3-Succinylated cholic acid (3-sucCA) is a microbially derived bile acid that plays a significant role in gut health and metabolic regulation. As a lumen-restricted metabolite, 3-sucCA has been shown to mitigate the progression of metabolic-associated fatty liver disease (MAFLD) to metabolic-associated steatohepatitis (MASH) in mouse models. Its protective effects are primarily attributed to its ability to reshape the gut microbiota, particularly by enhancing the growth of Akkermansia muciniphila, a beneficial bacterium associated with improved metabolic health. Notably, patients with biopsy-confirmed MAFLD exhibit reduced levels of 3-sucCA, underscoring its potential as a biomarker and therapeutic target for managing metabolic liver diseases. This unique metabolite highlights the intricate interplay between gut microbiota and liver health, offering promising avenues for intervention.

FBnG is a key component of the non-ribosomal peptide synthetase/polyketide synthase (NRPS/PKS) machinery, playing a crucial role in the biosynthesis of fabrubactin (FBN). This versatile molecule can be utilized in the synthesis of AUTAC4, specifically as part of the compound FBnG-(Cys-acetamide)-CH2-PEG3-CH2-CH2-CH2-NH2 (HY-150408). By leveraging its integration into this synthetic pathway, FBnG serves as a valuable building block for the development of AUTAC4, highlighting its potential in advancing research and therapeutic applications related to targeted protein degradation and related biological processes.

BBO-8520 represents a groundbreaking first-in-class covalent inhibitor that uniquely targets both the active (ON) and inactive (OFF) states of KRAS-G12C. Through comprehensive global cysteine proteome analysis, BBO-8520 demonstrates exceptional binding specificity to KRAS-G12C, showcasing a remarkable selectivity of over 100-fold compared to wild-type KRAS and other mutant isoforms. Notably, BBO-8520 exhibits no detectable activity against N-RAS or H-RAS, underscoring its precision in selectively inhibiting KRAS-G12C. This distinctive profile positions BBO-8520 as a promising therapeutic candidate for addressing KRAS-G12C-driven cancers, offering a novel approach to targeting this historically challenging oncogenic mutation.

N4-Acetylcytidine triphosphate sodium serves as an efficient substrate in T7 Polymerase-driven in vitro transcription reactions, demonstrating its ability to be successfully incorporated into various templates. This modified nucleotide offers a unique advantage in expanding the scope of RNA synthesis, enabling the production of acetylated RNA molecules with potential applications in research and therapeutic development. Its compatibility with T7 Polymerase highlights its utility in generating tailored RNA constructs, providing researchers with a versatile tool for exploring RNA modifications and their functional implications.

N4-Acetylcytidine triphosphate is efficiently used as a substrate in T7 Polymerase-catalyzed in vitro transcription and can be incorporated into multiple templates.

MK-0752 is a moderately potent γ-secretase inhibitor, it is used as a head group of MK16 which is a a novel blood-brain barrier (BBB)-crossing lipid nanoparticle (BLNP) platform developed for efficient mRNA delivery to the central nervous system (CNS).

NCATS-SM7270 is a highly specific small molecule inhibitor of NADPH oxidase 2 (NOX2), exhibiting an IC50 value of 2.1 µM in polymorphonuclear leukocytes (PMN). It effectively inhibits NOX2 activity in both primary human and mouse granulocytes, demonstrating its targeted and potent mechanism of action.

Endosidine 5 (ES5), is one of the most potent small molecules interferes with recycling endosomes through Annexin A6, thereby promoting the release and expression of mRNA into the cytoplasm. The delivered mRNAs is greatly enhanced via inhibition of endocytic recycling in cells and in live mice. NAV2729 (NAV) and endosidin 5 (ES5), resulted in significant enhancement (1.5–2 folds) of LNP-mediated delivery of Fluc mRNAs. Incubation of NAV and ES5 together caused modest further increases in Fluc expression in comparison to the sole application of either compound.

ART558 is a nanomolar potent, selective, low molecular weight, allosteric DNA polymerase activity of Polθ inhibitor (IC50=7.9 nM). Inhibition of TMEJ with ART558 increases the efficiency of HDR-mediated repair on Cas9-induced DSBs, most profoundly in combination with a NHEJ/DNA-PKcs inhibitor.

Bropirimine is a synthetic agonist for toll-like receptor 7 (TLR7). Bropirimine inhibits differentiation of osteoclast precursor cells into osteoclasts via TLR7-mediated production of IFN-β. Bropirimine is an orally active immunomodulator that has demonstrated anticancer activity in transitional cell carcinoma in situ (CIS) in both the bladder and upper urinary Bropirimine could enhance the transfection efficiency to 2-fold to 5-fold of Lipofectamine 3000 and LNP, all of which are currently approved for clinical use in the treatment of the most common malignant tumors.

SCH 58261 is a potent and selective A2a adenosine receptor antagonist with Ki of 2.3 nM and 2 nM for rat A2a and bovine A2a. SCH58261 could enhance the transfection efficiency to 2-fold to 5-fold of Lipofectamine 3000 and LNP, all of which are currently approved for clinical use in the treatment of the most common malignant tumors.

HSL119 is a highly effective and selective inhibitor of hormonally upregulated neu-associated kinase (HUNK), demonstrating complete suppression of HUNK kinase activity at a concentration of 1 µM in biochemical assays. This compound highlights its precision and potency in targeting HUNK.

SH-BC-893 is a water-soluble, orally bioavailable synthetic sphingolipid that triggers nutrient transporter internalization and also blocks lysosome-dependent nutrient generation pathways in cancer cells, activates protein phosphatase 2A (PP2A), leading to mislocalization of the lipid kinase PIKfyve; selectively kills cells expressing an activated form of the anabolic oncogene Ras in vitro and in vivo.SH-BC-893, increases the activity of antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) up to 200-fold in vitro without permeabilizing endosomes. SH-BC-893 treatment trapped endocytosed oligonucleotides within extra-lysosomal compartments thought to be more permeable due to frequent membrane fission and fusion events. Simultaneous disruption of ARF6-dependent endocytic recycling and PIKfyve-dependent lysosomal fusion was necessary and sufficient for SH-BC-893 to increase non-lysosomal oligonucleotide levels and enhance their activity. In mice, oral administration of SH-BC-893 increased ASO potency in the liver by 15-fold without toxicity. More importantly, SH-BC-893 enabled target RNA knockdown in the CNS and lungs of mice treated subcutaneously with cholesterol-functionalized duplexed oligonucleotides or unmodified ASOs, respectively. Together, these results establish the feasibility of using a small molecule that disrupts endolysosomal trafficking to improve the activity of oligonucleotides in extrahepatic tissues.

NAV 2729 (NAV2729) is a chemical potentiator for mRNA-LNPs, increasing the delivery efficiencies of mRNA-LNPs in vitro and in vivo.

AZD-7648 is a potent and selective DNA-PK inhibitor. AZD7648,enhances HDR efficiency in CRISPR-Cas9 gene editing by shifting DNA repair from the error-prone NHEJ pathway to the precise HDR pathway, significantly improving gene targeting outcomes in human cells for effective ex vivo gene therapies.

BP-1-102 is a potent, orally bioavailable and selective STAT3 inhibitor.BP-1-102 could enhance the transfection efficiency to 2-fold to 5-fold of Lipofectamine 3000 and LNP, all of which are currently approved for clinical use in the treatment of the most common malignant tumors.

FL-1607, a novel Fam20C inhibitor, has shown remarkable anti-proliferative effects in triple-negative breast cancer (TNBC) cells. This compound induces apoptosis and significantly reduces the migration ability of MDA-MB-468 cells, underscoring its potential as a promising therapeutic agent for TNBC treatment.

mCLB073 is an advanced, orally active small molecule agonist specifically designed to target Mtb adenylyl cyclase Rv1625c. As an optimized derivative of V-59, it demonstrates significantly enhanced potency and efficacy for in vivo applications. In cholesterol-based media, mCLB073 shows a remarkable 17-fold increase in activity against Mtb compared to its predecessor, V-59, while retaining favorable pharmacokinetic characteristics and a strong safety profile. In preclinical studies, oral administration of mCLB073 at 30 mg/kg led to a substantial reduction in Mtb colony-forming units (CFUs) in the lungs of mice, accompanied by a 45% decrease in lung pathology severity. These findings highlight its potential as a promising therapeutic candidate for tuberculosis treatment.

PIM1-IN-2 is a highly effective and ATP-competitive inhibitor of Pim-1, demonstrating a Ki value of 91 nM. Unlike traditional inhibitors, it interacts with the ATP-binding kinase hinge region without relying on the formation of standard hydrogen bonds, showcasing a unique mechanism of action. This distinctive approach highlights its potential as a promising therapeutic agent.

ATV006 is a potent, orally active antiviral agent and ester prodrugs of GS-441524. ATV006 inhibits the replication of SARS-CoV-2 and its variants. ATV006 can be used for SARS-CoV-2 research.

Pepinemab (VX 15/2503) is a human monoclonal antibody against SEMA4D, a signalling protein 4D (SEMA4D), also known as CD100, which is a regulator of neuronal development and plays a role in a variety of cellular processes. Pepinemab can be used in the study of various neurodegenerative diseases such as Alzheimer's disease by blocking the activity of SEMA4D.

Atinumab (6A3-IgG4) is an antibody. Teropavimab can be used for the research of spinal cord injury (SCI).