patient-derived

PF-9363 (CTX-3648) 是一种有效且高选择性的 KAT6A KAT6B 抑制剂，可用于癌症研究。

FGTI-2734 是 有效的RAS C-末端法尼基转移酶 (FT) 和香叶烯基转移酶-1 (GGT-1) 抑制剂，IC50s 分别为 250 nM 和 520 nM。 它可以阻断 KRAS 的膜定位，从而解决 KRAS 耐药性问题，并抑制突变的 KRAS 胰腺肿瘤。

HR68是一种抗癌化合物，是过氧化物酶体增殖物激活受体（PPAR）激动剂非诺贝酯的衍生物。它能降低LN-229胶质母细胞瘤细胞的存活率（IC50 = 1.17 µM）。HR68能够穿越血脑屏障，在对替莫唑胺耐药的原位患者衍生的异种移植（PDX）小鼠胶质母细胞瘤模型中发挥作用。

Selective peptide-based β-catenin Degrader. Comprises a β-catenin-targeted stapled peptide, xStAx, linked to a VHL-binding peptide. Reduces β-catenin levels in HEK293T cells and colorectal cancer cell lines. Selectively degrades β-catenin over other Wnt signaling pathway components. Inhibits tumor growth in APCmin + mice, with constitutively active Wnt signaling, and reduces survival of patient-derived colorectal cancer cell organoids.

CAY10722 is an inhibitor of sirtuin 3 (SIRT3), a class III HDAC (71% inhibition at 200 μM). SIRT3 is involved in modulating metabolic homeostasis as a NAD+-dependent protein deacetylase in the mitochondria. SIRT3 functions as either an oncogene or tumor suppressor, depending on cancer cell type. High SIRT3 expression in patient-derived esophageal cancer tissues is associated with shorter survival and, in mice, downregulation leads to a lower tumor load. In contrast, low SIRT3 expression in patient-derived breast cancer cells is correlated with shorter survival.

6-(1-Hydroxyethyl)-5,6-dihydrochelerythrine 显著影响细胞器特性，涉及早期内体、线粒体及自噬体（源自帕金森病患者的嗅觉细胞）。

W1131 是一种有效的STAT3抑制剂，可引发铁死亡。W1131 在胃癌皮下异种移植模型、类器官模型和 PDX 模型中抑制癌症进展。W1131 有效减轻癌细胞对 5-FU 化学耐药性。W1131 调节细胞周期、DNA 损伤反应和氧化磷酸化，包括IL6-JAK-STAT3通路和铁死亡 (ferroptosis) 通路。

DS89002333 是一种强效的、口服具有活力的 PRKACA 抑制剂 (IC50: 0.3 nM)。DS89002333 在表达 DNAJB1-PRKACA 融合基因的 FL-HCC 患者源性异种移植模型中具有良好的抗肿瘤作用。DS89002333 能够用于研究纤维化肝细胞癌 (FL-HCC)。

Promotes proteasomal degradation of Miro1 (mitochondrial Rho GTPase 1). Reduces Miro1 levels in fibroblasts from Parkinson's disease (PD) patients (IC50 = 7.8 μM). Exhibits no significant effect on related outer mitochondrial membrane protein Mitofusin. Reduces stress-induced degeneration of dopaminergic neurons derived from PD patient iPSCs. Rescues age-dependent neuronal loss and prolongs lifespan in fly PD models.

KSL-128114是一种对膜调节剂syntenin的肽抑制剂（Ki = 300 nM）。它能够依赖浓度降低A2058黑色素瘤和患者来源的多形性胶质母细胞瘤（GBM）细胞的存活率。在使用患者来源的GBM细胞并在颅内植入前用KSL-128114培养的患者衍生异种移植（PDX）小鼠模型中，KSL-128114（50 µM）提高了动物的存活时间。

FGTI-2734 mesylate, a RAS C-terminal mimetic compound with dual farnesyl transferase (FT) and geranylgeranyl transferase-1 (GGT) inhibitory activities (IC50s of 250 nM and 520 nM for FT and GGT, respectively), mitigates KRAS resistance by preventing its membrane localization, effectively impeding mutant KRAS patient-derived pancreatic tumors.

Darinaparsin is a dimethylated arsenic linked to glutathione. It is cytotoxic to DU145, LNCaP, and PC3 prostate cancer cells (IC50s = 5-10 µM) and patient-derived primary prostate cancer cells (IC50s = 2.5-20 µM), as well as Jurkat T cell lymphoma and L540 Hodgkin lymphoma cells (IC50s = 2.7 and 1.3 µM, respectively). [1][2] It decreases the tumor-initiating subpopulation in DU145 and PC3 cells and halts the cell cycle in the G2 M phase. Darinaparsin decreases transcription of Gli-2, a transcription factor that mediates Sonic hedgehog signaling, when used at a concentration of 1.5 but not 3 µM. It decreases SHP1 phosphatase activity and increases ERK phosphorylation. [2] Darinaparsin reduces tumor growth in DU145 and PC3 prostate cancer mouse xenograft models when administered at a dose of 100 mg kg every other day.[1]

Potent and selective BTK Degrader (IC50 = 5.1 nM); degrades BTK in a proteasome- and CRBN-dependent manner. Suppresses BTK signaling and proliferation in mantle cell lymphoma (MCL) cells by degrading BTK, IKFZ1, and IKFZ3 (3 validated targets in B-cell malignancies). Also degrades Ibrutinib (Cat. No. 6813) -resistant C481S-BTK mutant cancer cells. Exhibits no binding against a panel of 468 kinases at 1 μM. Reduces tumor burden and extends survival in lymphoma patient-derived xenograft models.

TH287 hydrochloride 是一种选择性MTH1抑制剂，IC50值为 0.8 nM。它可用于治疗癌症的研究。

5-Chlorouracil is a chlorinated derivative of the pyrimidine nucleoside base uracil . In vivo, it is converted into chlorodeoxyuridine, which is mutagenic and genotoxic.1 Uracil is chlorinated at the 5 position in a cell-free myeloperoxidase, peroxide, and chloride system in which hypochlorous acid is formed.2 5-Chlorouracil has been found in human neutrophils stimulated with phorbol 12-myristate 13-acetate in vitro and in inflammatory human exudate isolated from sites of superficial infection. Levels of 5-chlorouracil are increased in exudate isolated from the site of inflammation in a rat model of carrageenan-induced inflammation and in patient-derived human atherosclerotic aortic tissue.3,4References 5-Chlorouracil is a chlorinated derivative of the pyrimidine nucleoside base uracil . In vivo, it is converted into chlorodeoxyuridine, which is mutagenic and genotoxic.1 Uracil is chlorinated at the 5 position in a cell-free myeloperoxidase, peroxide, and chloride system in which hypochlorous acid is formed.2 5-Chlorouracil has been found in human neutrophils stimulated with phorbol 12-myristate 13-acetate in vitro and in inflammatory human exudate isolated from sites of superficial infection. Levels of 5-chlorouracil are increased in exudate isolated from the site of inflammation in a rat model of carrageenan-induced inflammation and in patient-derived human atherosclerotic aortic tissue.3,4 References

1,2-Dipalmitoyl-sn-glycero-3-PE-N-(cap biotin) is a biotinylated phospholipid. It has been used in PEGylated polyamidoamine-dendrimer-conjugated supported lipid bilayers (SLB) to isolate circulating tumor cells and tumor cell microembolis from patient-derived blood by antibody-coated microfluidics. [1] It has also been used as a component of SLBs to detect protein-ligand binding with ortho-conjugated Texas Red DHPE. [2] In addition, 1,2-dipalmitoyl-sn-glycero-3-PE-N-(cap biotin) has been used in SLBs partitioned into nanowells to create DNA curtains, which can be used as a high-throughput tool for detection of protein-DNA interactions at the single molecule level.[3]

VT02956 是一种LATS 抑制剂 (IC50: LATS1 为 0.76 nM，LATS2 为 0.52 nM)。VT02956 靶向Hippo 信号通路。VT02956 抑制 ER+ 乳腺癌细胞系和患者来源的肿瘤类器官的生长和 ESR1 表达。

SJ11646是一种高效的LCK（淋巴细胞特异性蛋白酪氨酸激酶）降解剂（PROTAC；DC50= 0.00838 pM）。该化合物由Dasatinib作为LCK配体，以及基于苯基谷氨酰亚胺的cereblon结合体组成。它在体外对LCK激活的T细胞急性淋巴细胞白血病（T-ALL）细胞株和初级白血病样本展现出细胞毒性。在体内，SJ11646在源自患者的T-ALL异种移植模型中显示出抗白血病效果。

YK-029A为口服活性的EGFR突变抑制剂，特异性针对EGFRT790M及EGFRex20ins。该化合物在PDX模型上对肿瘤展现良好的抑制效果，显示出明显的抗肿瘤活性。

L-778123 is an inhibitor of FPTase and GGPTase-I, which was developed in part because it can completely inhibit Ki-Ras prenylation. The combination of L-778,123 and radiotherapy at dose level 1 showed acceptable toxicity in patients with locally advanced pancreatic cancer. Radiosensitization of a patient-derived pancreatic cancer cell line was observed.

Seribantumab (MM 121) 是一种靶向 HER3 的人源化单克隆抗体，抑制 NRG1 刺激的 MCF-7 细胞生长和患者来源的乳腺和肺癌细胞的生长携带 NRG1 融合或 NRG1 扩增，抑制表达 CD74-NRG1 融合的同基因 HBEC 细胞的生长，并诱导 MDA-MB-175-VII 和 LUAD-0061AS3 细胞凋亡。

Flotetuzumab (MGD006; S80880) 是一种双特异性 CD123 CD3双亲和再靶向 (DART) 抗体。Flotetuzumab 通过同时结合靶细胞的 CD123和效应 T 细胞的CD3，而重新激活 T 细胞，导致 T 细胞介导的靶细胞的细胞毒性。Flotetuzumab 对小鼠中急性髓系白血病 (AML) 患者来源异种移植物 (PDX) 模型具有抑制效力。

ALDH3A1-IN-1 是一种小分子 ALDH3A1 抑制剂，（IC50:1.61 μM)，可用于研究高脯氨酸血症和干燥-拉森综合征。

MS40, 作为一种WDR5 PROTAC，选择性降解WDR5及免疫调节化合物(IMiDs)的新底物：CRBN和Ikaros锌指(IKZF)转录因子IKZF1与IKZF3。MS40促使WDR5降解，导致MLL KMT2A复合体从染色质上解离，从而降低H3K4me2的水平。在体外，MS40抑制了原发性白血病患者细胞的增长，在体内则抑制了患者源异种移植物的生长。