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.

MOCHOL is an ionizable morpholine-modified cholesterol derivative. Unlike ordinary cholesterol that only stabilizes lipid bilayers, its tertiary amine head protonates in acidic endosomes to boost mRNA release via proton sponge effect. It reduces LNP hemolysis and serum lipid exchange, weakens hepatic tropism, and works compatibly with SM-102, AO12 and other ionizable lipids for extrahepatic mRNA delivery.

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​​.

Trp-L1-T4 is a novel tryptophan-derived ionizable lipid that serves as the core functional component of the optimized lipid nanoparticle (TLNP/RLNP) platform. Its primary function is to enable the efficient encapsulation and in vivo delivery of self-amplifying RNA (saRNA) cargo. Specifically, it facilitates high transfection efficiency and cytosolic release of the RNA payload in target follicular helper T (Tfh) cells, with minimal cytotoxicity. This capability is crucial for reprogramming pathogenic Tfh cells into regulatory CAR-Tfh cells, forming the foundation of the study's therapeutic strategy.

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

A5-CE-C7-6 is an ionizable lipid engineered for spleen-targeted mRNA delivery, integrating a hydroxylated dual-amine core (A5) for enhanced mRNA binding and endosomal escape, a biodegradable carbonate ester linker (CE) enabling rapid hydrolysis (61% degradation in 24 h), and branched heptyl hydrophobic tails (C7-6) that optimize nanoparticle stability and spleen tropism.​​ When formulated into cholesterol-free lipid nanoparticles (B-8 formulation), its unique architecture—combining hydroxyl groups for cellular uptake, carbonate-mediated biodegradability, and branched-chain fluidity—achieves unprecedented efficiency: low pKa (~6.0) minimizes liver accumulation while enabling ​​21% transfection of splenic NK cells​​, outperforming benchmark systems like MC3 SORT LNPs by >10-fold in spleen-specific delivery and establishing a new standard for in vivo immune cell engineering.

KC‑34 (SPC‑A9) is a novel stereopure, diketopiperazine-based ionizable cationic lipid engineered to overcome traditional liver-restricted delivery, achieving balanced multi-organ mRNA transfection. Upon systemic intravenous administration, its precisely optimized chiral configuration allows the lipid nanoparticles (LNPs) to efficiently cross endothelial barriers and target the bone marrow, offering immense therapeutic potential for in vivo hematopoietic stem cell gene editing. Concurrently, KC-34 mediates robust and long-lasting protein expression in the spleen and lungs with minimal hepatic off-target toxicity. Its stable structure provides excellent biocompatibility and high in vivo tolerance, making it ideal for systemic, multi-dose mRNA therapies.

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.

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.

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.

dBET6 is a highly potent, selective and cell-permeable degrader of BET with an IC50 of 14 nM, and has antitumor activity.

ARS-1620 is a covalent compound with high potency and selectivity for KRAS-G12C.

AMG514 is a novel ionizable lipid designed for formulating spleen-targeting lipid nanoparticles (LNPs) to deliver immune‑remodeling mRNAs (IR‑mRNAs). Its key advantage lies in the formation of a unique “protein corona” enriched with vitronectin, coagulation factors, and specific apolipoproteins (e.g., ApoA‑IV), together with its relatively high apparent pKa (~7.5), which actively redirects LNPs to the spleen instead of the liver. This precise spleen‑targeting property enables efficient transfection of splenic antigen‑presenting cells (APCs). As a vaccine adjuvant, AMG514‑LNPs therefore elicit a more robust activation of adaptive immunity compared to conventional LNPs (e.g., cKK‑E12), generating significantly enhanced antigen‑specific CD8⁺ T‑cell and antibody responses, and inducing durable anti‑tumor immune memory in preclinical models.

G-Glu-Val, also known as gamma-Glutamyl-L-valine or H-gGlu-Val-OH, is a taste-modulating dipeptide and a main contributor to the "kokumi" taste of edible beans.

MAK683 is a novel PRC2/EED inhibitor.

CO-1686 is a novel, irreversible and orally delivered kinase inhibitor that specifically targets the mutant forms of EGFR including T790M(IC50=21 nM).

DS-6051b is a potent and selective ROS1 and TRK family inhibitor with IC50 of 0.207 nM, 0.622 nM and 0.980 nM against ROS1, NTRK1 and NTRK3. DS-6051b especially inhibits ROS1 G2032R and other crizotinib-resistant ROS1 mutants.