| DC60794 |
SABA1
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SABA1 is an antibacterial agent effective against Pseudomonas aeruginosa and Escherichia coli. It works by inhibiting biotin carboxylase (BC), an enzyme that catalyzes the first step of the acetyl-CoA carboxylase (ACC) reaction, a process essential for bacterial fatty acid synthesis. What makes SABA1 particularly notable is its atypical inhibition mechanism: it binds to the biotin binding site of BC in the presence of ADP. This unique mode of action distinguishes SABA1 as a promising candidate for the development of new antibiotics, offering a potential solution to the growing challenge of antimicrobial resistance. |
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| DC60795 |
Boscalid
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Boscalid is a broad-spectrum fungicide widely used in agriculture to protect crops from fungal diseases. Its bioactivity stems from its ability to inhibit fungal respiration by targeting succinate dehydrogenase (Complex II) in the mitochondrial electron transport chain. By binding to this enzyme, Boscalid blocks electron transfer, disrupting ATP synthesis and energy production in fungal cells, ultimately leading to their death. This mechanism makes Boscalid highly effective against a wide range of fungal pathogens, including Botrytis, Alternaria, Sclerotinia, and powdery mildew species. |
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| DC67295 |
Lipid MK16
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MK16 is a novel blood-brain barrier (BBB)-crossing lipid nanoparticle (BLNP) platform developed for efficient mRNA delivery to the central nervous system (CNS). Designed through structure-guided optimization, MK16 lipids integrate a γ-secretase inhibitor-derived BBB-penetrating module (MK-0752) with ionizable amino lipids, enabling systemic mRNA transport into the brain.MK16 was selected from a library of 72 BBB-crossing lipids synthesized by conjugating small-molecule BBB transporters (e.g., L-DOPA, D-serine, MK-0752) with lipid tails. Structural refinements, including acetal-functionalized alkyl chains and optimized lipid-to-mRNA ratios (12.5:1 w/w), enhanced brain delivery efficiency. MK16 BLNPs exhibit a particle size of ~137 nm, 85% mRNA encapsulation, and a pKa of 6.7–6.9, facilitating endosomal escape.MK16 BLNPs cross the BBB via caveolae- and γ-secretase-mediated transcytosis, as validated by inhibitor studies. In mice, intravenous MK16 BLNPs delivered mRNA broadly to neurons (7.4% GFP+), astrocytes (9.7% GFP+), and brain endothelial cells (9.2% GFP+), outperforming FDA-approved LNPs (MC3, SM-102) by 6–8-fold in brain luminescence. Functional studies in Ai14 mice demonstrated Cre mRNA-mediated tdTomato activation across the hippocampus, thalamus, and cortex, with triple dosing doubling transfection efficiency.Cocaine Addiction: MK16-delivered ΔFosb mRNA enhanced cocaine-conditioned place preference in mice, mimicking addiction-related neural plasticity.
Glioblastoma (GBM): Systemic MK16-Pten mRNA inhibited orthotopic U-118MG tumor growth, achieving 70% survival at 120 days vs. controls.
Human Translation: Ex vivo human brain slices showed ΔFOSB expression in neurons (4.0%) and astrocytes (6.5%), confirming clinical potential.MK16 BLNPs exhibited minimal toxicity in multi-dose regimens, with normal blood biomarkers, cytokine levels, and histopathology. Unlike MK-0752, MK16 did NOT alter NOTCH pathway genes, mitigating off-target risks. |
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| DC60796 |
HIFN
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HIFN (2-fluoro-N-(2-(5-hydroxy-1H-indol-3-yl)ethyl)nicotinamide) is a synthetic small-molecule agonist of the tropomyosin-related kinase B (TrkB) receptor, designed to mimic brain-derived neurotrophic factor (BDNF) signaling. Structurally, HIFN replaces the six-membered lactam ring of its parent compound HIOC with a fluoropyridine moiety, rendering it achiral and configurationally stable. This modification enhances binding affinity and pharmacokinetic properties.
HIFN activates TrkB by inducing receptor dimerization and phosphorylation, triggering downstream survival pathways (PI3K/Akt, MAPK/Erk). In vitro, HIFN outperforms HIOC in TrkB activation (10 nM concentration) in primary neurons and NIH-3T3-TrkB cells. In vivo, systemic administration of HIFN (30–40 mg/kg) mitigates blast-induced retinal ganglion cell (RGC) degeneration and preserves visual function (contrast sensitivity, acuity) in mice for up to 8 weeks post-injury. Its effects are TrkB-dependent, as co-treatment with the TrkB antagonist ANA-12 abolishes neuroprotection.
HIFN exhibits a critical therapeutic window of ≤3 hours post-injury and dose-dependent efficacy, with no toxicity observed at 600 mg/kg (acute) or 40 mg/kg/day (40-day chronic). Safety assessments reveal no histopathological or biochemical abnormalities in vital organs.
By combining potent TrkB activation, blood-retina barrier penetration, and a robust safety profile, HIFN emerges as a promising therapeutic candidate for traumatic optic neuropathy and broader CNS disorders involving TrkB dysregulation. |
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| DC67296 |
Galnac GLS-15
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GLS-15 is a novel GalNAc-derived small molecule structure designed for siRNA delivery. |
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| DC67297 |
SM6.1 (αvβ6 ligand)
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SM-6.1 is a small molecule delivery vehicle developed by Arrowhead, targeting αvβ6, which selectively delivers therapeutic siRNA to lung epithelial cells and mediates durable gene silencing post-inhalation. |
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| DC67298 |
Lipid 5D8
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Lipid 5D8 is a novel biodegradable ionizable lipid (IL) developed through a combinatorial chemistry strategy to overcome the limitations of conventional lipid nanoparticles (LNPs) in mRNA delivery. Synthesized via a one-step, solvent-free Michael addition reaction between amine and thiol monomers, 5D8 features asymmetric lipid tails and a biodegradable ester backbone, ensuring both structural versatility and reduced toxicity. In preclinical studies, 5D8-based LNPs demonstrated exceptional liver-targeting efficiency and mRNA delivery performance. A single intravenous dose (1 mg/kg) achieved 61% CRISPR-Cas9-mediated editing of the TTR gene in mice, reducing serum TTR protein by 90%, outperforming benchmark lipids like C12-200 (51% editing). Moreover, 5D8 enabled efficient delivery of base editors (ABE8.8 and CBE4max), achieving 42% PCSK9 editing (74% serum protein reduction) and correcting hereditary tyrosinemia in mice, significantly extending survival. Beyond gene editing, 5D8 LNPs effectively delivered siRNA (complete serum TTR clearance at 0.05 mg/kg) and enhanced hepatocyte targeting by enriching apolipoprotein E on particle surfaces. Crucially, 5D8 exhibited superior biocompatibility with no hepatotoxicity (normal ALT/AST levels), contrasting traditional LNPs. Its rapid biodegradability and "plug-and-play" design make 5D8 a versatile platform for mRNA therapeutics, holding broad potential for treating genetic disorders, cardiovascular diseases, and beyond. This innovation represents a critical advancement toward safer, high-efficiency clinical translation of gene-editing therapies.L |
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| DC67299 |
N4-Acetylcytidine triphosphate sodium
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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. |
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| DC67300 |
FBnG
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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. |
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| DC67301 |
3-sucCA
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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. |
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| DC67302 |
KAT modulator-1
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KAT modulator-1 (Compound 3) is a novel modulator of lysine acetyltransferases (KATs) with a unique mechanism of action. It specifically interacts with the full-length p300 protein but does not engage with its isolated catalytic domain, highlighting its distinctive binding properties. This compound serves as a valuable tool for epigenetics research, enabling the exploration of p300's role in chromatin remodeling, gene regulation, and other epigenetic processes. Its selective interaction with full-length p300 offers insights into the structural and functional complexities of KATs, paving the way for the development of targeted epigenetic therapies. |
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| DC67303 |
I-152
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I-152 is a novel conjugate composed of N-acetyl-cysteine (NAC) and cysteamine (MEA), designed to harness the synergistic effects of these two bioactive compounds. It demonstrates the ability to activate key cellular signaling pathways, including NRF2 and ATF4, which are involved in oxidative stress response and cellular homeostasis. Additionally, I-152 exhibits potent anti-proliferative properties, making it a promising candidate for research in conditions characterized by uncontrolled cell growth, such as cancer. This unique combination of NAC and MEA in I-152 offers a multifaceted approach to modulating cellular pathways and addressing pathological processes. |
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| DC67304 |
JMV6944
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JMV6944 is a potent agonist of the pregnane X receptor (PXR), demonstrating its ability to competitively inhibit the binding of the human PXR ligand-binding domain (LBD) with an IC50 value of 680 nM. This compound effectively induces the expression of CYP3A4 mRNA in freshly isolated primary human hepatocyte cultures, highlighting its role in modulating drug metabolism and detoxification pathways. JMV6944 serves as a valuable tool for investigating PXR-mediated transcriptional regulation and its implications in xenobiotic metabolism, liver function, and therapeutic interventions. Its dual functionality as a PXR agonist and CYP3A4 inducer underscores its potential in both research and drug development. |
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| DC67305 |
BMS-1166-N-piperidine-COOH
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BMS-1166-N-piperidine-COOH, a derivative of the potent PD-1/PD-L1 interaction inhibitor BMS-1166, serves as a key component in the design of PROTAC PD-1/PD-L1 degrader-1 (HY-131183). By binding to an E3 ligase ligand via a linker, this moiety facilitates the targeted degradation of PD-1/PD-L1, offering a novel approach to modulate immune checkpoint pathways. BMS-1166 itself is a highly effective inhibitor of the PD-1/PD-L1 interaction, with an IC50 of 1.4 nM, and it counteracts the immune-suppressive effects of the PD-1/PD-L1 checkpoint on T cell activation. This innovative strategy combines the inhibitory potency of BMS-1166 with the degradation capability of PROTAC technology, providing a promising tool for advancing cancer immunotherapy and immune-related research. |
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| DC60798 |
6-[1-(Hexyloxy)ethoxy]hexanal(MK16 tail)
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6-[1-(Hexyloxy)ethoxy]hexanal is the tail fragement for MK16.MK16 is a novel blood-brain barrier (BBB)-crossing lipid nanoparticle (BLNP) platform developed for efficient mRNA delivery to the central nervous system (CNS). |
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| DC67306 |
1-Propanethiol, 3-(dimethylamino)-, 4-methylbenzenesulfonate (1:1)
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| DC67307 |
1-Piperazineethanamine, N-(phenylmethyl)-
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| DC67308 |
CbzNH-PEG1-Br
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| DC67309 |
H2N(CH2)3N(tert-butoxycarbonyl)(CH2)4NH2
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| DC67310 |
1,4-Piperazinediethylamine
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| DC67311 |
1-Propanaminium, 2,3-dihydroxy-N,N,N-trimethyl-, chloride (1:1), (2R)-
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| DC67312 |
1-Propanethiol, 3-(diMethylaMino)-, hydrochloride
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| DC67313 |
6-Oxohexyl 2-hexyldecanoate
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