Fosaprepitant, also known as MK0517, is an antiemetic drug, administered intravenously. It is a prodrug of aprepitant. Fosaprepitant was developed by Merck & Co. and was approved. It is a prodrug of Aprepitant. It aids in the prevention of acute and delayed nausea and vomiting associated with chemotherapy treatment. Fosaprepitant is a weak inhibitor of CYP3A4, and aprepitant, the active moiety, is a substrate, inhibitor, and inducer of CYP3A4

T0080 is a FPR-1 antagonist. T0080 reduces the cell apoptosis, inhibits ROS production and pro-inflammatory cytokines (TNF-α and IL-1β) from plaque macrophages, which attenuates atherosclerotic progression in ApoE−/− mice.

1A7B13 is a top-performing "tripod-like" lung-targeting (LuT) lipid. It forms lipid nanoparticles (LNPs) that, after intravenous injection, deliver genetic medicines (like mRNA and CRISPR-Cas9) to the lungs with over 90% selectivity, particularly favoring epithelial cells. Compared to the benchmark DOTAP LNPs, 1A7B13 LNPs achieve a 25.5-fold increase in mRNA delivery and a 9.2-fold improvement in gene-editing efficiency within the lungs. Its therapeutic potential was demonstrated by successfully delivering IL-10 mRNA to treat acute lung injury in mice.

MitoCKi (Creatine kinase inhibitor CKi-26) is a selective, covalent inhibitor of creatine kinases (CKs), inhibits recombinant creatine kinase B (CKB) phosphotransfer activity with IC50 of 185 nM, depletes creatine phosphagen energetics in cells.

GSK 525762A is a potent small molecule inhibitors that disrupt the function of the BET family of bromodomains (Brd2, Brd3, and Brd4).

A novel potent, selective, allosteric and orally active dopamine D1 receptor potentiator with Kb of 26 nM.

GSK-3923868 (PI4KB inhibitor 30) is a potent, selective, slow-dissociating PI4KB (PI4kIII-beta) inhibitor with pKi of 9.8, >1000-fold selectivity over PI4KA, inhibits HRV viral replication in COPD BECs.

T26A is a competitive prostaglandin transporter (PGT) inhibitor (Ki=378 nM). T26A is promising for research of prostaglandin signaling and inflammatory diseases (e.g., glaucoma, pulmonary hypertension) .

HQY426 is a specific, orally bioavailable inhibitor of pre-messenger RNA endonuclease cleavage and polyadenylation specificity factor 3 (CPSF3) with binding IC50 of 0.32 uM, exhibits potent antitumor activity.

LDN-0130436 is a novel TDP-43::GFP aggregation inhibitor.

IBS007125 is a small molecule inhibitor of c-Maf, inhibits multiple myeloma proliferation by targeting cMaf transcriptional activity.

KDOBA67 is a novel cell-permeable inhibitor of H3K27 lysine demethylase KDM6A/B, inhibits TBXT expression chordoma cell lines.KDOBA67 displayed in vitro inhibitory activity in the low micromolar range with IC50 values of 2 to 5 uM in various chordoma cell lines, leading to induction of apoptosis.H3K27 demethylase inhibition via KDOBA67 alters chromatin state at TBXT and inhibits its expression.TBXT was a KDM6A/B target gene, and chromatin changes at TBXT following KDOBA67 treatment were associated with a reduction in TBXT protein levels.KDOBA67 treatment downregulated expression of a network of transcription factors critical for chordoma survival and upregulated pathways dominated by ATF4-driven stress and proapoptotic responses.

H5T5 is a leading ionizable lipid nanoparticle (LNP) formulation optimized for in vivomRNA delivery, featuring a pKa of 6.51, a size of ~154 nm, and a narrow polydispersity index (PDI) of 0.05. It demonstrated superior in vitromRNA transfection efficiency in primary immune cells, such as bone marrow-derived macrophages. Following intravenous administration, H5T5 exhibits precise organotropism, predominantly targeting the spleen and bone marrow, where it effectively delivers mRNA to a broad spectrum of immune cells, including macrophages, dendritic cells, T cells, B cells, and NK cells. This capability enables its core application: the in vivogeneration of "pan-CAR" immune cells. When loaded with anti-HER2 CAR mRNA, the H5T5-based therapy achieved potent tumor regression and prolonged survival in multiple solid tumor models. Preliminary safety assessments indicated a manageable cytokine profile and no significant organ toxicity, positioning it as a promising platform for in vivocell engineering.

A1F5C5 is a core fluorinated ionizable lipid that forms the basis of the F5-LNP platform. Its key biological functions are multifaceted. Primarily, it enables efficient, targeted mRNA delivery in vivo. Following intravenous administration, F5-LNPs exhibit a strong tropism for the spleen and tumor sites, successfully transfecting over 70% of splenic macrophages and more than 20% of tumor-infiltrating macrophages. This allows for in situ cell engineering. Beyond delivery, A1F5C5 possesses intrinsic immunostimulatory activity. It promotes the maturation and activation of antigen-presenting cells (e.g., upregulating CD80/86 on dendritic cells) and enriches immune-related pathways like "cytokine-cytokine receptor interaction." Mechanistically, its unique 5-fluorine (F5) configuration confers superior membrane fusion capability, which is critical for efficient endosomal escape and cytosolic mRNA release. Therapeutically, when loaded with CAR mRNA, it serves as a platform for in vivo generation of CAR-macrophages (CAR-M). These CAR-M cells phagocytose tumors, reprogram the tumor microenvironment by shifting macrophages to an M1 phenotype, and activate CD8+ T cells. Notably, this approach synergizes powerfully with anti-PD-L1 therapy, achieving complete tumor regression in preclinical models.

Cyclovirobuxine D (CVB-D) is the main active component of the traditional Chinese medicine Buxus microphylla. Cyclovirobuxine D induces autophagy and attenuates the phosphorylation of Akt and mTOR. Cyclovirobuxine D inhibits cell proliferation of gastric cancer cells through suppression of cell cycle progression and inducement of mitochondria-mediated apoptosis. Cyclovirobuxine D is beneficial for heart failure induced by myocardial infarction.

P3B is a biodegradable ionizable lipid engineered to function as a highly efficient delivery vehicle for genome-editing machinery (e.g., CRISPR-Cas9 and adenine base editors) specifically to the central nervous system (CNS). Its primary function is to encapsulate and transport large mRNA payloads across the brain-CSF interface following intrathecal administration, enabling robust and widespread gene editing in neurons and astrocytes across multiple brain regions, including the hippocampus, cortex, and thalamus. Notably, it facilitates precise single-nucleotide correction via base editing. Its targeting is intrinsically achieved by the intrathecal injection route, which localizes the nanoparticles within the cerebrospinal fluid, coupled with an optimized formulation that promotes efficient uptake and activity within CNS parenchyma while minimizing off-target exposure in peripheral organs.

MN132.0262 is a small molecule, dual inhibitor of A3G (APOBEC3G, IC50=20 uM).

MN256.0102 is a small-molecule APOBEC3G DNA cytosine deaminase inhibitor with IC50 of 3.9 uM.

BiP-20 is a branched ionizable phospholipid identified as a lead compound for efficient hepatic mRNA delivery.BiP-20 is a novel, efficient, and safe liver-targeted LNP delivery vehicle. With an ideal pKa of 6.56, it achieves highly efficient liver targeting and endosomal escape primarily through the ApoE/LDL-R pathway. It demonstrates exceptional performance in gene editing at very low doses: for CRISPR-Cas9-mediated editing of TTR, a 10 μg dose achieved ~64% efficiency, which is 8-fold higher than the clinical benchmark lipid LPO1. In Prime Editing targeting the PCSK9 gene, its efficiency (4.30%) also significantly surpassed that of MC3, SM102, and LPO1. Furthermore, it mediates a 5.9-fold increase in hepatic protein expression compared to MC3. Safety assessments indicate it does not induce liver function abnormalities, showing strong therapeutic potential.

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.

DMG-PEG-Mannose is a specialized functionalized lipid used primarily for the targeted delivery of lipid nanoparticles (LNPs) and liposomes to cells expressing mannose receptors, such as macrophages and dendritic cells. It combines a 1,2-dimyristoyl-sn-glycerol (DMG) lipid anchor with a polyethylene glycol (PEG) spacer and a terminal mannose sugar moiety.

VL422 is a novel ionizable cationic lipid, as a high-performance "molecular engine" for next-generation Lipid Nanoparticles (LNPs), specifically engineered for the precise delivery of CRISPR base editors and mRNA. Its sophisticated chemical architecture is designed to remain neutral in systemic circulation for enhanced safety, while rapidly protonating within the acidic cellular environment to trigger efficient endosomal escape and cargo release. Validated by groundbreaking research in liver-targeted gene silencing, VL422 has become a critical benchmark molecule for developing permanent, transformative therapies for cardiovascular and metabolic diseases.

UNC10013 is a SETDB1 allosteric modulator that forms a covalent bond with Cys385 in the 3TD domain, exhibiting negative allosteric regulatory activity. It has a kinact/KI value of 1.0 × 106 M-1*s-1. UNC10013 effectively disrupts SETDB1-mediated Akt methylation and holds potential value for research in cancer and neurodegenerative diseases.

A28-C6B2 is a biodegradable ionizable lipid specifically engineered for spleen-targeted delivery. Through its unique branched structure, it bypasses hepatic uptake to achieve highly efficient transfection of F4/80+ macrophages and CD11c+ dendritic cells within the spleen. This lipid remains neutral in the bloodstream to minimize non-specific interactions, while undergoing protonation in the acidic environment of the endosome to facilitate nucleic acid release, thereby significantly enhancing the potency of mRNA vaccines and immunotherapies.

AF12198 is a potent, selective and specific peptide antagonist for human type I interleukin-1 receptor (IL1-R1) (IC50=8 nM) but not the human type II receptor (IC50=6.7 μM) or the murine type I receptor (IC50>200 μM). AF12198 inhibits IL-1-induced IL-8 production (IC50=25 nM) and IL-1-induced intercellular adhesion molecule-1 (ICAM-1) expression (IC50=9 nM) in vitro. AF12198 has anti-inflammatory activities and?blocks responses to IL-1 in vivo.

A-1331852 is a high affinity BH3 mimetic Ligand of BCL protein BCL-XL.

TS41 is a trisulfide-derived ionizable lipid engineered for lipid nanoparticles (LNPs) to deliver mRNA therapeutics against multidrug-resistant bacterial pneumonia. Its optimized formulation, TS41S LNP, combines TS41 with helper lipids (e.g., DOPE, cholesterol) at a precise ratio, achieving a hydrodynamic diameter of ~105 nm, low polydispersity, and high mRNA encapsulation efficiency (~84%). This design enables efficient pulmonary delivery via intratracheal administration, with luminescence signals in lungs 4.8-fold higher than clinical benchmarks like SM-102 LNPs, ensuring targeted expression in epithelial cells, macrophages, and neutrophils. Crucially, TS41 LNPs exhibit potent anti-inflammatory properties by scavenging reactive oxygen species (ROS), reducing neutrophil infiltration and proinflammatory cytokines (e.g., IL-6, TNF-α) in infected lungs. In preclinical models, TS41S LNP encoding PB9 peptibody mRNA eradicated pathogens like Staphylococcus aureus and Pseudomonas aeruginosa, improved survival rates to 80%, and minimized tissue damage without systemic toxicity. Its ROS-scavenging capability synergizes with antibacterial effects, offering a promising, translatable platform for combating resistant infections while controlling inflammation. Future enhancements, such as codon optimization or inhalation delivery, could further broaden its therapeutic potential.

4A2-B8-PH is an optimally designed thioketal-incorporated biodegradable ionizable lipid (TBIL) for mRNA delivery to pancreatic ductal epithelial cells. It features a 4A2 headgroup with three tertiary amines, a biodegradable thioketal-based B8 linker, and a branched PH tail. The thioketal linker enables ROS-responsive degradation in the tumor microenvironment, enhancing endosomal escape and mRNA release. In vivo, 4A2-B8-PH LNPs achieve 98.3% pancreas-specific targeting after intraperitoneal administration, with a 218-fold improvement in delivery efficiency compared to previous benchmarks. It successfully transfects 30.5% of pancreatic ductal epithelial cells and induces complete tumor regression in orthotopic PDAC models via IL-12 mRNA therapy, demonstrating high efficacy and safety.

6F Lipid is a Fluorinated Ionizable Lipid breakthrough in mitochondria-targeted gene delivery

Lipid 5 is an ionizable lipid based on a macrocyclic cyclam headgroup. Its structure incorporates a benzylmethyl carbonate (BMC) linker, which contains an aromatic benzene ring, and a saturated C18 hydrophobic tail. Lipid 5 was mixed with helper lipids at a fixed molar ratio and formulated into mRNA-loaded lipid nanoparticles (LNPs) using microfluidic technology. Characterization data show that these LNPs have a hydrodynamic diameter of approximately 50-80 nanometers and a polydispersity index (PDI) below 0.2, indicating a small particle size with a uniform distribution. Their zeta potential at physiological pH is near neutral (ranging from -3 to +3 mV). The mRNA encapsulation efficiency, as determined by the Ribogreen assay, exceeds 95%. Cryo-transmission electron microscopy images reveal that the LNPs exhibit a typical spherical bilayer structure. In in vitro experiments, Lipid 5 LNPs mediated a higher level of luciferase protein expression in HEK293FT cells compared to the benchmark lipid DLin-MC3-DMA. In Balb/c mice, intravenous injection of LNPs encapsulating luciferase mRNA resulted in in vivo imaging signals predominantly concentrated in the lungs. Quantitative analysis indicated that the signal intensity in the lungs was over 100 times greater than that in the liver, with more than 95% of the total signal distributed in the lungs. In Ai9 reporter gene mice, two intravenous injections of Lipid 5 LNPs encapsulating Cre mRNA led to quantitative analysis of lung tissue sections showing that approximately 30% of lung cells were positive for tdTomato signal.