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.

Lipid-87​​ is an ionizable lipid developed by Generation Bio, characterized by its ​​tertiary amine group​​ for pH-dependent protonation and ​​dual C16/C17 aliphatic chains​​ that enhance hydrophobic stability.As the core component (47.5–57.5 mol%) of stealth lipid nanoparticles (LNPs), Lipid87 enables extended blood circulation (>24-hour half-life vs. 30 min for conventional LNPs) by synergizing with steric-stabilizing polymers (e.g., DSG-PEG₂₀₀₀-OMe), achieves >95% encapsulation efficiency for mRNA/ceDNA with low cytotoxicity (IC₅₀ >100 μM), and drives liver-specific targeting (>80% hepatocyte transfection at 0.5 mpk), effectively restoring 40% FIX activity in hemophilia B models for over 7 days.

2Ac3-C18 is a unique ionizable lipid with a distinct degradable core structure：featuring 2 acrylate units and 3 amine groups—linked to a C18 alkyl chain. Its LNPs (formulated with DOPE/cholesterol/DMG-PEG2000) exhibit spleen-specific mRNA delivery in vivo.

KC3-OA, chemically known as 3-((S)-2,2-di((Z)-octadec-9-en-1-yl)-1,3-dioxolan-4-yl)-N,N-dimethylpropan-1-amine, is an ionizable cationic lipid (ICL) optimized for lipid nanoparticle (LNP) formulations in nucleic acid delivery, particularly for mRNA vaccines. It features a unique structure with mono-unsaturated alkyl chains (C18:1), which enhances oxidative stability compared to polyunsaturated analogs like KC3, while maintaining efficient membrane fusion and endosomal escape capabilities. In LNP compositions, KC3-OA is typically incorporated at 46–54 mol% of total lipids, with an N/P ratio of 4–6 relative to mRNA, ensuring high encapsulation efficiency and transfection potency. Experimental data demonstrate that KC3-OA-based LNPs achieve superior mRNA expression in human dendritic cells, outperforming alternatives like KC3-PA or KC3-01 in both in vitro and in vivo models. For instance, in FIG. 2, KC3-OA LNPs showed ~2-fold higher mCherry expression at low mRNA doses (0.1 μg/mL) due to improved cellular uptake and reduced degradation. Its synergy with anionic phospholipids like DPPS (5 mol%) further enhances dendritic cell targeting via receptor-mediated internalization, leading to robust CD4+ and CD8+ T-cell responses against Mycobacterium tuberculosis antigens. This balance of stability, efficiency, and immunogenicity makes KC3-OA a leading candidate for next-generation vaccines.

L31 is identified as a novel, proprietary ionizable cationic lipid that serves as the critical functional component within lipid nanoparticles (LNPs) engineered for CRISPR/Cas9 genome editing in head and neck squamous cell carcinoma (HNSCC). It was selected from a screened library of lipids for its superior performance. LNPs formulated with L31 exhibited excellent physicochemical properties, including a uniform size of 80-100 nm, low polydispersity, and high encapsulation efficiency (>85%) for both Cas9 mRNA and sgRNA. In vitro, L31-based LNPs demonstrated outstanding therapeutic efficacy, achieving approximately 68% gene editing of the oncogene SOX2 and an 88% reduction in cancer cell viability.For in vivo applications, L31-LNPs were further functionalized with anti-EGFR antibodies using the ASSET linker strategy to create targeted nanoparticles (tLNPs). This modification enhanced specific uptake by tumor cells. In a xenograft mouse model, intratumoral injection of these targeted L31-cLNPs co-encapsulating Cas9 mRNA and sgSOX2 led to potent tumor growth inhibition (90%) and a significant increase in survival, with tumor disappearance observed in half of the treated mice. In conclusion, L31 is a highly efficient ionizable lipid that forms the foundation of a potent targeted LNP platform for precise CRISPR-based cancer therapy against solid tumors.

Diamino lipid DAL4 is diamino lipid for the preparation of lipid nanoparticles (LNPs) encapsulated with mRNAs encoding cytokines including IL-12, IL-27 and GM-CSF. Diamino lipid DAL4 delivers mRNA to tumor cells to exert anti-tumor activity.

KT-001 is a novel ionizable cationic lipid disclosed in patent US 2026/0007612 A1

XH-07 is an innovative ionizable cationic lipid that forms the backbone of the JCXH-211 lipid nanoparticle (LNP) delivery system. This complex is engineered to encapsulate and deliver self-replicating RNA (srRNA) encoding interleukin-12 (IL-12), a potent immunostimulatory cytokine. The LNP formulation featuring XH-07 exhibits optimal physicochemical properties, such as a mean particle size of approximately 82.12 nm with low polydispersity, and a near-neutral zeta potential around -3.181 mV, which facilitates stable circulation and efficient cellular uptake upon intravenous administration. Upon delivery, the srRNA leverages the host cell's machinery to produce sustained levels of IL-12p70, as demonstrated in B16F10 tumor-bearing mice, where a single dose led to peak cytokine production in sera and tumors. This induced IL-12 expression activates T cells and NK cells, generating a robust antitumor response. In murine models of melanoma and breast cancer, JCXH-211 monotherapy resulted in significant tumor regression and complete responses in some subjects, and it synergized with anti-PD-1 therapy to enhance efficacy. Importantly, the safety profile was acceptable, with transient liver enzyme elevations in mice that normalized quickly, and no significant adverse events in cynomolgus monkeys after repeated dosing, as evidenced by stable clinical observations and pathology tests. Thus, XH-07 is pivotal for enabling the safe and effective delivery of IL-12 encoding RNA, positioning JCXH-211 as a promising cancer immunotherapy.

Lipid 48​ is a leading ionizable lipid designed for therapeutic nucleic acid delivery. Its key function is to form the core of lipid nanoparticles (LNPs) that efficiently encapsulate and deliver cargoes like mRNA and CRISPR guide RNAs into cells. Its optimized structure allows it to remain neutral in the bloodstream for low toxicity but become positively charged in acidic cellular compartments (endosomes), where it disrupts the membrane to release the therapeutic payload. Data from the patent demonstrates its superior profile: it achieves high gene editing efficiency (e.g., ~80% indel rates in vitro and 16.2% in vivo in mouse liver) while maintaining low cytotoxicity (cell viability >80% at effective doses), establishing it as an ideal candidate for gene therapy applications due to its exceptional balance of potency and safety.

244-9-cis is a novel ionizable lipid disclosed in United States Patent US 2026/0014075 A1, specifically engineered for advanced lipid nanoparticle (LNP) delivery systems. Its distinctive molecular architecture features biodegradable ester bonds, which contribute to excellent physicochemical properties such as a near-neutral surface charge (approximately -3 mV) for improved biocompatibility, an optimal pKa of about 6.2 to facilitate endosomal escape, and consistently high nucleic acid encapsulation efficiency exceeding 90%. In vivo studies confirm significantly enhanced delivery to hepatocytes and markedly higher therapeutic protein expression compared to control formulations, positioning 244-9-cis as a promising candidate for next-generation genetic medicines.

AP60 is a novel, biomimetic ionizable lipid, identified as the lead compound from a library of 67 aminophosphonate-derived lipids. Inspired by the structure of natural phospholipids, it forms the core component of lipid nanoparticles within the CROSS delivery platform. AP60-based LNPs demonstrate superior efficiency in delivering mRNA and circular RNA to neuronal cells and astrocytes compared to the clinically used MC3 LNP. In a mouse model of spinal cord injury, AP60 LNPs achieved significantly higher protein expression at the lesion site (13.7-fold locally, 4.6-fold intravenously) while concurrently reducing off-target accumulation in the liver by nearly 5-fold. This indicates improved targeting to the central nervous system injury site and a potentially better safety profile. Its cellular uptake is primarily mediated by clathrin-mediated endocytosis and macropinocytosis, followed by efficient endosomal escape. By encapsulating therapeutic circular RNAs encoding Sox2, Ascl1, and GDNF, AP60 LNPs enable effective in vivo reprogramming and neuroprotection, leading to significant functional recovery of bladder and locomotor functions after spinal cord injury.

Lipid OC7, as described in the patent WO2022207938A1, is a novel ionizable lipid that serves as the core functional component of the saNppa-LNP delivery system. Its key innovation lies in its unique biodegradable structure featuring an internal ester bond. Under typical physiological conditions, this bond hydrolyzes, triggering a charge shift from a cationic form that complexes nucleic acids to a zwitterionic form that releases them. This property is central to its role in enabling long-acting self-amplifying RNA (saRNA) therapies. Specifically, OC7 facilitates immune stealth by mitigating early interferon responses, supports sustained and efficient intracellular replication of saRNA even at low doses, and enables therapeutic protein expression that persists for over 28 days from a single administration. This combination of efficient delivery, controlled release, and extended duration of action makes OC7-based LNPs a promising platform for long-term treatments, such as for myocardial infarction, as demonstrated in the referenced research.

First Inhibitor of PEX14-PEX5 Protein-Protein Interaction (PPI) with Trypanocidal Activity

Lysophosphatidylcholine 18:2 (1-Linoleoyl-2-Hydroxy-sn-glycero-3-PC), a lysophospholipid, is a potential biomarker identified from insulin resistance (IR) polycystic ovary syndrome (PCOS). Low plasma Lysophosphatidylcholine 18:2 also has been shown to predict impaired glucose tolerance, insulin resistance, type 2 diabetes, coronary artery disease, and memory impairment.

SLW131 (Compound 10) is the antagonist for CCR7 with a good affinity of Ki of 9.85 nM. SLW131 inhibits CCL19-induced Go protein activation with an IC50 of 29.4 μM, inhibits β-arrestin2 recruitment with an IC50 of 6.0 μM. SLW131 inhibits CCL19-induced cell morphological changes in primary BMDCs, and CCR7-mediated migration in mouse CD4+ T cell.

Novel modulator of NF-Y transcription, inhibiting the binding of NF-Y to DNA, blocking cellular proliferation and cell cycle progression, interacting with a variety of CCAAT-containing promoters leading to p53-independent cell cycle arrest

BEN-28010 is a potent selective, orally bioavailable and brain-penetrant checkpoint kinase 1 (CHK1) inhibitor with IC50 of 4 nM, 450-fold selective over CHK2.

NP1867 is a potent, selective, covalent PMS2 inhibitor. NP1867 functionally inhibits DNA mismatch repair. NP1867 enhances immune surveillance. NP1867 can be used in the research of colorectal cancer.

A potent, selective and orally bioavailable TRPV1 receptor antagonist with IC50 of 65 nM and 102 nM for hTRPV1 and rTRPV1, respectively.

CCR7 inhibitor Cmp1205 is an allosteric ligand and antagonist for human CC chemokine receptor 7 (CCR7) with Kd of 3 nM, suppresses arrestin binding in response to activation by CCL19 with IC50 of 7.3 uM.Cmp1205 binds to a pocket at the intracellular part of CCR7 between the ends of TM1, TM2, TM3, and TM6 and the loop between TM7 and H8.Cmp1205 allosterically inhibits binding of the native chemokine CCL19 ligand in a membrane-based competition binding experiment with IC50 of 35 nM.

ASGPR ligand-1 is a compound that targets the asialoglycoprotein receptor (ASGPR) and is utilized in various disease research contexts .