PPIL4 is a member of the cyclophilin family, characterized by its peptidyl-prolyl cis–trans isomerase activity, which facilitates the folding of target proteins through accelerated rotation around proline imidic peptide bonds. In addition to this isomerase function, PPIL4 harbors an RNA-recognition motif (RRM), implicating it in post-transcriptional processes. This dual-domain architecture suggests that PPIL4 not only participates in protein conformation quality control but also interfaces with the spliceosomal machinery and broader RNA metabolic pathways, potentially coupling protein folding events with co-transcriptional or post-transcriptional regulation.

AX6​​ is an ionizable lipid in the ​​F32 LNP​​ formulation, engineered by ReNAgade/Orna Therapeutics for targeted mRNA delivery to T cells. AX-6's unique ​​bridged bicyclic/polycyclic core​​ with a ​​tertiary amine group​​ enables pH-dependent protonation and endosomal escape, while ​​C14-C18 hydrophobic tails​​ (optionally branched/fluorinated) enhance bilayer stability and mRNA encapsulation. Demonstrating ​​exceptional T-cell tropism​​, AX6 achieves high transfection efficiency in CD4+/CD8+ T cells (validated in NHP/humanized models) with minimal toxicity. Compared to clinical benchmarks (SM-102, ALC-0315), its rigid core offers superior ​​serum stability​​ and ​​immune-cell specificity​​, positioning it as an ideal candidate for ​​CAR-T/NK therapies​​ and ​​next-gen vaccines​​. The F32 LNP system's proven efficacy (e.g., in vivo B-cell depletion) underscores AX 6's transformative potential for ​​cell engineering​​ and ​​immunotherapies​​.

Alogabat (example 8) is a GABAA α5 receptor positive allosteric modulators (PAMs) (extracted from patent WO2018104419A1).

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.

KPT-6566 is a covalent PIN1 inhibitor selectively targets cancer cells by a dual mechanism of action.

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

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.

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.

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.

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.

Natamycin is a naturally occurring polyene macrolide antifungal agent produced by Streptomyces natalensis, exhibiting potent in vitro and in vivo activity against a broad spectrum of yeasts and filamentous fungi. In vitro studies show strong inhibition of Candida spp., Aspergillus spp., and other food- and ocular-relevant fungi, with reported MIC values typically in the low µg/mL range (often ~1–10 µg/mL depending on species and strain).

Lixisenatide is a glucagon-like peptide-1 (GLP-1) receptor agonist. Lixisenatide inhibits the inflammatory response through down regulation of pro-inflammatory cytokines, and suppresses of the Akt-MEK1/2 signaling pathway. Lixisenatide can inhibit oxidative stress, mitochondrial dysfunction and apoptosis. Lixisenatide can be used for the researches of inflammation, metabolic disease, neurological disease and cardiovascular disease, such as rheumatoid arthritis, diabetes, Alzheimer's disease and atherosclerosis.

dIRF4-2 is a first-in-class proteolysis-targeting chimera of IRF4, by linking (S)-H1 to E3 ligase ligands of cereblon. dIRF4-2 achieves highly selective IRF4 knockdown (DC50 ~2.2–2.3 µM) without degrading off-target neosubstrates such as IKZF1/3 or GSPT1, and demonstrates strong cytotoxic effects across all multiple myeloma cell lines tested in vitro.

dHuR-2 is the optimized analogue of dHuR-1 and exhibits exceptional nanomolar potency (DC50=3.8 nM) and oral bioavailability, effectively suppressing BRAF-mutant colorectal cancer by inducing unique BRAF mRNA exon skipping and overcoming existing inhibitor resistance.

dHuR-1 is the first-in-class molecular glue degrader that recruits the CRBN E3 ligase to selectively eliminate the oncogenic RNA-binding protein HuR.

Ganaxolone, also known as CCD 1042 and C1042, is a CNS-selective GABAA modulator that acts on well-characterized targets in the brain known to have anxiolytic and anticonvulsant effects. Ganaxolone protects against seizures in diverse animal models, including the pentylenetetrazol, 6 Hz and amygdala kindling models. Ganaxolone is a positive allosteric modulator of the action of the GABAA receptor and, unlike benzodiazepines, there does not appear to be tolerance to the anticonvulsant effects of ganaxolone.

SBI-810 hydrochloride is a functionally selected β-arrestin-biased neurotensin receptor 1 (NTSR1) allosteric modulator. SBI-810 hydrochloride modulates NTSR1 G protein signaling in a G protein-specific manner in the presence of the endogenous ligand, neurotensin (NT). SBI-810 hydrochloride fully antagonizes NT-induced activation of Gq, partially antagonizes NT-induced activation of Gi1 and is permissive of NTSR1 activation of GoA and G12.

SBI-810 is a functionally selected β-arrestin-biased neurotensin receptor 1 (NTSR1) allosteric modulator. SBI-810 modulates NTSR1 G protein signaling in a G protein-specific manner in the presence of the endogenous ligand, neurotensin (NT). SBI-810 fully antagonizes NT-induced activation of Gq, partially antagonizes NT-induced activation of Gi1 and is permissive of NTSR1 activation of GoA and G12.

KOTX1 is a potent, selective and reversible ALDH1A3 inhibitor with IC50 of 5.14 nM by Aldefluor assay in A375 cells.