A28-C6B2 is a biodegradable ionizable lipid specifically engineered for spleen-targeted delivery. Through its unique branched structure, it bypasses hepatic uptake to achieve highly efficient transfection of F4/80+ macrophages and CD11c+ dendritic cells within the spleen. This lipid remains neutral in the bloodstream to minimize non-specific interactions, while undergoing protonation in the acidic environment of the endosome to facilitate nucleic acid release, thereby significantly enhancing the potency of mRNA vaccines and immunotherapies.

MMG-1, a monomycoloyl glycerol, is a C-type lectin receptor agonist. MMG-1, a lipid membrane stabilizer, is an immunopotentiator in liposomes. MMG-1 can be used to prepare lipid nanoparticles for drug delivery.

Triantennary GalNAc ligand is known to bind Asialo Glycoprotein Receptor (ASGPR). The ligand is functionalized with dibenzocyclooctyne for click chemistry.

Triantennary GalNAc ligand is known to bind Asialo Glycoprotein Receptor (ASGPR). The ligand is functionalized with dibenzocyclooctyne for click chemistry

(β-D-GalNAc-sp)3-NHC(O)-(CH2)2-NHC(O)-PEG3-(CH2)2-N3 Tris-β-D-GalNAc ligands bind with asialoglycoprotein receptors (ASGPR) that are highly expressed on hepatocytes resulting in rapid endocytosis of the ligand along with conjugated payloads

An amine-reactive asialo glycoprotein receptor (ASGPR) ligand.

VL422 is a novel ionizable cationic lipid, as a high-performance "molecular engine" for next-generation Lipid Nanoparticles (LNPs), specifically engineered for the precise delivery of CRISPR base editors and mRNA. Its sophisticated chemical architecture is designed to remain neutral in systemic circulation for enhanced safety, while rapidly protonating within the acidic cellular environment to trigger efficient endosomal escape and cargo release. Validated by groundbreaking research in liver-targeted gene silencing, VL422 has become a critical benchmark molecule for developing permanent, transformative therapies for cardiovascular and metabolic diseases.

H5T5 is a leading ionizable lipid nanoparticle (LNP) formulation optimized for in vivomRNA delivery, featuring a pKa of 6.51, a size of ~154 nm, and a narrow polydispersity index (PDI) of 0.05. It demonstrated superior in vitromRNA transfection efficiency in primary immune cells, such as bone marrow-derived macrophages. Following intravenous administration, H5T5 exhibits precise organotropism, predominantly targeting the spleen and bone marrow, where it effectively delivers mRNA to a broad spectrum of immune cells, including macrophages, dendritic cells, T cells, B cells, and NK cells. This capability enables its core application: the in vivogeneration of "pan-CAR" immune cells. When loaded with anti-HER2 CAR mRNA, the H5T5-based therapy achieved potent tumor regression and prolonged survival in multiple solid tumor models. Preliminary safety assessments indicated a manageable cytokine profile and no significant organ toxicity, positioning it as a promising platform for in vivocell engineering.

Sail Lipid 2308​ is a novel ionizable lipid targeting to spleen developed by Sai Biomedicine.As described on US20250205167A1, Lipid 2308 was designed with a ​​piperidine core​​ (6-membered ring) and asymmetric C17/C11 chains, this lipid achieves unprecedented ​​spleen-specificity​​. It demonstrates dominant spleen accumulation (Spleen RLU: ​​7.8E+06​​, 91.8% of total signal) with a record ​​spleen-to-liver ratio of 112.7​​ (9× higher than 2231). Despite lower protein expression (hEPO: 11,000 ng/mL), near-zero liver uptake (Liver RLU: 66,000) makes Lipid 2308 unparalleled for vaccine/immunotherapy applications targeting splenic immune cells.

CICL-242​ is a constrained ionizable cationic lipid highlighted in patent US 20250127728A1 developed by Capstan as a promising candidate for advanced therapeutic delivery, particularly in stem cell and gene editing applications. Its structure features a rigid amine headgroup similar to CICL-207, which likely facilitates efficient endosomal escape and reduces non-specific uptake, enhancing targeted nucleic acid delivery. Although detailed performance data is not fully disclosed in the patent, CICL-242 is explicitly synthesized and included in gene editing experimental systems (e.g., CRISPR-Cas9 workflows), suggesting its potential for high-efficiency transfection in hard-to-transfect cells​ like hematopoietic stem cells (CD34⁺). This makes it a strong candidate for ex vivo cell engineering and regenerative medicine, where precision and low off-target effects are critical. While further validation is needed to quantify its efficacy and safety profile, CICL-242 represents a strategic innovation in the lipid library for next-generation genetic therapies.

CHCha-10 is an amino acid-derived ionizable lipid engineered for pulmonary gene therapy. Its optimal surface charge (-0.126 mV) enables efficient mucus penetration, while a pKa of ~7.0 facilitates endosomal escape. Its unique conical structure promotes membrane fusion and mRNA release. In animal models, CHCha-10-based LNPs achieved highly efficient and specific editing of lung epithelial cells, particularly stem-like basal cells (~44%), with effects persisting through tissue renewal. It also demonstrated excellent efficacy and safety in ferrets, a model closely mimicking human lung physiology.

ISM5939 is an orally active and selective ENPP1 inhibitor with an IC50 of 0.63 nM for 2,3-cGAMP degradation and 9.28 nM for ATP hydrolysis. ISM5939 has antitumor activity.

BioE-1197 is a novel Pask inhibitor. BioE-1197 is a compound that enhances T cell function. Functionally, treatment with BIOE-1197 significantly boosts the ability of effector T cells (such as CD8+, Th1, and Th2 cells) to produce lineage-specific cytokines upon restimulation. BIOE-1197 is considered a novel agent with potential applications in tumor immunotherapy and enhancing vaccine efficacy.

Emupertinib (example 37) is a potent EGFR inhibitor with IC50 values of <0.3 nM, 0.52 nM, 0.5 nM, 0.69 nM and 0.92 nM for EGFR (d746-750/T790M/C779S), EGFR (L858R/T790M/C797S), EGFR (d746-750/C797S), EGFR (L858R/C797S), and EGFR (wild-type), respectively.

KVS0001 is a specific and bioavailable small molecule inhibitor of SMG1 kinase, disrupts nonsense mediated decay (NMD).

AURKB PROTAC MS44 is a potent, selective, first-in-class Aurora B kinase (AURKB) PROTAC degrader with DC50 of 103 nM in HPAF-11 cells, Dmax of 89%.

STING inhbiitor, which inhibited the activation of the STING signal pathway and to prevent or treat a STING-​mediated disease.

RO5126766(CH5126766) is a Raf/MEK dual kinase inhibitor.

GSK898 is a potent, highly selective kynurenine monooxygenase (KMO) inhibitor with pIC50 of 8.8.

seMpai is a high-performance near-infrared (NIR) luciferin analogue engineered for enhanced deep-tissue bioluminescence imaging, producing a robust 675 nm emission comparable to top-tier substrates. In vitro, it effectively supports mutant luciferase systems like Akaluc, while in vivo, it enables detection of pulmonary micro-metastases, overcoming traditional D-luciferin limitations. The compound features an optimized, neutral pH formulation that reduces hepatic background, ensuring superior signal-to-noise ratios without significant toxicity, making it ideal for longitudinal studies. For optimal solubility and performance, seMpai is a critical tool for preclinical imaging and drug discovery. Learn more at MedKoo. NOTE. seMpai (Cat#465342) is the free acid form and is less water soluble. seMpai Sodium (CAT#333158) is water soluble.

YXN47931, also known as Paclitaxel succinate sulfo-NHS ester, is a paclitaxel derivative with a succinic acid linker, in which the carboxy group is activated with an sulfo-NHS ester. The sulfo-NHS ester group is highly reactive to amino group or hydroxy group, and can be used to conjugate with other molecules such as peptides, proteins, antibodies or enzymes, or polymers. Paclitaxel-Succinic acid is a useful agent to make Paclitaxel-conjugate for drug delivery, nanodrug research. Note: Paclitaxel-Succinic acid (CAT#620101), Paclitaxel-Succinate-NHS (also called DCN10809, CAT#408092) are also in stock. This product has no formal name at the moment. For the convenience of communication, a temporal code name was therefore proposed according to MedKoo Chemical Nomenclature (see web page: https://www.medkoo.com/page/naming). NOTE: YXN47931 sodium (CAT#408203) is water soluble. YXN47931 free acid (CAT#408211) has very low solubility in water.

AM-001, a small-molecule EPAC1 (exchange protein directly activated by cAMP) inhibitor, showed potent in vitro antiviral activity against SARS-CoV-2 and influenza A in cultured cells. In Vero E6 cells, AM-001 reduced SARS-CoV-2 viral RNA and infectious titres with IC₅₀ ≈ 355–388 nM and in human lung Calu-3 cells with IC₅₀ ≈ 147–167 nM, achieving ~10⁴-fold suppression at 20 µM without cytotoxicity (CC₅₀ > 40 µM; selectivity index >100). AM-001 also inhibited influenza A replication in Calu-3 cells with IC₅₀ ~1 µM. These effects reflect host-targeted EPAC1 inhibition rather than direct virucidal action. No in vivo animal model data were included in the study’s antiviral evaluation.

CT-996 is an orally active GLP-1RA agonist, with an EC50 of 0.49 nM. CT-996 reduces the β-arrestin recruitment and GLP-1R internalization. CT-996 suppresses postprandial blood glucose following a mixed meal tolerance test (MMTT) in mice expressing the human GLP-1 receptor and enhances glucose stimulated insulin secretion (GSIS) during an intravenous glucose challenge in obese monkeys. CT-996 can be used for the study of type 2 diabetes (T2D) and obesity.

Selumetinib (AZD6244) is selective, non-ATP-competitive oral MEK1/2 inhibitor, with an IC50 of 14 nM for MEK1. Selumetinib (AZD6244) inhibits ERK1/2 phosphorylation.

Opitor-0 is a potent and selective inhibitor of the mitochondrial GTPase Optic atrophy protein 1 (OPA1) with an IC50 of approximately 3 µM. In cellular studies, Opitor-0 disrupts OPA1 oligomer stability, leading to mitochondrial fragmentation and remodeling of mitochondrial cristae. These structural changes facilitate the release of Cytochrome c from mitochondria into the cytosol, triggering apoptotic signaling. The compound exhibits significant pro-apoptotic and antitumor effects in cancer cell models. Importantly, Opitor-0 demonstrates strong synergistic activity with Bcl-2 inhibitors such as ABT-737 and Venetoclax, enhancing tumor cell death. These results suggest that Opitor-0 effectively targets mitochondrial dynamics and apoptosis pathways in cancer cells.

DOTA-GPC3 is a ligand for radiotracer targeting glypican GPC3 . DOTA-GPC3 molecule is consisted of a chelator DOTA for radioactive metal ions and a GPC targeting peptide for binding glypican GPC3. Glypican-3 (GPC3) is highly expressed in HCC but not in normal liver tissue, making it a promising target for PET-based molecular imaging, which may complement existing approaches for HCC diagnosis.

Moxonidine (BDF5895) hydrochloride is an orally active imidazoline type 1 receptor (I1-R) agonist. Moxonidine hydrochloride activates imidazoline I1 receptors and α2 adrenoceptors, affecting oxidized low-density lipoprotein uptake. Moxonidine hydrochloride reduces atherosclerotic lesions and lowers blood pressure. Moxonidine hydrochloride can be used in the study of hypertension, heart failure, and atherosclerosis.

DPI-4452 is a CAIX-targeting cyclic peptide with a DOTA chelator. DPI-4452 can be chelated with radionuclide for CAIX-expressing tumor PET-CT imaging. DPI-4452 was used to prepare [68Ga]Ga-DPI-4452, which is a first-in-class carbonic anhydrase IX-binding radiolabeled peptide, and is the imaging agent of a theranostic pair with [177Lu]Lu-DPI-4452, developed for selecting and treating patients with carbonic anhydrase IX-expressing tumors. [68Ga]Ga-DPI-4452 showed exceptional tumor uptake in patients with clear cell renal cell carcinoma, with very high tumor-to-background ratios and no significant adverse events, suggesting potential diagnostic and patient selection application.

Mortaparib is a dual inhibitor of mortalin-PARP1 interaction, and a p53 activating cytotoxic compound, induces activation of growth arrest and apoptosis signaling in cancer cells in vitro and in vivo.