Resumen de: WO2025199151A1
The present disclosure relates to compounds and compositions, and methods of uing the compounds and compositions for inducing the degradation of proteins that are relevant to cancer such as. for example. KDM4B and VCL. Also described are methods of treating cancer (e.g, a sarcoma, a carcinoma, a hematological cancer, a solid tumor, breast cancer, cervical cancer, gastrointestinal cancer, colorectal cancer, brain cancer, skin cancer, prostate cancer, ovarian cancer, non-small cell lung carcinoma, thyroid cancer, testicular cancer, pancreatic cancer, liver cancer, endometrial cancer, melanoma, glioma, leukemia, lymphoma, chronic myeloproliferative disorder, myelodysplastic syndrome, myeloproliferative neoplasm, plasma cell neoplasm (myeloma)) using the disclosed compounds and compositions. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.
Resumen de: AU2024232412A1
An aqueous pharmaceutical formulation having improved stability includes denosumab and a poloxamer, and preferably a histidine buffer and/or sugar or sugar alcohol. The formulation is for use in treating or preventing osteoporosis, loss of bone mass, skeletal-related events associated with multiple myeloma, solid tumor bone metastases, giant cell tumors of the bone or hypercalcemia.
Resumen de: AU2025230652A1
22046882_1 (GHMatters) P122730.AU.1 The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β- thalassemia (e.g., β-thalassemia major and β-thalassemia intermedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, parkinson’s Disease, Alzheimer’s Disease or Friedreich’s Ataxia. The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, ß- thalassemia (e.g., ß-thalassemia major and ß-thalassemia intermedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, parkinson's Disease, Alzheimer's Disease or Friedre
Resumen de: US2025295771A1
Provided herein are uses of T cells, e.g., chimeric antigen receptor (CAR) T cells, for treating a tumor or a cancer (such as B cell related cancer, e.g., multiple myeloma) wherein the subject being treated has previously received a topoisomerase inhibitor, a proteasome inhibitor, an anti-CD38 agent, an immunomodulatory agent, or an anti-SLAMF agent therapy.
Resumen de: US2025299796A1
Provided herein are methods and immune biomarkers that identify progression and treatment options for hematological malignancies (e.g., smoldering multiple myeloma (SMM), monoclonal gammopathy of undetermined significance (MGUS), or multiple myeloma (MM)). Also provided are materials and methods for the prognosis, staging, and monitoring of SMM, MGUS, or MM based on the presence of the immune biomarkers in a sample (e.g., a blood sample or a bone marrow sample), as well as methods for monitoring the progression of SMM, MGUS, or MM, determining the efficacy of a therapeutic agent, determining a treatment for SMM, MGUS (e.g., before progression to MM), or MM, and/or treating SMM, MGUS, or MM. The methods provided herein provide several advantages over invasive biopsies.
Resumen de: WO2025194094A1
This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of carbothioamide (and structurally related) small-molecule compounds which function as inhibitors of SIRT5, and their use as therapeutics for the treatment of diseases associated with posttranslational modification functions (e.g., diseases associated with SIRT5 activity) (e.g., cancer (e.g., melanoma, non-small cell lung cancer (NSCLC), hepatocellular carcinoma (HCC), ovarian cancer, colorectal cancer (CRC), acute myeloid leukemia (AML), Ewing's sarcoma, brain cancer, pancreatic cancer, renal cancer, breast cancer, prostate cancer, lung cancer, leukemia and lymphoma), diabetes, autoimmune diseases, inflammatory diseases, fibrotic diseases, cardiovascular diseases, and neurodegenerative diseases).
Resumen de: US2025290148A1
Disclosed herein is a method for preventing or reversing CAR-T cell resistance and/or radioresistance in a relapsed and refractory diffuse large B-cell lymphoma (R/R DLBCL) of a subject, that involves assaying a sample from the subject for mRNA sequences of genes with roles in DNA damage, apoptosis, immune activation, and/or c-MYC signaling; detecting aberrant splicing in one or more of the mRNA sequences; and administering to the subject an antisense oligonucleotide (ASO) that prevents the aberrant splicing.
Resumen de: US2025289837A1
The present invention relates to compounds of formula (I), processes for their preparation, and pharmaceutical compositions containing them as the active ingredient. Compounds of the present invention may be useful as mitochondrial KV1.3 inhibitors (mitoKV1.3) to treat cancer diseases and the like, including breast, colon, and prostate tumors, melanoma, smooth muscle, and skeletal muscle cancer, chronic lymphocytic leukemia, glioblastoma, and pancreatic ductal adenocarcinoma.
Resumen de: AU2024253832A1
Aspects of the disclosure provide anti-TMPRSS6 antibodies and methods of using the same for promoting hepcidin expression, and treating iron overload associated conditions, such as hemochromatosis, sickle cell disease, thalassemia, hemolysis, Diamond-Blackfan anemia, myelodysplastic syndrome (MDS), blood transfusion.
Resumen de: US2025289806A1
Disclosed herein are novel compounds with STK17A inhibitory activity. The compounds may be used to treat proliferative disorders, including myelodysplastic syndrome and leukemia.
Resumen de: WO2025189988A1
Provided is use of a monophosphate deoxyribose fluorouracil nucleoside prodrug in the preparation of a drug for preventing and/or treating tumors. The monophosphate deoxyribose fluorouracil nucleoside prodrug is a derivative formed by substituting at least one thymine in nucleolin aptamer AS1411 with fluorouracil. The tumors include at least one of lung cancer, breast cancer, prostate cancer, pancreatic cancer, kidney cancer, cervical cancer, leukemia and lymphoma, melanoma, glioblastoma, neuroblastoma, sarcoma, and gastric cancer. The monophosphate deoxyribose fluorouracil nucleoside prodrug binds to the nucleolin protein and selectively enters tumor cells under the action of the nucleolin protein, and release an antimetabolite to inhibit thymine nucleotide synthase, thereby achieving an anti-tumor effect.
Resumen de: US2025288669A1
A novel human T-cell acute lymphoblastic leukemia (T-ALL) cell line called INB16 (ATCC Deposit no. PTA-125809) induces memory like function on natural killer cells upon contact therewith, which memory like natural killer cells have demonstrated ability to identify and kill cancer cells, including hematologic and solid tumor cells. Useful applications of the INB16 cell line include research, a cancer therapeutic agent comprising replication incompetent INB16 cells and/or membrane portions thereof for in vivo administration and restoring function of a patient's own NK cells, and related methods of treating cancer.
Resumen de: WO2025191110A1
The invention relates to an in vitro method for establishing the prognosis of a subject diagnosed as suffering or having suffered from a diffuse large B-cell lymphoma and including: - an identification step of at least two markers comprising 2-aminobutyrate or its acid derivative thereof and LDL-1 lipoprotein optionally in combination with at least one marker selected from : 2-hydroxybutyrate or its acid derivative thereof, 3-hydroxybutyrate or its acid derivative thereof, LDL-2 lipoprotein, LDL-1-CH lipoprotein, LDL-1 lipoprotein phospholipids, the Apo-B subfraction of LDL-1 lipoprotein, LDL-1 lipoprotein triglycerides, LDL-2 lipoprotein triglycerides, LDL-3 lipoprotein triglycerides, formic acid and acetyl acetic acid, identifying said at least two markers being the prognosis of, or a risk of, a bad clinical course of the diffuse large B-cell lymphoma in the subject.
Resumen de: WO2025193685A1
Provided herein are methods of treating a subject who has multiple myeloma and has received one to three prior treatment(s). Infusions of chimeric antigen receptor (CAR)-T cells comprising a CAR capable of specifically binding to an epitope of BCMA are administered to the subject.
Resumen de: US2025290890A1
The present disclosure teaches a diagnostic home kit to detect midkine level in blood samples. The kit includes an analyte receiver to receive the blood sample. Further, the kit includes a cantilever biosensor coated with a piezoelectric material and may also be immobilized by Anaplastic Lymphoma Kinase (ALK) receptors configured to attach midkine from the blood sample. The cantilevers may deflect when midkine binds to the ALK receptors, and this deflection may be captured by the piezoelectric material transducing a signal corresponding to the attached midkine. Furthermore, the kit includes an amplifier to receive and amplify the transduced signal. Moreover, the kit includes a signal processor to process the amplified signals to determine the level of midkine in the received blood sample. Additionally, the kit includes an output display unit to display midkine level in the blood sample.
Resumen de: WO2024100250A1
In a first aspect, the invention relates to a combination of at least one menin inhibitor with at least one immunoproteasome inhibitor for use as medicament, preferably for use in the treatment of leukemia. A second aspect of the invention is related to a pharmaceutical preparation comprising at least one menin inhibitor and at least one immunoproteasome inhibitor, optionally one or more pharmaceutically acceptable carrier(s) and optionally one or more pharmaceutically acceptable adjuvant(s).
Resumen de: MX2025005421A
A pharmaceutical combination includes a beta-lactam antibiotic alone or in combination with a beta-lactamase inhibitor useful for treating multiple myeloma, monoclonal gammopathy of undetermined significance, and smoldering multiple myeloma. A method for treating multiple myeloma, monoclonal gammopathy of undetermined significance, or smoldering multiple myeloma includes administering to a patient in need thereof an effective amount of a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor. Beta-lactam antibiotic for use in combination with a beta-lactamase inhibitor for the treatment of multiple myeloma, monoclonal gammopathy of undetermined significance, or smoldering multiple myeloma in a patient in need of the treatment is also described.
Resumen de: EP4617665A1
The invention relates to an in vitro method for establishing the prognosis of a subject diagnosed as suffering or having suffered from a diffuse large B-cell lymphoma and including:- an identification step of at least two markers selected from : 2-aminobutyrate or its acid derivative thereof, 2-hydroxybutyrate or its acid derivative thereof, 3-hydroxybutyrate or its acid derivative thereof, LDL-1 lipoprotein, LDL-2 lipoprotein, LDL-1-CH lipoprotein, LDL-1 lipoprotein phospholipids, the Apo-B subfraction of LDL-1 lipoprotein, LDL-1 lipoprotein triglycerides, LDL-2 lipoprotein triglycerides, LDL-3 lipoprotein triglycerides, formic acid and acetyl acetic acid, identifying said at least two markers being the prognosis of, or a risk of, a bad clinical course of the diffuse large B-cell lymphoma in the subject.
Resumen de: MX2025005311A
The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer, in particular of hematological neoplasms, such as acute myeloid leukemia (AML). The present invention furthermore relates to tumor-associated T-cell peptide epitopes that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.
Resumen de: WO2025185680A1
Disclosed in the present application are compounds having a benzamide structure as shown in formula (I), a preparation method therefor, and the use thereof in preparing pharmaceutical formulations for preventing and/or treating diseases caused by CRBN abnormality. The compounds with the structure shown as formula (I) are small molecule compounds having activity of covalently binding to CRBN so as to inhibit same. The diseases caused by CRBN abnormality are tumors or autoimmune diseases, the tumors including mantle cell lymphoma, multiple myeloma, non-Hodgkin's lymphoma or solid tumors, and the autoimmune diseases including rheumatoid arthritis, systemic lupus erythematosus, ankylosing spondylitis or psoriasis.
Resumen de: WO2025188713A1
Provided are highly conserved antigens and epitopes of Feline Leukemia Virus that can be used in vaccines and to produce binding gp70 or pl5E proteins (e.g., antibodies) for treating, preventing, or reducing the risks of infections caused by Feline Leukemia Virus, and as targets for detecting Feline Leukemia Virus infection.
Resumen de: US2025281449A1
The invention relates compound of formula (I), enantiomers, mixture of enantiomers, diastereoisomers and mixture of diastereoisomers thereof:wherein W, X, Y and Z are as defined, for use in the treatment of Multiple Myeloma (MM). A pharmaceutical composition including a pharmaceutical acceptable vehicle and at least a compound of formula (I) is also included.
Resumen de: US2025281654A1
The invention provides methods for treating a cancer, such as acute myeloid leukemia, in a mammalian subject that include administering to the subject (i) an epigenetic drug such as one or both an HDAC inhibitor and an LSD1/KDM1A inhibitor, and (ii) a radioisotope-labeled agent that targets cancer cells in the subject, wherein the amounts of the epigenetic drug(s) and radiolabeled agent, when administered in conjunction with one another, are therapeutically effective.
Resumen de: US2025281502A1
A method is disclosed for treating an individual afflicted by a multiple myeloma by a composition comprising at least one G-quadruplex (G4) stabilizer. Also disclosed is a composition comprising at least one G-quadruplex (G4) stabilizer for its use in a method for treating an individual afflicted by a multiple myeloma.
Nº publicación: US2025282818A1 11/09/2025
Solicitante:
UNIV OF FLORIDA RESEARCH FOUNDATION INCORPORATED [US]
UNIVERSITY OF FLORIDA RESEARCH FOUNDATION,INCORPORATED
Resumen de: US2025282818A1
One aspect of the invention is any compound, or salt thereof, described herein. Another aspect is a method of treating a disease, disorder, or symptom thereof, in a subject, comprising administration to the subject of a compound, or salt thereof, herein. Another aspect is a method of inhibiting a proteasome in a subject, comprising administration to the subject of a compound, or salt thereof, herein. Another aspect is a method of making a compound, or salt thereof, described herein using one or more reagents, chemical transformations, or chemical intermediate compounds as described herein.