Syn-TEF1 is a molecule actively enables transcription across repressive GAA repeats that silence frataxin expression in Friedreich's ataxia, license transcription elongation at targeted genomic loci.

Aca-Bio is a specialized chemical probe designed for the specific enrichment and detection of lysine acetoacetylation (Kacac). Its core architecture consists of a hydroxylamine group, a polyethylene glycol (PEG) linker, and a biotin tag. Functionally, the hydroxylamine group selectively reacts with the unique ketone carbonyl moiety of Kacac under physiological conditions to form a stable, covalent oxime bond. This specific targeting allows Aca-Bio to accurately label Kacac-modified peptides in complex biological samples. Subsequently, the attached biotin tag enables efficient isolation of these labeled peptides via streptavidin-mediated affinity purification. A critical feature of Aca-Bio is its pH-responsive reversibility; the oxime bond remains stable at neutral pH but undergoes controlled cleavage in acidic environments. This allows for the gentle and effective elution of enriched peptides, facilitating subsequent mass spectrometry analysis. By overcoming the limitations of traditional antibody-based methods, Aca-Bio serves as a highly specific and sensitive tool for global Kacac substrate profiling.

S-Ac7-DOg​​ is an ​​ionizable lipid​​ engineered for optimized mRNA delivery to the retina, featuring a ​​sulfur-based ester bond​​ (S-Ac) and ​​dual oleyl glyceride chains​​ (DOg). Its pKa (~6.74) is finely tuned to enhance ​​endosomal escape​​ in acidic environments, enabling efficient cytosolic mRNA release. Unlike traditional lipids (e.g., C12-200, MC3), S-Ac7-DOg incorporates ​​biodegradable ester linkages​​ that hydrolyze intracellularly, minimizing lipid accumulation and reducing innate immune activation. In vitro, S-Ac7-DOg LNPs achieved >80% transfection efficiency in retinal cells (ARPE-19, MIO-M1) with ​​negligible cytokine secretion​​, outperforming MC3 and rivaling C12-200 while avoiding the latter’s high immunogenicity. In vivo, intravitreal delivery in mice showed ​​robust protein expression​​ in the optic nerve head (ONH) and Müller glia (75–100% of eyes), sustained for ≥7 days. Critically, it induced the ​​lowest immunogenicity​​ among tested lipids: minimal leukocyte infiltration (<1.5-fold vs. PBS), no microglial reactivity, and reduced GFAP upregulation.

CGX1321 (CGX-1321) is a nove potent and specific inhibitor of porcupine (PORCN) that inhibits Wnt pathway, block secretion of WNT proteins with IC50 of 1 nM; CGX1321 displayed >500 times selective for Porcupine over the most related target enzyme, hedgehog acyltransferase. CGX1321 increased Ki67+ and phosphohistone H3 (PH3+) cardiomyocytes in culture, up-regulated cell cycle regulating genes such as Ccnb1 and Ccne1, did not alter YAP protein phosphorylation and nuclear translocation in cardiomyocytes. CGX1321 administration blocked the secretion of Wnt proteins, and inhibited both canonical and non-canonical Wnt signaling pathways in MI injury mice. CGX1321 improved cardiac function, reduced myocardial infarct size, and fibrosis of post-MI hearts. CGX1321 significantly increased newly formed cardiomyocytes in infarct border zone of post-MI hearts, evidenced by the increased EdU+ cardiomyocytes.

BiP-20 is a branched ionizable phospholipid identified as a lead compound for efficient hepatic mRNA delivery.BiP-20 is a novel, efficient, and safe liver-targeted LNP delivery vehicle. With an ideal pKa of 6.56, it achieves highly efficient liver targeting and endosomal escape primarily through the ApoE/LDL-R pathway. It demonstrates exceptional performance in gene editing at very low doses: for CRISPR-Cas9-mediated editing of TTR, a 10 μg dose achieved ~64% efficiency, which is 8-fold higher than the clinical benchmark lipid LP-01. In Prime Editing targeting the PCSK9 gene, its efficiency (4.30%) also significantly surpassed that of MC3, SM102, and LPO1. Furthermore, it mediates a 5.9-fold increase in hepatic protein expression compared to MC3. Safety assessments indicate it does not induce liver function abnormalities, showing strong therapeutic potential.

SH-273 is a dual targeting compound that stimulates STING to activate myeloid cells and inhibits PI3Kγ to decrease Breg cell populations.

VVD-065 is a first-in-class NRF2 inhibitor that acts via an unprecedented allosteric molecular glue mechanism. VVD-065 specifically and covalently engages Cys151 on KEAP1, which in turn promotes KEAP1-CUL3 complex formation, leading to enhancement of NRF2 degradation.

113-AA-C8C14 is a leading β-hydroxy thioether-derived ionizable lipid that forms lipid nanoparticles (LNPs) for highly efficient, spleen-tropic mRNA delivery. Its key function is to enable targeted gene expression in the spleen, achieving a 57-fold increase in mRNA delivery compared to benchmark formulations. This capability allows it to efficiently engineer chimeric antigen receptor (CAR) T cells directly in vivo. Specifically, when loaded with CD19-CAR mRNA, 113-AA-C8C14 LNPs generate 2.7 times more CAR T cells, enhance effector cytokine production, and significantly inhibit tumor growth in mouse models, demonstrating great potential for in vivo CAR T cell therapy.

T2M-010 is a potent, brain-penetrant TREM2 agonist (Kd = 0.83 μM). T2M-010 activates receptor-proximal signaling, inducing SYK phosphorylation in TREM2-expressing cells, and promotes microglial phagocytosis. T2M-010 can be used for the study of protective microglial responses relevant to Alzheimer’s disease (AD).

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.

CL15F6 is an ionizable cationic lipid (pKa = 6.75).1 It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA and polymer-lipid hybrid nanoparticles for the delivery of plasmid DNA in vitro.1,2

Novel sialidase-activatable fluorescence probe with improved stability for the sensitive detection of sialidase

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.

Tri-b-GalNAc-b-Ala-PEG3-MAL contains three terminal beta-N-acetylgalactosamine (GalNAc) sugars arranged in a trivalent configuration, facilitating high-affinity binding to the asialoglycoprotein receptor (ASGPR) expressed on hepatocyte surfaces. The crucial functional group is a terminal maleimide (MAL) moiety, enabling chemoselective bioconjugation with biomolecules harboring thiol (-SH) groups. This strategy allows for site-specific conjugation under mild conditions, valuable for developing targeted conjugates in disciplines like targeted drug delivery and antibody-drug conjugate (ADC) development for hepatocellular carcinoma.

A potent, orally bioavailable, dual Src/Abl kinase inhibitor with IC50 of 0.5, 0.4, 0.5 and <1 nM for Src, Lck, Yes and Bcr-Abl, respectively.

PAA-38 is a highly potent selective inhibitor targeting bacterial prolyl-tRNA synthetase (ProRS). PAA-38 againsts Pseudomonas aeruginosa ProRS (PaProRS) with a Kd value of 0.399 nM and an IC50 value of 4.97 nM. PAA-38 againsts human cytoplasmic ProRSs (HsProRS) with an IC50 value of 35.5 nM. PAA-38 demonstrates an in vitro antibacterial activity of minimum inhibitory concentration (MIC) = 4-8 μg/mL.

CSP1 is a potent and selective ComD1 receptor agonist, with an IC50 of 10.3 nM. CSP1 is a major variants of competence-stimulating peptide (CSP), and it can regulate genetic transformation of S. pneumonia by modulating quorum sensing (QS). CSP1 can act as an antibacterial agent.

PAMP-12(human, porcine) is a major component of immunoreactive (ir)-PAMP, is processed from the adrenomedullin precursor, is a potent hypotensive peptide and participates in cardiovascular control.

PACAP-38 (31-38), human, mouse, rat is a fragment containing the C-terminal 31-38 fragment of PACAP (1-38), human, ovine, rat (HY-P0221). PACAP-38 (31-38), human, mouse,

Nedisertib (M3814, MSC2490484A) is a highly potent, selective, orally bioavailable inhibitor of DNA-PK.

M10 is a piperazine-derived bis-tertiary amine ionizable lipid. With an optimal pKa of 6.56, it enables efficient liver-targeted CRISPR/Cas9 delivery, achieving durable PCSK9 silencing and LDL-C reduction after a single dose, alongside a favorable safety profile.

Lipid M3 is a novel ionizable lipid. Lipid M3's primary role is to enable the efficient co-encapsulation and delivery of CRISPR/Cas9 components—Cas9 mRNA and sgRNA targeting the VEGFA gene—into human retinal endothelial cells. M3 facilitates critical steps for successful gene editing, including stabilizing the nucleic acid cargo, promoting cellular uptake, and enabling effective endosomal escape to release the payload into the cytoplasm. This results in high gene-editing efficiency (indel frequency ~28.7%). A single intravitreal injection of the M3-F4 LNP carrying this CRISPR system demonstrated potent therapeutic effects in mouse models of diabetic retinopathy by significantly inhibiting pathological neovascularization and vascular leakage, while maintaining excellent biocompatibility.

Cell permeable derivative of calcein, becoming fluorescent on hydrolysis

Calcium Green 1AM is a cell-permeant fluorescent calcium indicator (Excitation 506 nm; Emission 531 nm). Calcium Green 1AM is converted to the fluorescent calcium indicator by intracellular esterases.

Rhod-2 potassium is a cell impermeant, red fluorescent calcium indicator. Rhod-2 potassium exhibits a significant shift in fluorescence intensity upon calcium binding (ex max=549 nm; calcium-free v. ex/em max=552/581 nm; calcium-bound). Unlike the UV-excitable indicators Fura-2 and Indo-1 (HY-D0121), there is no accompanying spectral shift.

Rhod-2 is a high-affinity visible light excitation wavelength Ca2+ fluorescent probe, Rhod-2, AM is an acetyl methyl ester derivative of Rhod-2, which has cell membrane permeability and can easily enter cells with simple culture. Once it enters the cell, it is sheared by its lactesterase to produce Rhod-2 without membrane permeability, which remains in the cell to perform the corresponding physiological functions. Maximum excitation/emission wavelength: 549/578 nm.

Fluo-3 pentaammonium is a cell-impermeable calcium ion (Ca2+) indicator. Fluo-3 pentaammonium itself is not fluorescent, but it becomes fluorescent after binding to calcium ions (Ex/Em = 488/525 nm).

Fluo-3 pentapotassium is a cell-impermeable calcium ion (Ca2+) indicator. Fluo-3 pentapotassium itself is not fluorescent, but it becomes fluorescent after binding to calcium ions (Ex/Em = 488/525 nm).

Fluo-3 is a cell-impermeable calcium ion (Ca2+) indicator. Fluo-3 itself is not fluorescent, but it becomes fluorescent after binding to calcium ions (Ex/Em = 488/525 nm).