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6 results about "Rhabdomyosarcoma" patented technology

A malignancy arising from tissue that forms the striated muscle.

Anti-human M-cadherin (cdh15) antibodies, conjugates and uses thereof for delivery of genetic payloads to muscle cells

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.
Owner:REGENERON PHARMACEUTICALS INC

FTO inhibitors, preparation method therefor and use thereof

PCT designated stageWO2025167758A1Nervous disorderOrganic chemistryDiseaseFormate
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.
Owner:SHANGHAI INSTITUTE OF MATERIA MEDICA CHINESE ACADEMY OF SCIENCES

A small molecule degrader of arylformamidines and uses thereof

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.
Owner:ZHEJIANG UNIV

Transgene cassettes and epigenetic silencers for the treatment of disorders

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.
Owner:OSPEDALE SAN RAFFAELE S R I +1