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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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.
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: 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: AU2025220739A1
The disclosure provides cells comprising CD19-directed chimeric antigen receptor (CAR) genetically modified autologous T cell immunotherapy for the treatment of, e.g., relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. Some aspects of the disclosure relate to methods of treatment and monitoring following infusion of T cell therapy provided herein. The disclosure provides cells comprising CD19-directed chimeric antigen receptor (CAR) genetically modified autologous T cell immunotherapy for the treatment of, e.g., relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. Some aspects of the disclosure relate to methods of treatment and monitoring following infusion of T cell therapy provided herein. ug h e d i s c l o s u r e p r o v i d e s c e l l s c o m p r i s i n g - d i r e c t e d c h i m e r i c a n t i g e n r e c e p t o r ( ) u g g e n e t i c a l l y m o d i f i e d a u t o l o g o u s c e l l i m m u n o t h e r a p y f o r t h e t r e a t m e n t o f , e g , r e l a p s e d o r r e f r a c t o r y l a r g e - c e l l l y m p
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.
Resumen de: US2025283177A1
The present disclosure is directed to methods of genetically subtypin peripheral t-cell lymphoma.
Resumen de: US2025282883A1
This invention provides antibodies that recognize the B Cell Maturation Antigen (BCMA) and that bind naïve B cells, plasma cells, and/or memory B cells. The invention further provides methods for depleting naïve B cells, plasma cells, and memory B cells, and for treating B cell-related disorders, including lymphomas and autoimmune diseases.
Resumen de: AU2024233069A1
The present disclosure describes antigen binding molecules, including antibodies, that specifically bind to the anti-CD20 scFv-14 or Gibbon ape leukemia virus gp70 protein, as well as molecules comprising the described sequences and cells presenting such molecules. The antigen binding molecules may be used in research, diagnostic, clinical, and other applications.
Resumen de: AU2024225818A1
The present invention relates to a compound of formula (I) or a pharmaceutically acceptable salt or solvate thereof, for use in the treatment of myeloid disorders, such as acute myeloid leukemia (AML), in subjects having at least one chromosome abnormality of 3q21 and/or 3q26.
Resumen de: US2025285767A1
Techniques for estimating a risk that a condition of a patient with a multiple myeloma (MM) precursor disease such as MGUS or SMM will progress into MM.
Resumen de: IE20230360A1
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: WO2024097758A1
Provided herein are methods of treating acute leukemia, such as acute myeloid leukemia (AML), in an individual, such as an individual having a mutation within the nucleophosmin 1 (NPM1) gene or a rearrangement within the lysineK-methyltransferase 2A (KMT2A) gene, wherein the methods comprise administering a menin inhibitor, ziftomenib, to the individual at a dose of 600 milligrams daily.
Resumen de: AU2023373683A1
Methods of treating non-Hodgkin lymphoma by administering a multispecific antibody to a patient in need are provided. Methods of making such antibodies, and compositions, including pharmaceutical compositions, comprising such antibodies, are also provided.
Resumen de: MX2025005091A
A method for assessing responsiveness of a subject to a treatment comprising T cells expressing a bivalent BCMA-targeting chimeric antigen receptor (CAR), comprising administering to the subject the T cells, and assessing the responsiveness of the subject to the treatment based on time length the subject maintains minimal residual disease (MRD) negative status.
Resumen de: US2025277271A1
Provided are improved methods for prognosing a clinical outcome in a subject or diagnosing the subject with high-risk or stable smoldering multiple myeloma (SMM), comprising determining a 3D telomeres organization signature of a test sample from the subject, the test sample comprising plasma cells, applying a classification model to the 3D telomeres organization signature to obtain an output classification that is indicative of the clinical outcome or diagnosis of the subject. The classification model is trained to distinguish between stable SMM and high-risk SMM and consists of the telomere parameters: a) nuclear telomere distribution, a/c ratio, and total telomere length; b) a/c ratio and telomere numbers; c) a/c ratio and nuclear telomere distribution, or d) a/c ratio and telomere aggregates. Also provided are methods for treating a subject with high-risk or stable SMM.
Resumen de: US2025276961A1
Provided herein are compounds, preferably compounds inhibiting tyrosine kinase enzymatic activity of a protein selected from Abelson protein (ABL1), Abelson-related protein (ABL2), or a chimeric protein BCR-ABL1, compositions thereof, and methods of their preparation, and methods of inhibiting tyrosine kinase enzymatic activity of a protein selected from Abelson protein (ABL1), Abelson-related protein (ABL2), or a chimeric protein BCR-ABL1, and methods for treating diseases wherein modulation of BCR-ABL1 activity prevents, inhibits, or ameliorates the pathology and/or symptomology of the disease.
Resumen de: AU2025217309A1
Provided herein are methods of treating a solid malignant tumor using antigen-specific T cells and methods of managing tumor flare in treatment of a solid malignant tumor using antigen-specific T cells. The methods provided herein improve the safety of treatment by informing the patient about the potential risks for tumor flare, telling the patient to contact his or her physician if tumor swelling occurs, counseling a patient with Waldeyer’s ring lymphadenopathy to contact his or her physician if shortness of breath or stridor occurs, or grading and managing tumor flare developed in the patient. Provided herein are methods of treating a solid malignant tumor using antigen-specific T cells and methods of managing tumor flare in treatment of a solid malignant tumor using antigen-specific T cells. The methods provided herein improve the safety of treatment by informing the patient about the potential risks for tumor flare, telling the patient to contact his or her physician if tumor swelling occurs, counseling a patient with Waldeyer's ring lymphadenopathy to contact his or her physician if shortness of breath or stridor occurs, or grading and managing tumor flare developed in the patient. ug u g r o v i d e d h e r e i n a r e m e t h o d s o f t r e a t i n g a s o l i d m a l i g n a n t t u m o r u s i n g a n t i g e n - s p e c i f i c c e l l s a n d m e t h o d s o f m a n a g i n g t u m o r f l a r e i n t r e a t m e n t o f a s o l i d m a l i g n a n t t u m o r u
Resumen de: US2025276012A1
The present invention provides engineering of T cells to express a Chimeric Antigen Receptor (CAR), and compositions comprising the same. The present invention related to novel CD19-targeting CAR T cell product comprising second and third generation CAR gene for treating B-cell disorders including leukaemia and lymphoma. The present invention is also related to process of preparing the novel CAR T cell products.
Resumen de: US2025277213A1
Among the various aspects of the present disclosure are provisions of compositions and methods related to multiple myeloma-associated long noncoding RNAs. The present disclosure describes a composition that comprises a long non-coding RNA (lncRNA) inhibitor targeting LINC01432. Also disclosed is a method to select a treatment for a multiple myeloma patient, as well as to treat a multiple myeloma patient, both related to targeting LINC01432.
Resumen de: US2025277060A1
The invention relates to antigen-binding molecules that specifically bind to IgM μ-chain antigen. In one embodiment, the antigen-binding molecule is an antibody that specifically binds to canine IgM μ-chain antigen. Chimeric molecules of the antibody conjugated to another heterologous moiety are also provided. In one embodiment, the heterologous moiety is monomethyl auristatin E (MMAE). A method of inhibiting cancer using the antibody or the chimeric molecule thereof is also provided. In another embodiment, the antibody-MMAE conjugate suppresses tumour development in a murine model of B cell lymphoma.
Resumen de: WO2025184298A1
The invention features methods of treating a subject having a cancer (e.g., leukemia (e.g., acute myeloid leukemia), solid tumor, breast cancer, lung cancer, colorectal cancer, or pancreatic cancer) by administering venetoclax or a pharmaceutically acceptable salt thereof in combination with zotatifin or a pharmaceutically acceptable salt thereof.
Resumen de: WO2025181039A1
The invention relates to combination and combination therapies, particularly for the treatment of a myeloid malignancy, such as acute myeloid leukemia (AML) or a myelodysplastic syndrome (MDS). The combination therapies include (i) a recombinant binding protein comprising (a) an ankyrin repeat domain specifically binding CD3 and (b) at least one, at least two, or at least three further ankyrin repeat domains, wherein each of the at least one, at least two, or at least three further ankyrin repeat domains specifically binds one tumor-associated antigen, suitably one selected from the group consisting of CD123, CD33 and CD70, and (ii) a Bcl-2 inhibitor, e.g., venetoclax, or a pharmaceutically acceptable salt thereof and/or (iii) a hypomethylating agent, e.g., azacitidine or decitabine.
Resumen de: WO2025184594A1
This application pertains to the use of Compound (A): or a pharmaceutically acceptable salt thereof, for the treatment of various diseases or disorders, including, for example, advanced non-Hodgkin lymphoma (NHL), relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL), transformed follicular lymphoma, nodal T-follicular helper cell lymphoma (nTFHL), relapsed/refractory (R/R) nodal T-follicular helper cell lymphoma, nodal T-follicular helper cell lymphoma - angioimmunoblastic type (nTFHL- Al) / advanced angioimmunoblastic T-cell lymphoma (AITL), or relapsed/refractory (R/R) nodal T-follicular helper cell lymphoma - angioimmunoblastic type (nTFHL-AI) / angioimmunoblastic T-cell lymphoma (AITL).
Resumen de: CN120153256A
The present invention relates to the diagnosis and assessment of juvenile granulocytic leukemia (JMML). In particular, it relates to a method of diagnosing JMML in a subject, said method comprising: a) determining the amount of at least one biomarker present on or in hematopoietic stem and progenitor cells (HSPC) in a biological sample, said at least one biomarker being selected from the group consisting of i) Group I consisting of CD52, RAMP1, LTB, LST1, JAML, IFITM3, CD7, CD69, CD164, CD74, TNF, TFPI, DLK1, CD82, IGHM, CALCRL, RALA, SLC2A5, HSPA5, HLA-DRA, RABI1A, SELL, VAMPS, FCMR, CLEC7A, NDFIP1, CLEC9A, HCST, b) comparing the quantity determined in step a) with a reference; and c) diagnosing the JMML based on the comparison of step b). In addition, the invention also relates to a method for classifying subjects suffering from JMML as a low-risk or high-risk group of JMML. Furthermore, the invention relates to the use of at least one biomarker present on or in HSPC in a biological sample for diagnosing JMML in a subject suffering from or at risk of developing into JMML as a low-risk or high-risk group of JMML. Furthermore, the present invention relates to a kit for diagnosing JMML in a subject or classifying a subject suffering from JMML as a low-risk group or a high-risk group of JMML. Furthermore, the present invention relates to the use of an inhibitor for the treatment and/or prevention of JMML, said inhibitor specifically inhibiting at least one biomarker selected fr
Resumen de: CN120112796A
The present invention provides methods of administration directed to the treatment of cancer, such as multiple myeloma, with an anti-crystallizable fragment receptor-like 5 (FcRH5)/anti-cluster of differentiation 3 (CD3) bispecific antibody.
Resumen de: EP4610654A2
The present invention relates to a chimeric antigen receptor (CAR) which comprises an antigen-binding domain which selectively binds TCR beta constant region 1 (TRBC1) or TRBC2; cells; such a T cells comprising such a CAR; and the use of such cells for the treatment of a T-cell lymphoma or leukaemia in a subject.
Resumen de: CN119119271A
Isolated human monoclonal antibodies and related antibody-based compositions and molecules that bind to human CD38 are described. Pharmaceutical compositions comprising the human antibodies and therapeutic and diagnostic methods of using the human antibodies are also described.
Resumen de: AU2025205481A1
The present invention relates to administration speed of obinutuzumab. The present invention relates to administration speed of obinutuzumab. ul u l h e p r e s e n t i n v e n t i o n r e l a t e s t o a d m i n i s t r a t i o n s p e e d o f o b i n u t u z u m a b
Resumen de: TW202440945A
Methods of monitoring therapeutic efficacy in a subject with myelodysplastic syndrome (MDS) are provided. Also provided is a method of identifying a subject with MDS for treatment with a telomerase inhibitor, and methods of treating MDS. The methods include administering to the subject a telomerase inhibitor and assessing variant allele frequency (VAF) for one or more of the following genes: SF3B1, TET2, DNMT3A, ASXL1, and CUX1 in a biological sample obtained from the subject after administration of the telomerase inhibitor. In some cases, a 25% or more reduction in VAF identifies a subject who has an increased likelihood of benefiting from treatment with a telomerase inhibitor. In some instances, the telomerase inhibitor is imetelstat or imetelstat sodium.
Resumen de: NZ755004A
Described herein are antibodies that target Leukemia Inhibitory Factor (LIF). Also described herein are uses of these antibodies for the treatment of cancer.
Resumen de: US2025269012A1
An oncolytic group B adenovirus suitable for treating a solid tumor (for example sarcoma, carcinoma and/or lymphoma) comprising a sequence of formula (I): 5′ITR-B1-BA-B2-BX-BB-BY-B3-3′ITR (I) wherein: a first transgene encodes a polypeptide comprising a target-sequence specific for a binding domain on the cells of a cell-based immunotherapy, for example bearing a (exogenous) recombinant surface expressed protein, such as a chimeric antigen receptor or an NKG2D receptor, in particular wherein the target-sequence specifically binds to said surface expressed protein (more especially the chimeric antigen receptor) on the immunotherapy cell; and a second transgene encodes a polypeptide comprising a molecule that promotes trafficking of the cell-based immunotherapy into and within the tumor.
Resumen de: US2025269024A1
Provided herein are methods of treating a subject who has multiple myeloma and has received an initial therapy, including a stem cell transplantation. Infusions of chimeric antigen receptor (CAR)-T cells comprising a BCMA CAR comprising a polypeptide are administered to the subject. In certain embodiments, the dose of CAR-T cells administered to the subject is from 1.0×105 to 5.0×106 of CAR-T cells per kilogram of the subject's mass. The method of treatment is effective in obtaining and maintaining minimal residual disease negativity status, as well as other beneficial clinical outcomes related to efficacy and safety.
Resumen de: US2025269048A1
The present invention relates bispecific antibodies and antigen binding fragments thereof for binding to CD33 and CD7 for use in treating CD33+ CD7+ hematological malignancies, and in particular Acute Myeloid Leukaemia (AML). In particular, the present invention relates to a bispecific antibody or antigen binding fragments thereof binding to CD33 and CD7, wherein the bispecific antibody or antigen binding fragments comprises a first binding region binding to human CD33 which comprises the sequences having at least 95% sequence identity to sequences: VH SEQ ID No. 81; and VL SEQ ID No. 85, and a second binding region binding to human CD7 which comprises the a VH sequence and a VL sequence having at least 95% sequence identity to the following sequences: VH SEQ ID No. 11; VH SEQ ID No. 21; VH SEQ ID No. 31; VH SEQ ID No. 51; VH SEQ ID No. 71; VL SEQ ID No. 15; VL SEQ ID No. 25; VL SEQ ID No. 35; VL SEQ ID No. 55; and VL SEQ ID No. 75 or wherein the bispecific antibody or antigen binding fragments comprises a first binding region binding to human CD33 which comprises the sequences having at least 95% sequence identity to sequences: VH SEQ ID No. 97; and VL SEQ ID No. 101, and a second binding region binding to human CD7 which comprises the a VH sequence and a VL sequence having at least 95% sequence identity to the following sequences: VH SEQ ID No. 1; VH SEQ ID No. 51; VH SEQ ID No. 71; VL SEQ ID No. 5; VL SEQ ID No. 55; and VL SEQ ID No. 75.
Resumen de: US2025268844A1
The invention discloses novel 5-substituted 4-hydroxy-cyclopent-2-en-1-one derivatives as active compounds in pharmaceutical compositions for the treatment of cancer. The invention confers cytotoxic effects of the said compounds on ovarian, colorectal, cervical, hepatocellular, lung, bladder and breast carcinomas, melanoma, lymphoma, leukemia and myeloma malignant cells, for the inhibition of cell cycle progression at the G2/M phase, for reducing the expression of DNA replication licensing factors and for having general cancer treatment effects. It further discloses the use of the said compositions for the treatment of platinum-resistant tumors. Another aspect of the invention is the synergetic effects of these compounds with existing cancer treatment medicaments as the proteasomal inhibitors. Finally, the synthetic methods of the active compounds of the said com-positions are disclosed.
Resumen de: US2025268878A1
The invention relates to the use of ferroportin inhibitor compounds of the general formula (I)for treating myelodysplastic syndromes (MDS).
Resumen de: US2025268920A1
Provided is a method for treating a blood cancer in an individual in need thereof by administrating to the individual an agent that inhibits activity or expression of one or more enzymes that participate in synthesis of either or both dihydrotestosterone (DHT) or androgen receptor (AR), or an antagonist AR, or a combination of an agent and the antagonist. The methods are shown in connection with acute myeloid leukemia (AML).
Resumen de: WO2025176039A1
Provided are use of an IL-1α inhibitor in preparing a medicament for treating leukemia therapy-induced cardiac injury and use of an IL1R1 antagonist in preparing a medicament for treating leukemia therapy-induced cardiac injury. The present disclosure can effectively reduce cardiovascular complications and the occurrence of long-term cardiovascular adverse events in the period of leukemia treatments, thereby helping alleviate the cardiac metabolic dysfunction and cardiac dysfunction after treatment.
Resumen de: WO2025178607A1
A method and apparatus for labeling a tissue section is provided. In certain aspects, the methods comprise labeling a tissue sample via a plurality of immunohistochemistry (IHC) assays for detection of markers for characterization of a non-Hodgkin's lymphoma. The disclosed IHC assays employ chromogen-based detection methods for improved sample efficiency and visualization of biomarkers. Further disclosed is an apparatus for carrying out the disclosed methods.
Resumen de: US2025269065A1
Provided herein is a gene therapy for BAG3 (B-cell Lymphoma 2-Associated Anthanogene 3), e.g., using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter, a cardiac troponin T (hTNNT2) promoter, a heat shock protein 70 (HSP70) promoter, or a ubiquitin C (UBC) promoter. The capsid may be an AAVrh.74 or AAV9 capsid or a functional variant thereof. In certain embodiments, the capsid is an AAVrh.74 capsid or functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the AAV vector, and other compositions and methods.
Resumen de: US2025270193A1
The present disclosure provides compounds which are useful in the treatment of oncological disorders, more particularly arylimidamides useful in the treatment of leukemias. Exemplary compounds include an azole moiety connected to a phenoxy or pyridyloxy moiety via an alkylene chain, the phenoxy or pyridyloxy moiety attached to a benzimidamide or pyridylimidamide function.
Resumen de: US2025270254A1
The present disclosure provides a probe specifically binding to CD138, a kit and a microfluidic chip comprising the probe, and a method of diagnosing diffuse large B-cell lymphoma in a subject using the probe, the kit, or the microfluidic chip. The present disclosure also provides a method of screening the probe for diagnosing diffuse large B-cell lymphoma.
Resumen de: AU2025213596A1
Provided is an active ingredient of a pharmaceutical composition for treating myeloma. Specifically, provided is an antibody whose epitope is present in the region of the amino acid 5 residue positions 33 to 109 of human integrin β7. Provided is an active ingredient of a pharmaceutical composition for treating myeloma. Specifically, provided is an antibody whose epitope is present in the region of the amino acid 5 residue positions 33 to 109 of human integrin ß7. ug r o v i d e d i s a n a c t i v e i n g r e d i e n t o f a p h a r m a c e u t i c a l u g c o m p o s i t i o n f o r t r e a t i n g m y e l o m a p e c i f i c a l l y , p r o v i d e d i s a n a n t i b o d y w h o s e e p i t o p e i s p r e s e n t i n t h e r e g i o n o f t h e a m i n o a c i d r e s i d u e p o s i t i o n s t o o f h u m a n i n t e g r i n ß
Resumen de: US2025268941A1
The present disclosure relates to BCMA/CD19 CAR T-cell products and methods for treating relapsed or refractory BCMA+ or CD19+ malignancies.
Nº publicación: AU2025217281A1 28/08/2025
Solicitante:
BIOGEN MA INC
Biogen MA Inc
Resumen de: AU2025217281A1
Provided herein, in some embodiments, are methods for reducing the risk of developing progressive multifocal leukemia in patients undergoing natalizumab therapy by switching to an extended interval dosing (EID) schedule. Provided herein, in some embodiments, are methods for reducing the risk of developing progressive multifocal leukemia in patients undergoing natalizumab therapy by switching to an extended interval dosing (EID) schedule. ug r o v i d e d h e r e i n , i n s o m e e m b o d i m e n t s , a r e m e t h o d s f o r r e d u c i n g t h e r i s k o f d e v e l o p i n g p r o g r e s s i v e m u l t i f o c a l l e u k e m i a i n p a t i e n t s u n d e r g o i n g n a t a l i z u m a b t h e r a p y b y u g s w i t c h i n g t o a n e x t e n d e d i n t e r v a l d o s i n g ( ) s c h e d u l e