Resumen de: WO2025101837A1
Described herein are biological devices and extracts useful for detection of different types of cancer. The biological devices include microbial cells transformed with a DNA construct containing genes for producing peroxidase, terminal deoxynucleotidyl transferase (TdT), CA 125, prostatic acid phosphatase, and CA 15-3. Methods for using the devices to detect various cancers, including leukemia, breast cancer, ovarian cancer, and prostate cancer, are also provided herein. In one aspect, the detection methods can make use of commercial ATP detection kits, electromagnetic measurements, or yellow photonic fluorescence measurements.
Resumen de: WO2025098432A1
Disclosed is an mIDH1/NAMPT dual-target inhibitor, which is selected from a compound having a structure as shown in formula (I) or formula (II), or a pharmaceutically acceptable salt, racemate, stereoisomer, prodrug, or solvent compound thereof. The dual-target inhibitor of the present invention can effectively overcome the shortcomings of a single mIDH1 inhibitor and single NAMPT inhibitor having poor efficacy against solid tumors. Said inhibitor can inhibit the malignant proliferation of tumors, has a good therapeutic effect, has low toxicity, and can penetrate the blood-brain barrier, and it is not easy to acquire drug resistance to said inhibitor. Moreover, the inhibitor can be used to prepare a medicament for treating cancer or tumor-related diseases, the cancer or tumor-related diseases comprising multiple myeloma, leukemia, breast cancer, prostate cancer, lung cancer, liver cancer, gastric cancer, bone cancer, brain cancer, head and neck cancer, intestinal cancer, pancreatic cancer, bladder cancer, testicular cancer, ovarian cancer, and endometrial cancer.
Resumen de: WO2025097336A1
The present invention provides an anti-CD30 antigen VHH nanobody. The antibody has the advantages of small relative molecular mass, high affinity, high specificity and stability, good solubility, low immunogenicity and the like. The present invention also constructs a CD30 chimeric antigen receptor and CAR-T cells expressing the chimeric antigen receptor. The use of the CAR-T cells of the present invention for cellular immunotherapy can have a certain treatment effect on various lymphomas.
Resumen de: US2025154599A1
The invention relates to new nucleophosmin protein (NPM) mutants, corresponding gene sequences and relative uses thereof for diagnosis, monitoring of minimal residual disease, prognostic evaluation and therapy of acute myeloid leukaemia (AML).
Resumen de: US2025154268A1
Disclosed in the present invention is an antibody targeting CD25 or a variant thereof. A monoclonal antibody targeting CD25 is a naturally occurring antibody, has an activity of binding to human CD25 and cynomolgus monkey CD25, and has a stronger ADCC effect after being modified with Fc. In-vitro experiments show that the antibody has the effect of clearing Treg and lymphoma cells without blocking an IL-2 signaling pathway.
Resumen de: WO2025099451A1
The present invention relates to compounds of Formula (I), and sub-formulae thereof, that may act as inhibitors of YTHDC1. The invention also relates to pharmaceutical compositions comprising those compounds, and to their use in the treatment of disease and conditions susceptible to YTHDC1 inhibition, such as cancer and in particular acute myeloid leukaemia.
Resumen de: US2025154144A1
Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
Resumen de: US2025154243A1
Provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody. Also provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody lenzilumab and a therapeutically effective amount of a second therapeutic agent. The subject may have a RAS pathway mutation or a RAS pathway mutation and at least one TET2 mutation identified in the tumor cells, an increased percentage of CD116 and CD131 in CD34+ stem and progenitor cells in the subject compared to a healthy subject and/or an increased percentage of CD14+ cells in the subject compared to a healthy subject. A therapeutically effective amount of a hypomethylating agent or hydroxyurea may b
Resumen de: US2025152707A1
The present invention provides combinations and methods using same for the treatment of malignancy, particularly a myeloid malignancy such as acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN), chronic myeloid leukemia (CML), and chronic myelomonocytic leukemia (CMML). The combination may comprise an antibody or antigen-binding fragment thereof that binds to CD70, and an inhibitor of BCL-2. In certain embodiments, the antibody is ARGX-110 (cusatuzumab). In certain embodiments, the BCL-2 inhibitor is venetoclax. In certain embodiments, the combination provides synergistic treatment of AML. The combination may additionally comprise at least one additional anti-cancer agent.
Resumen de: WO2025099437A1
A method of predicting the risk of progression of a cancer of a subject is described. The method can be used to infer the historical growth dynamics of an individual's cancer using fluctuating methylation clocks (FMCs). In the case of, for example, chronic lymphocytic leukaemia (CLL) (at least), the inferred growth rate holds valuable prognostic information, enabling patient stratification beyond that of standard clinical practice.
Resumen de: US2025154263A1
Methods are disclosed for treating chronic lymphocytic leukemia (CLL), and other diseases, comprising administrating to a patient an anti-CLECL1 antibody or antigen-binding fragment thereof, as soluble molecules or as components of CAR-T cells or as bi- or tetra-specific antibodies that co-ligate T cells or NK cells and abnormal cells in CLL or other diseases or as bi- or tetra-specific antibodies that bind only the B cells in CLL or only abnormal cells in other diseases. Methods are disclosed for determining residual disease after therapy or predicting disease relapse in a patient comprising analyzing a blood or bone sample from a patient treated for the disease for the presence of CLECL1+ PF cells using a monoclonal antibody to CLECL1 or antigen-binding fragment thereof, wherein reappearance of CLECL1+ PF cells in the blood of the patient is indicative of development of resistance to therapy. Monoclonal antibodies to CLECL1 are disclosed.
Resumen de: TW202417498A
The present disclosure relates to anti-SCUBE1 antibodies, antigen-binding fragments thereof, and antibody drug conjugates (ADCs), as well as methods of treating and/or preventing SCUBE1-expressing cancers.
Resumen de: WO2024008659A1
Extranodal natural killer/T-cell lymphoma (ENKTCL) is an aggressive haematological malignancy. The treatment of ENKTCL is dependent on the extent of the tumor. However the use of L-asparaginase-containing regimens obtained impressive outcomes as induction or salvage treatment for ENKTCL. Although more than 70% of early-stage patients can achieve long-term survival, patients with advanced-stage disease had extremely poor prognosis even after asparaginase-based chemotherapy regimens. There is thus a medical need for improving the treatment of ENKTCL with L-asparaginase. Now the inventors demonstrate the interest of the use of L-asparaginase in combination with a ferroptosis inducer for the treatment of ENKTCL. In particular, combination of APR-246 and Erwinase® has synergistic effects in KHYG-1 cells. The present invention thus relates to the use of a L-asparaginase in combination with a ferroptosis inducer for the treatment of ENKTCL.
Resumen de: WO2025097084A1
Familial Platelet Disorder (FPD) is associated with germline RUNX1 mutations, establishing a preleukemic state and increasing the risk of developing acute leukemia. Conventional approaches lack intervention strategies that may delay or prevent leukemia progression in patients. The present disclosure integrates single-cell transcriptomics, flow cytometry, and spatial proteomics data analyses for a large cohort of FPD patient samples to show that RUNX1 mutations unleash inflammatory signaling in familial platelet disorder. Specifically, the receptor CD74 has been identified as a biomarker and a master regulator of downstream signaling pathways. Embodiments of the present disclosure provide diagnostic and treatment methods for FPD in patients.
Resumen de: WO2025096692A2
Provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody. Also provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody lenzilumab and a therapeutically effective amount of a second therapeutic agent. The subject may have a RAS pathway mutation or a RAS pathway mutation and at least one TET2 mutation identified in the tumor cells, an increased percentage of CD116 and CD131 in CD34+ stem and progenitor cells in the subject compared to a healthy subject and/or an increased percentage of CD14+ cells in the subject compared to a healthy subject. A therapeutically effective amount of a hypomethylating agent or hydroxyurea may b
Resumen de: WO2025097150A1
Provided herein are methods of preparing an immunotherapeutic with enhanced efficacy by treating lymphocytes with a B-cell lymphoma 2 (BCL-2) inhibitor, immunotherapeutic compositions produced by the methods herein, and methods of treating cancer therewith.
Resumen de: WO2025096532A1
Provided herein are methods for monitoring, identifying, assessing, circulating tumor DNA (ctDNA) for treating a subject or monitoring a subject having cancer, and related methods and uses thereof. In some embodiments, the disease or condition is a B cell lymphoma (BCL) relapsed or refractory.
Resumen de: WO2025097015A1
Targeted therapeutics for the treatment of cancers that express CLEC2A are described. The targeted therapeutics include anti-CLEC2A binding domains and engineered formats thereof such as multi-domain binding molecules, antibody conjugates, and recombinant receptors expressed by an immune cell. The targeted therapeutics can be used to treat acute myeloid leukemia (AML), such as KMT2A-r AML cell. Furthermore, the binding domains can be used to isolate cells expressing CLEC2A or to target such cells ex vivo or in vivo for research, diagnostic, or therapeutic purposes.
Resumen de: WO2025096605A1
The present disclosure relates to methods of treating a subject having multiple myeloma, including refractory/resistant multiple myeloma, comprising administering to the subject a CD73 inhibitor and an anti-CD38 agent.
Resumen de: WO2025096203A1
Chimeric RNAs and their protein products, including BCR-ABL in chronic myelogenous leukemia and EML4-ALK4 in lung cancer, have been well-established as ideal biomarkers and drug targets for malignant cancers. Using RNA-Sequencing data from The Cancer Genome Atlas (TCGA) database, a chimeric RNA SLC2A11-MIF was identified that is present in nearly 50% of CRC samples but absent in non-cancer colon tissue. Disclosed herein is a method for diagnosing and treating cancers expressing this chimeric RNA and protein.
Resumen de: US2025145706A1
The present invention provides antibodies for the treatment of chronic lymphocytic leukemia (CLL). These antibodies target the B-cell receptor (BCR) of CLL cells characterised by R110-mutated immunoglobulin lambda variable 3-21 (IGLV3-21R110). The invention also provides nucleic acid sequences encoding the forgoing antibodies, vectors containing the same, pharmaceutical compositions and kits with instructions for use.
Resumen de: US2025145635A1
A compound containing a bis(azanylylidene) sulfonyl structure of formula (I) as a PKR agonist and/or USP9X inhibitor, which can be used for treating related diseases mediated and regulated by PKR and USP9X, wherein the related diseases comprise, but are not limited to: sickle-cell anemia, β-thalassemia, hereditary non-spherocytic hemolytic anemia, hemolytic anemia, hereditary spherocytosis, hereditary elliptocytosis, abetalipoproteinemia, paroxysmal nocturnal hemoglobinuria, acquired hemolytic anemia, congenital anemia, anemia of chronic diseases, colorectal cancer, kidney cancer, pancreatic cancer, breast cancer, lung cancer, esophageal cancer, melanoma, lymphoma, glioblastoma, or multiple myeloma.
Resumen de: US2025144189A1
The present disclosure provides a novel recombinant unidirectional asparagine racemase for the conversion of L-asparagine to D-asparagine. Such unidirectional asparagine racemase could be used in treating cancers (e.g., leukemias or lymphomas) that require exogenous L-asparagine. This innovative design offers a promising new approach to reduce detrimental side effects caused by current therapies.
Resumen de: US2025144100A1
The present invention relates to a tablet composition comprising Ibrutinib and one ore more pharmaceutically acceptable excipients. The invention further relates to the use of said composition as a medicament, particularly in the treatment of lymphocytic leukaemia (CLL), mantle cell lymphoma (MCL), Waldenström's macroglobulinaemia (WM) and chronic graft versus host disease (cGVHD).
Nº publicación: US2025144023A1 08/05/2025
Solicitante:
ONCOPEPTIDES INNOVATION AB [SE]
Oncopeptides Innovation AB
Resumen de: US2025144023A1
The present invention providesmelflufen (melphalan flufenamide; L-Melphalanyl-4-fluoro-L-phenylalanine ethyl ester), or a salt thereof, for use in the treatment or prophylaxis of multiple myeloma, wherein a dosage of melflufen (excluding the mass of any salt) is administered as a parenteral dosage at an infusion rate of 1.0 to 1.8 mg/min. Also provided is melflufen, or a salt thereof, for use in the treatment or prophylaxis of a cancer, for example a solid cancer, wherein a dosage of melflufen is administered as a parenteral dosage at an infusion rate less than 0.8 mg/min (for example 0.3 to 1.0 mg/minor for example 0.3 to 0.8 mg/min).