GalNac-L96, the G-rich oligonucleotides carrying the longer GalNAc linker that can be used for delivery of nucleic acid drugs[1].

LS-170 is a potent, selective and cell-active chemical inhibitor that targets the ATAC-specific YEATS2 YEATS domain with IC50 of 0.14 μM. LS-170 specifically reduces the chromatin occupancy of the ATAC complex, decreases the ATAC-dependent histone acetylation level and downregulates the expression of ATAC-governed genes, leading to significantly suppressed tumor growth in a lung cancer mouse model.

Compound 78 is a molecular glue for the unique composite surface of the 14-3-3/C-RAF259 complex. Compound 78 results in 210-fold stabilization of the 14-3-3σ/C-RAF259 complex in fluorescence anisotropy assays and has an EC50 of 1.2 μM in a cellular 14-3-3/C-RAF NanoBRET assay. Compound 78 shows greater selectivity for C-RAF in the NanoBRET assay and the protection of phosphorylation.

KRAS inhibitor-9, a potent KRAS inhibitor (Kd=92 μM), blocks the formation of GTP-KRAS and downstream activation of KRAS. KRAS inhibitor-9 binds to KRAS G12D, KRAS G12C and KRAS Q61H protein with a moderate binding affinity. KRAS inhibitor-9 causes G2/M cell cycle arrest and induces apoptosis. KRAS inhibitor-9 selectively inhibits the proliferation of NSCLC cells with KRAS mutation but not normal lung cells.

AMG1556 is a biodegradable, cyclic amino alcohol-based ionizable lipid from the AMG series, designed specifically for mRNA delivery via lipid nanoparticles (LNPs).

ATN-224 is a choline salt of tetrathiomolybdate and an inhibitor of superoxide dismutase 1 (SOD1; IC50s = 2.91 and 3.51 µM in human and mouse blood cells, respectively, in vitro).

The trivalent β-GalNAc ligand is specific for Asialo Glycoprotein Receptors (ASGPR) on hepatocyte cells. β-Ala-PEG3 acts as linker/spacer between triantennary GalNAc and Fluorescein isothiocyanate (FITC).

A thiol-reactive asialo glycoprotein receptor (ASGPR) ligand.

Tris-GalNAc-GABA-NH2 linked via PEG2k to phospholipid (DSPE or 1,2-distearoyl-sn-glycero-3-phosphoethanolamine) where GABA = gamma-Aminobutyric acid, or γ-aminobutyric acid.

An amine-reactive asialo glycoprotein receptor (ASGPR) ligand.

Triantennary GalNAc ligand is known to bind Asialo Glycoprotein Receptor (ASGPR). The ligand is functionalized with dibenzocyclooctyne for click chemistry.

Triantennary GalNAc ligand is known to bind Asialo Glycoprotein Receptor (ASGPR). The ligand is functionalized with dibenzocyclooctyne for click chemistry

(β-D-GalNAc-sp)3-NHC(O)-(CH2)2-NHC(O)-PEG3-(CH2)2-N3 Tris-β-D-GalNAc ligands bind with asialoglycoprotein receptors (ASGPR) that are highly expressed on hepatocytes resulting in rapid endocytosis of the ligand along with conjugated payloads

Trivalent β-GalNAc Ligand with discrete, monodisperse, azido-functionalized PEG3 linker/spacer

Tris-GalNAc ligands bind to Ashwell-Morell (also Asialoglycoprotein receptor or ASGPR) receptors and are validated ligands in medicinal chemistry for liver-specific drug delivery.

Tri-GaNAc-PEG8-Gly-Gly-Gly is a compound that targets the asialoglycoprotein receptor (ASGPR) and is utilized to conjutate with antibody.

The trivalent β-GalNAc Ligand is specific for Asialo Glycoprotein Receptors on hepatocyte cells. β-Alanine-PEG3 acts as linker/spacer between multivalent GalNAc and biotin.

Tri-GaNAc-PEG8-NH2-CH2CH2-NH2-bromoacetyl is a compound that targets the asialoglycoprotein receptor (ASGPR) and is utilized to conjutate with antibody.

A2-Iso5-2DC18 is a top-performing lipid for mRNA delivery in bone marrow-derived dendritic cells (BMDCs), BMDMs and HeLa cells.

LIPID168(pKa ~6.5) ​​ is an optimized ionizable lipid engineered for in vivo mRNA delivery to hematopoietic stem cells (HSCs) in bone marrow. Developed by ​​Yoltech Therapeutics​​ through high-throughput screening of lipid libraries, it features a ​​diethylamino head group​​ and a tailored hydrophobic tail structure that enables antibody-free targeting. When Lipid 168 was formulated into lipid nanoparticles (LNPs), it achieved ​​48.5% base editing efficiency​​ in bone marrow cells —surpassing benchmarks like LIPID-028 (19.7%)—and reduced off-target liver editing from 71% to 19% by incorporating ​​miR-122 target sequences​​. In humanized β-thalassemia models, LNP 168 delivered ABE8e mRNA/sgRNA to patient-derived HSCs, yielding ​​42.6% editing at the HBG promoter​​, reactivating fetal hemoglobin (γ-globin) and rescuing erythroid defects . Its bone marrow specificity is driven by a unique ​​protein corona​​ enriched in albumin, fibronectin, and fibrinogen . Safety studies confirmed transient immune responses and no cumulative toxicity . LIPID-168 represents a promising non-viral platform for curative gene therapies in blood disorders.

Lipid 10 is a novel ionizable cationic lipid be used for delivery of therapeutic RNA to the Bone Marrow in Multiple Myeloma Using CD38-Targeted with Lipid 10-LNP.

HY-501​​ is a next-generation cationically ionizable lipid engineered for high-efficiency RNA delivery developed by Biontech. Formulated at ​​40–50 mol%​​ in lipid nanoparticles (LNPs) alongside DSPC, cholesterol, and polysarcosine-conjugated lipid ​​C14pSar23​​, HY-501 yields uniform, stable particles (80–100 nm) with >90% RNA encapsulation. It demonstrates ​​superior in vivo performance​​: driving 2-fold higher protein expression than benchmark lipids (EA-405/HY-405) in muscle tissue, minimizing off-target liver accumulation, and reducing immunogenic risks (near-zero complement activation and <5% hemolysis). Preclinically, HY-501-based LNPs encoding SARS-CoV-2 spike protein elicit potent neutralizing antibodies and T-cell responses, underscoring its utility in precision vaccines. Its combination of scalable synthesis, exceptional transfection efficiency, and biosafety establishes HY-501 as a transformative vector for therapeutic RNA delivery.

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.

TCL053 is an ionizable amino lipid.1 It has been used in the generation of lipid nanoparticles (LNPs) and has a pKa value of 6.8. LNPs containing TCL053 and encapsulating mRNA encoding the Cas9 nuclease, in combination with LNPs containing TCL053 and encapsulating single-guide RNA (sgRNA) targeting the Rosa26 locus, have been used to induce CRISPR-mediated gene editing in the mouse gastrocnemius muscle.TCL053 is an ionizable lipid that has received FDA approval for preparing mRNA vaccines. It is a three-tailed ionizable lipid to overcome the disadvantage of nonrepeatable administration of AAV vectors. In addition, combined with limb perfusion administration, TCL053 iLNPs could transiently deliver CRISPR-Cas9 mRNA and sgRNA to multiple muscle tissues, reducing immunogenicity and increasing the safety of iLNPs. It is great progress for treating Duchenne muscular dystrophy and other diseases that require multiple doses.

Iso-A11B5C1 is an ionizable lipid. The iso-A11B5C1 LNP demonstrates a high level of muscle-specific mRNA delivery efficiency. exhibiting transfection efficiency comparable to the commercially available lipid SM-102, while considerably reducing inadvertent mRNA expression in main organs such as the liver and spleen.Additionally, study results show that intramuscular administration of mRNA formulated with iso-A11B5C1 LNP caused potent cellular immune responses, even with limited expression observed in lymph nodes.

(±)-H3RESCA-TFP ((±)-H3L28) is a tetrafluorophenyl ester derivative of restrained complexing agent (RESCA). (±)-H3RESCA-TFP can be used to conjugate the chelator with a biomolecule via amine coupling (e.g., N terminus and/or the ε-amino groups of lysine).

FAS-IN-1 is a potent inhibitor of fatty acid synthase (FAS) wtih IC50 of 10 nM..