12 results about "Rhabdomyosarcoma" patented technology

An epigenetic silencer factor (ESF) for use in the treatment of cancer, or a polynucleotide encoding the same, wherein the ESF comprises a transcription factor DNA-binding domain functionally linked to at least one epigenetic effector domain, the transcription factor is an oncogenic transcription factor or a cancer-related transcription factor, and the cancer is selected from the group consisting of glioma, glioblastoma, medulloblastoma, astrocytoma, neuroblastoma, epithelioma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g., human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g., colon adenocarcinoma), colorectal cancer (CRC), or metastases of any of the foregoing.

The present invention belongs to the technical field of medicinal chemistry, specifically relates to a compound capable of binding to a CRBN protein and a composition thereof, and also relates to a protein degradation agent based on a CRBN protein and the use thereof. Specifically disclosed is a compound capable of binding to a CRBN protein, wherein the compound is composed of two sub-structure units A and B, the two sub-structures A and B are connected by means of a chemical single bond between Y of the sub-structure A and one of C2 (carbon at position 2), C4 (carbon at position 4), C5 (carbon at position 5), C6 (carbon at position 6) and C7 (carbon at position 7) of the sub-structure B, and the compound is as shown in formula I. The compound can be used as a structural unit of an E3 ubiquitin ligase ligand in PROTAC technology, thereby providing more therapeutic means for cancers or diseases such as prostate cancer, breast cancer, non-small cell lung cancer, Alzheimer's disease, neuroblastoma, hepatocellular carcinoma, colorectal cancer, pancreatic cancer, malignant rhabdomyosarcoma, and oral squamous cell carcinoma.

Chimeric antigen receptor (CAR) with a binding domain that binds STEAP1 are disclosed. The CAR disclosed herein can be used in the treatment of prostate cancer, the Ewing family of tumors (EFT), bladder cancer, ovarian cancer, and rhabdomyosarcoma. The CAR disclosed herein can bind and elicit cytotoxic effects even in low antigen density conditions.

Provided herein are antibodies, portions thereof, and drug conjugates directed against cadherin 15. Also provided are nucleic acid sequences encoding the same; viral particles comprising the same, for example, for re-targeting viral particles to muscle cells; compositions comprising the same and methods of using the same, for example, for treating a subject in need thereof, in some embodiments, the composition may be used in a subject, such as a subject having a skeletal muscle related condition (e.g., X-linked myotubular myopathy (XLMTM), Duchenne muscular dystrophy (DMD), ankylosing muscular dystrophy (DMI), facial shoulder brachial muscular dystrophy type 1 (FSHD), congenital muscular dystrophy type 1 (MDC1A), limb girdle muscular dystrophy, anti-amyotrophy related glycoprotein disease, etc.) or rhabdomyosarcoma.

Provided in the present invention are a class of FTO inhibitors, a preparation method therefor and the use thereof. Specifically, disclosed in the present invention are a 2-(substituted phenyl hetero) aromatic formate as shown in general formula (I) and a derivative compound thereof, and a pharmaceutically acceptable salt, hydrate or solvate thereof. The compound can be used as an FTO target inhibitor for treating diseases associated with the FTO target, such as leukemia, lymphoma, myelodysplastic syndrome, obesity, metabolic syndrome (MS), type 2 diabetes (T2D), Alzheimer's disease, breast cancer, kidney cancer, colorectal cancer, pancreatic cancer, liver cancer, small cell lung cancer, human bone marrow rhabdomyosarcoma, pancreatic cancer and malignant glioblastoma.

The present invention provides a compound of Formula I, a salt thereof, or a solvate thereof, as described herein, the composition is used for treating MYC positive cancers including lung cancer, leukemia, breast cancer, myeloproliferative diseases, colorectal cancer, medulloblastoma, kidney cancer, hepatocellular cancer, melanoma, ovarian cancer, prostate cancer, esophageal adenocarcinoma, liposarcoma, esophageal squamous carcinoma, gastrointestinal stromal tumor, glioma, mucus fibrosarcoma, smooth muscle sarcoma, neuroblastoma, synovial sarcoma, mesothelioma, and the like. Gastric cancer, thyroid cancer, lymphoma, osteosarcoma, rhabdomyosarcoma, fibrosarcoma, epithelial cancer and neurological cancer.

The application discloses a small molecule degrading agent of aryl formamides and application thereof, and has a structure shown in general formula I or a pharmaceutically acceptable salt, an isotopically labeled compound thereof. The compound of the application has a targeted degradation activity on MYC protein, and has a significant proliferation inhibiting activity on various solid tumor cells such as neuroblastoma and rhabdomyosarcoma. The compound of the application can be used for preparing a degrading agent targeting MYC protein, and can also be used for preparing a drug for treating diseases related to MYC gene amplification, fusion, mutation, expression imbalance, conformational change and function abnormality.

The present disclosure provides a compound of formula (I) or a pharmaceutically acceptable salt thereof, a preparation method thereof, and use thereof in the treatment and / or diagnosis of cancers such as neuroblastoma, prostate cancer, pancreatic cancer, leukemia, osteosarcoma, hepatoblastoma, lung cancer, colon cancer, breast cancer, skin cancer, thyroid cancer and / or rhabdomyoid cancer.

The present invention relates to IL-8 inhibitor compounds, preferably dual CXCR1 / CXCR2 receptor inhibitors, useful in the treatment and / or prevention of some sarcomas, preferably in the treatment and / or prevention of osteosarcoma, Ewing sarcoma, rhabdomyosarcoma or lung metastasis associated thereof.

An epigenetic silencing factor (ESF) or a polynucleotide encoding the same for use in the treatment of cancer wherein the ESF comprises a transcription factor DNA binding domain operably linked to at least one epigenetic effector domain wherein the transcription factor is an oncogenic transcription factor or a cancer-related transcription factor, wherein the cancer is selected from the group consisting of glioma, glioblastoma, myeloblastoma, astroblastoma, neuroblastoma, ventricular tubuloma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g., human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g., human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g., human pancreatic ductal adenocarcinoma). E.g., colonic adenocarcinoma), colorectal cancer (CRC), or metastases of any of the foregoing.

Provided herein are 7-((3,5-dimethoxyphenyl)amino)quinoxaline derivatives of the general Formula I; and stereoisomers and pharmaceutically acceptable salts or solvates thereof, in which X2 is N and Ring A, Ring B, Ring C, X1, X3, R1, L, and W have the meanings given in the specification, which are inhibitors of FGFR1, FGFR2, FGFR3 and / or FGFR4 and are useful in the treatment and prevention of diseases which can be treated with an FGFR inhibitor, such as e.g. cancer, e g. bladder cancer, brain cancer, breast cancer, cholangiocarcinoma, head and neck cancer, lung cancer, multiple myeloma, rhabdomyosarcoma, urethral cancer and uterine cancer. The present description discloses the preparation of exemplary compounds as well as pharmacological data thereof (e.g. pages 276 to 308; examples 1 to 30; examples A to E; tables CA to EE). An exemplary compound is e.g. 1-(3-(4-(4-(7-((3,5-dimethoxy phenyl) amino)quinoxalin-2-yl)-1H-pyrazol-1-yl)piperidine-1-carbonyl) azetidin-1-yl)prop-2-en-1-one (e.g. example 1).

An epigenetic silencer factor (ESP), or polynucleotide encoding therefor, for use in the treatment of cancer, wherein the ESF comprises a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor, wherein the cancer is selected from the group consisting of: glioma, gliobastoma, medulloblastoma, astrocytoma, neuroblastomas, ependymoma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g. human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g. colon adenocarcinoma), colon-rectal cancer (CRC), or a metastasis of any of the foregoing.