VL422 is an ionizable cationic lipid. It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA for gene editing in vitro and in vivo. LNPs containing VL422 and encapsulating Cas9 mRNA and sgRNA targeting the gene encoding angiopoietin-related protein 3 (ANGPTL3), a protein whose loss-of-function decreases LDL, HDL, and cholesterol plasma levels, induce a deletion in a premature stop codon in ANGPTL3 in the livers of cynomolgus monkeys.

ALC-0159 (di-C18) is an analog of ALC-0159, in which the original C14 chain is replaced with C18 chain. C18-ALC-0159 demonstrates high stability in vivo, effectively prevents binding with proteins such as ApoE, and achieves a prolonged blood circulation time. It also reduces liver targeting and accumulation.

Sail Lipid 2308​ is a novel ionizable lipid targeting to spleen developed by Sai Biomedicine.As described on US20250205167A1, Lipid 2308 was designed with a ​​piperidine core​​ (6-membered ring) and asymmetric C17/C11 chains, this lipid achieves unprecedented ​​spleen-specificity​​. It demonstrates dominant spleen accumulation (Spleen RLU: ​​7.8E+06​​, 91.8% of total signal) with a record ​​spleen-to-liver ratio of 112.7​​ (9× higher than 2231). Despite lower protein expression (hEPO: 11,000 ng/mL), near-zero liver uptake (Liver RLU: 66,000) makes Lipid 2308 unparalleled for vaccine/immunotherapy applications targeting splenic immune cells.

Sail Lipid 2231 is a novel ionizable lipid targeting to spleen developed by Sai Biomedicine.As described on US20250205167A1 Lipid 2231 features  a ​​pyrrolidine core​​ (5-membered ring) with biodegradable ester linkages and asymmetric C17/C11 hydrophobic chains. In vivo data shows moderate spleen targeting (Spleen RLU: ​​3.8E+06​​) with a spleen-to-liver ratio of ​​12.767​​. 

​​Lipid 10a-26​​ is an ionizable lipid developed by Orna Therapeutics for lipid nanoparticle (LNP) formulations. It features a biodegradable ester backbone and an ionizable headgroup, enabling efficient encapsulation and delivery of circular RNA (oRNA). Experimental data show that Lipid 10a-26 mediates robust protein expression in hepatocytes and immune cells (e.g., T cells), with strong liver-targeting specificity observed in vivo. Its optimized hydrolysis profile ensures stable oRNA delivery and reduced immunogenicity. For instance, LNPs formulated with Lipid 10a-26 (molar ratio 50:10:38.5:1.5) demonstrate high transfection efficiency in splenic B cells and sustained therapeutic protein production.The lipid’s design balances efficacy and safety, making it ideal for applications like CAR-T therapy and hepatic protein replacement.

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​​.

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.

CLC-2-IN-AK42 (CLC-2 inhibitor AK42, AK42) is a potent, specific small-molecule inhibitor of voltage-gated chloride channel CLC-2 with IC50 of 17 nM (human CLC-2).AK42 displays unprecedented selectivity (>1,000-fold) over CLC-1, the closest CLC-2 homolog, and no off-target engagement a diverse panel of 61 CNS receptors, channels, and transporters expressed in brain tissue.AK-42 binds to an extracellular vestibule above the channel pore. AK-42 is almost three orders-of-magnitude more potent than MCFA, demonstrates high potency (IC50=14 nM for rat CLC-2) in manual patch-clamp experiments on CHO cells transiently transfected with rat CLC-2.AK-42 inhibits hyperpolarization-activated current in hippocampal cells, attenuates steady-state currents and eliminated relaxation currents in recorded neurons.AK-42 is a powerful tool for investigating CLC-2 neurophysiology.

TE AA3-Dlin is an optimized lipid nanoparticle (LNP) carrier designed for mRNA-based cancer immunotherapy, enabling precise in vivo dendritic cell (DC) reprogramming to enhance antitumor immunity. TE AA3-Dlin LNP exhibits superior serum stability, maintaining consistent particle size and low turbidity under physiological conditions, while protecting mRNA from degradation, which is crucial for effective delivery. Functionally, TE AA3-Dlin preferentially targets splenic DCs by leveraging ApoE-enriched protein coronas, facilitating efficient cellular uptake and mRNA expression, as demonstrated by enhanced EGFP signals in DCs.This targeting promotes DC maturation, antigen presentation, and membrane-bound IL-15 expression, activating cytotoxic T lymphocytes (CTLs) for tumor rejection. In models like melanoma and colon carcinoma, it synergizes with checkpoint inhibitors, showing minimal toxicity and robust immunological memory.

​​BMS-986463​​ is a novel, first-in-class targeted protein degrader designed to specifically eliminate WEE1 kinase, a master regulator of cell cycle progression.

Aminoxyrone is a novel peptidometic C-terminal HSP90 inhibitor by targeting HSP90 dimerization via the C-terminal domain (CTD) with Kd of 27.4 uM; destabilizes BCR-ABL1 without inducing HSR in vitro and in vivo, additionally reduces pAKT-S473, pS6 expression and expression of client proteins associated with HSP90 chaperone activity, involving t-AKT, t-STAT5a, t-CRKL, cMYC, and BCL2; triggers the degradation of HSP90 client proteins without elevating the expression of HSPs (HSP70, HSP40 & HSP27); significantly inhibits cell growth and induces apoptosis of human leukemic stem cells (LSCs) with average EC50 of 20.94 uM, Aminoxyrone is effective in imatinib resistant CML and lacks heat shock response.

LY315920 (Varespladib) is a potent and selective human non-pancreatic secretory phospholipase A2 (hnsPLA) inhibitor with IC50 of 7 nM,. Phase 3.

F10T5 is a tetrahedral tetrahydrofuran (THF)-derived lipid nanoparticle (LNP) engineered with four acid-labile acetal tails, designed for efficient mRNA delivery to the central nervous system. This lipid features a mono-THF core conjugated with branched hydrophobic chains that balance lipophilicity (LogD ≈11) and endosomal escape capability. Preclinical studies demonstrated F10T5 LNPs bypass the blood-brain barrier via meningeal lymphatic vessels (MLVs) after subcutaneous neck injection, showing 40-fold higher brain luciferase expression than FDA-approved SM102 LNPs. Cryo-EM revealed spherical nanoparticles (~170 nm diameter) with 91.9% mRNA encapsulation. In Neuro-2a cells, F10T5 exhibited superior cytoplasmic mRNA release through enhanced endosomal membrane disruption, evidenced by diffuse calcein fluorescence. Flow cytometry confirmed neuron-predicted delivery (8.8% GFP+ neurons vs 1.28% with SM102) in mice, with functional validation in Ai14 transgenic models where Cre mRNA-loaded F10T5 induced tdTomato expression in neurons and glial cells. Safety assessments showed normal hepatic/renal biomarkers and no histopathological abnormalities. The THF core and acetal tail design synergistically optimize lymphatic trafficking, brain penetration, and biodegradability, positioning F10T5 as a transformative platform for mRNA-based therapies targeting neurodegenerative diseases.

GS-7340(Tenofovir alafenamide) is a prodrug of tenofovir (TFV) that more efficiently delivers TFV into lymphoid cells and tissues than TFV disoproxil fumarate.

CID 3117694 is a selective, non-competitive inhibitor of ADAM10 with IC50 of 1.1 uM, does not inhibit ADAM17.

GNE551 is a potent, selective, non-covalent agonist of TRPA1 ion channel with EC50 of 254 nM in Ca2+ influx assays.

Cagrilintide is an investigational novel long-acting acylated amylin analogue, acts as nonselective amylin receptors (AMYR) and calcitonin G protein-coupled receptor (CTR) agonist. Cagrilintide induces significant weight loss and reduces food intake. Cagrilintide has the potential for the research of obesity.

Modified phospholipid products: it is to modify the amino group (primary amino group) -NH3 at the end of DSPE into NHS, COOH, N3, MAL, Thiol (SH), OPSS, FITC, FA, Biotin and other different active groups. Phospholipids DSPE belong to the lipid family of biopolymers. Phospholipids consist of two fatty acids, a glycerol unit, a phosphate group, and a polar molecule. The phosphate groups and polar head regions of the molecule are hydrophilic (attracted to water), while the fatty acid tail is hydrophobic (repelled by water). When placed in water, the phospholipids Orient themselves into a double layer, where the non-polar tail region faces the inner region of the double layer. The polar head region faces outward and interacts with the water.

ARV-102 is an orally bioavailable PROTAC LRRK2 degrader with DC50 of 0.14 nM. ARV-102 demonstrates substantial reductions in peripheral LRRK2 proteinlevels.

Novel peptidomimetic antagonist of the PHF1 Tudor domain, binding both PHF1 Tudor domain and the related protein PHF19

MRT-6160 represents a groundbreaking molecular glue degrader designed to selectively target VAV1, achieving its proteasomal degradation with a DC50 value of 7 nM. This innovative compound showcases its unique mechanism and therapeutic potential.

STING inhbiitor, which inhibited the activation of the STING signal pathway and to prevent or treat a STING-​mediated disease.

SB-405483 is a specific small-molecule chemical compound used in scientific research as an allosteric ligand for the protein cereblon (CRBN). It is primarily a biochemical tool for studying protein degradation pathways and has potential implications for drug discovery.

Lipid C3 is an ionizable cationic lipid (pKa = 5.05-5.671).1,2 It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA in vitro and in vivo.

CCI-38 is an effective YTHDF2 inhibitor with Kd of 10.1 μM. CCI-38 can suppress YTHDF2-mediated post-transcriptional regulation of target gene expression. CCI-38 also exhibits potent therapeutic efficacy and synergies with T cell-based immunotherapy in treating B cell malignancies.

DD-CIP2 is a DNA damage chemical inducer of proximity. DD-CIP2 demonstrates dramatically increased potency in SU-DHL-5 and MOLT-4 cells with EC50 of 0.78 nM and 4.6 nM, respectively. DD-CIP2 induces robust DNA damage and apoptosis in vitro and exhibits antitumor efficacy in vivo.

UDSP-Hep is a bacterial ADP-heptose analogue. UDSP-Hep is a potent ALPK1 agonist with EC50 of 0.0423 μM. UDSP-Hep distinguishs Alpk1 polymorphism and exhibits a stronger Alpk1-mediated antitumour effect and synergized with checkpoint inhibitors.

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.

D927 is a molecular glue and promotes glucose uptake in the absence of insulin. D927 also increases the affinity of RAS binding to PI3Kα by ~500-fold. In vivo, D927 mimicks the effects of insulin and rapidly lowers blood glucose concentrations.

PT-179 is a novel orthogonal immunomodulatory drug (IMiD) derivative that selectively binds to CRBN without inducing degradation of off-target proteins. It demonstrates potent activity in degrading proteins fused to SD40, regardless of whether the fusion occurs at the N or C terminus.