approved SM-102-LNPs. 4N4T-LNPs are successfully applied to DS mRNA vaccine and the vaccines worked well against SARS-CoV-2 and its variants, including Delta and Omicron.

LB42708 is an orally active farnesyltransferase (FTase) inhibitor with IC50 of 0.8, 1.2, and 2.0 nM toward H-ras, N-ras, and K-ras, respectively

C18 NC-TNP is a novel noncationic thiourea lipid without positively charged groups. It binds nucleic acids via hydrogen bonds instead of electrostatic attraction, avoiding cation-triggered systemic inflammation. Formulated into nanoparticles, it efficiently encapsulates mRNA, siRNA and plasmids, shows excellent serum tolerance and long-term liquid/lyophilized storage stability. It enters cells mainly through macropinocytosis, escapes endosomes intact to reduce nucleic acid degradation. In vivo, it targets spleen preferentially, induces robust long-lasting Th1-type cellular and humoral immunity with minimal organ toxicity, superior to SM102 LNPs for mRNA cancer vaccine delivery.

S-enantiomer of ARV-102. ARV-102 is a highly potent, orally bioavailable PROTAC that targets LRRK2 with a of 0.14 nM, designed to cross the blood-brain barrier to address neurodegenerative diseases. By hijacking the body’s ubiquitin-proteasome system, it achieves deep and sustained degradation of LRRK2 protein in both peripheral tissues and the central nervous system, offering a potentially superior therapeutic approach over traditional kinase inhibitors.

CPL500036 (CPL-500036) is a highly potent, selective and orally bioavailable PDE10A inhibitor with IC50 of 35 nM.CPL500036 is highly selective for PDE10A over other PDEs and common off-targets.CPL500036 effectively penetrates the brain where increases cAMP/cGMP levels and phosphorylates effector proteins like AMPA subunits.CPL500036 reduced sensorimotor deficits in 6-OHDA-lesioned rats.Acute and chronic CPL500036 did not reduce effects of L-DOPA in sensorimotor tests.Chronic CPL500036 treatment did not decrease L-DOPA-induced contralateral rotation.

GP1 precursor has high affinity to GPIIb/IIIa receptors of activated platelets. GP1 precursor can be radiolabeled with [18F] and used as a PET Tracer for visualizing active platelet aggregation at the molecular level. GP1 precursor can be used for the detection of Thrombi.