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.

BBO-11818 is an orally active, highly selective (relative to NRAS and HRAS), non-covalent pan-KRAS inhibitor (IC50=28-120 nM). BBO-11818 specifically binds to the Switch-II/Helix 3 pocket, disrupts the KRAS:RAF1 interaction by inducing conformational changes, and blocks the MAPK signaling pathway. BBO-11818 exhibits significant anti-tumor activity, which not only inhibits cell proliferation and induces apoptosis, but also drives tumor regression in xenograft models. BBO-11818 produces synergistic effects when combined with Cetuximab, anti-PD-1 antibody or PI3Kα inhibitor. BBO-11818 is used in the research of KRAS mutation-related malignancies such as pancreatic cancer, non-small cell lung cancer and colorectal cancer.

MMV009085 is a potent and selective inhibitor of the Plasmodium falciparum hexose transporter (PfHT1), designed to target malaria by blocking glucose uptake, with an IC50 of 2.6 μM for the transporter and an EC50 of 1.23 μM against the 3D7 strain. It acts as a probe-like molecule with high selectivity for PfHT1 over human glucose transporters (GLUTs). In vitro, it shows selective inhibition of Neospora caninum tachyzoite proliferation in infected host cell assays, with an IC₅₀ of approximately 150 ± 30 nM, while also displaying activity against related apicomplexan pathogens such as Cryptosporidium parvum. Mechanistically, related studies suggest MMV009085 may interfere with hexose transport–dependent metabolism, likely acting as a glucose transporter mimic or inhibitor, thereby disrupting parasite energy uptake and intracellular replication.

HDB-1 is a P2Y14R inhibitor for ameliorating liver fibrosis by suppressing hepatic stellate cell activation. HDB-1 demonstrates exceptional potency (IC50 = 0.026 nM) and superior metabolic stability. HDB-1 exhibited antihepatic fibrosis activity both in vivo and in vitro. Mechanistically, HDB-1 inhibits P2Y14R-mediated signaling by suppressing the PKA/Raf1/MEK/ERK cascade, thereby preventing the activation of hepatic stellate cells-the central pathological event in fibrosis development.

PROTAC BCL6 Degrader-3 (compound 92) is a BCL6 PROTAC degrader. PROTAC BCL6 Degrader-3 can be used for the study of cancer or an autoimmune disease.

UR778Br is a small-molecule inhibitor targeting the GTPase-activating protein–related (GRD) domain of IQGAP1, with demonstrated anti-leukemia activity in both in vitro and in vivo preclinical models. In human AML cell lines (e.g., MOLM13, MV4-11, THP-1, U937) and primary AML blasts, UR778Br shows dose-dependent suppression of proliferation (effective low-µM range), induces G2/M cell-cycle arrest, and triggers apoptosis.

JT71 is a small-molecule inhibitor of the bacterial transcription regulator MrkH, showing potent anti-biofilm and anti-virulence activity in both in vitro and in vivo-relevant infection models. In vitro, JT71 inhibits MrkH-mediated transcription of the mrkA operon and suppresses type 3 fimbriae production in Klebsiella pneumoniae, resulting in ~50% reduction of mrkA promoter activity at 50 μM without affecting bacterial viability. It significantly reduces biofilm formation across multiple clinical and multidrug-resistant K. pneumoniae isolates as well as Citrobacter koseri, and downregulates both mrkH and mrkA expression, confirming dual transcriptional suppression. Mechanistically, JT71 binds to MrkH and interferes with c-di-GMP–dependent activation and/or DNA-binding activity, disrupting fimbriae assembly and surface adhesion.

NSC73306 is a cell penetrant, cytotoxic agent that exhibits greater toxicity against cells expressing functional P-gp (P-glycoprotein) than against other cells. Irrespective of variations in cell line background, NSC73306 consistently demonstrated a Pgp-potentiated MDR-selective toxicity.

HBC599 is a HBC analog. HBC is nonfluorescent in solution, but emits strong fluorescence upon forming tight complex with Pepper RNA aptamer. HBC-Pepper complex can be used to visualize RNA dynamics in live cells.