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61
resultados
Última actualización
16/07/2025 [06:46:00]
Resumen de: WO2025147212A1
The invention relates to a kit for disrupting binding sites of repressor leukemia/lymphoma-related factor (LRF) of a promotor of a hemoglobin gamma (HbG) locus, compositions comprising said kit, and medical use thereof for treating and preventing β-hemoglobinopathy in a subject, wherein the kit comprising a CRISPR-Cas9 nickase and a pair of guide RNAs (gRNAs), wherein the pair of gRNAs binds to target sequences and results in a disruption of binding sites of repressor LRF in a promotor of HbG locus.
Resumen de: WO2025145959A1
Disclosed in the present invention are three crystal forms of thienopyrimidine compound A and a preparation method therefor, wherein crystal form I is of a trihydrochloride salt, and crystal forms II and III are of dihydrochloride salts. The three crystal forms all have good stability and solubility, with a solubility of ≥4 mg/mL in a 5% aqueous glucose solution, so that the basic requirements for the solubility of a compound to be prepared into a lyophilized preparation for injection are met. The three crystal forms of thienopyrimidine compound A prepared in the present invention have good stability and solubility, can be prepared into a lyophilized preparation for clinical injection, and can be used for the effective treatment of patients with advanced, recurrent, etc. lymphoma, myeloma and lymphocytic leukemia, or drug-resistant patients.
Resumen de: WO2025145476A1
Disclosed in the present invention is a use of an MP1 gene copy number variation detection reagent in preparation of an NK/T cell lymphoma prognosis kit. The present invention further provides a detection kit comprising the LMP1 gene copy number variation detection reagent, a use thereof, and an NK/T cell lymphoma prognosis method. In the present invention, a copy number variation event of LMP1 gene in an EB virus genome is used as a novel prognosis marker, which has a good prediction value for the overall survival and progression-free survival prognosis of an NK/T cell lymphoma patient, and can realize more accurate and effective prognostic assessment for the NK/T cell lymphoma patient.
Resumen de: AU2023385514A1
The instant invention relates to combinations of an LSD1 inhibitor (or a pharmaceutically acceptable salt thereof) and a Menin inhibitor (or a pharmaceutically acceptable salt thereof). The combinations are particularly useful for treating cancer, including hematological cancers, such as acute myeloid leukemia or myelodysplastic syndrome.
Resumen de: US2025222107A1
The present disclosure provides chimeric antigen receptor (CAR) molecule specific for B cell maturation antigen (BC-MA), compositions and methods for treating immune-related disorders, specifically, plasma cell pathologies such as multiple myeloma (MM).
Resumen de: US2025221961A1
The disclosure herein provides combination therapies for the treatment of cancers such as Leukemia, lymphoma and triple negative breast cancer. The disclosure provides combination therapies of CDK inhibitors, e.g., a CDK inhibitor represented by Formula I:or a pharmaceutically acceptable salt thereof together with a BCL-2 inhibitor or proteasome inhibitor for the treatment of cancer.
Resumen de: US2025223360A1
The present invention provides bispecific antigen-binding molecules comprising a first antigen-binding domain that specifically binds human CD28, and a second antigen-binding molecule that specifically binds human CD-22. In certain embodiments, the bispecific antigen-binding molecules of the present invention are capable of inhibiting the growth of tumors expressing CD-22, such as B-cell lymphomas. The antibodies and bispecific antigen-binding molecules of the invention are useful for the treatment of diseases and disorders in which an up-regulated or induced targeted immune response is desired and/or therapeutically beneficial.
Resumen de: US2025223230A1
Introduction: Rituximab (R) is an integral component of therapy for B-cell lymphoid malignancies; bortezomib (Btz) has shown provocative single agent activity in Follicular Lymphoma (FL), Mantle Cell Lymphoma (MCL) and Waldenstrom's Macroglobulinaemia (WM), providing the rationale for investigating the combination.Patients+Methods: Forty-five adult patients (pts.) (30 men, 15 women) with histologically confirmed recurrent CD20+ve FL, MCL or WM, median age 60 years (range 45-79), FL: 17, MCL: 18, WM: 10, stage III/IV 40 (93%), bone marrow (BM) infiltration 32 (73%), elevated LDH 22 (49%), performance status ≥1 22 (49%), were enrolled in a randomised trial comparing 2 schedules of Brz+R: Arm A (twice weekly) Btz: 1.3 mg/m2 (on days 1, 4, 8, 11 of a 21-day cycle) and R: 375 mg/m2 (on day 1) for 8 cycles, or Arm B (weekly) Btz: 1.6 mg/m2 (on days 1, 8, 15, 22 of a 35-day cycle) and R: 375 mg/m2 (on days 1, 8, 15, 22 of cycles 1 and 4) for 6 cycles (23 arm A, 22 arm B). The median number of previous treatments was 2 (range 1-7). Seventeen pts. had received a R-containing regimen, with response lasting >6 months, and 8 high-dose treatment. Response was evaluated using the IWR criteria (Cheson et al, JCO 17:1244, 1999) and the updated response criteria from the 3rd International Workshop on WM (Treon et al, Blood 107:3442, 2006)Results: Ability to deliver the therapy, toxicity and efficacy were equivalent in both arms. The median number of cycles given in arm A was 4 and 5 in arm
Resumen de: US2025222068A1
The present invention relates to a composition for preventing or treating lupus or glomerulonephritis comprising a recombinant stabilized Galectin-9 protein. Specifically, the recombinant stabilized Galectin-9 protein of the present invention has been confirmed to exhibit safety in a systemic lupus erythematosus (SLE) animal model, reduce skin lesions, lymphadenopathy, and proteinuria caused by lupus, ameliorate lupus nephritis and glomerulonephritis, and decrease the concentration of anti-dsDNA antibodies in plasma. Accordingly, the recombinant stabilized Galectin-9 protein of the present invention can be effectively used as an active ingredient in a composition for preventing or treating lupus or glomerulonephritis.
Resumen de: US2025221944A1
The present invention provides a novel compound having a chromanone or its ring-opening form, phenylpropenone, as backbone and compositions for the preventing or treating multiple myeloma, comprising the same. The compounds of the invention exhibit significant killing effects against various multiple myeloma cell lines and show in vivo anti-cancer effects that exceed those of lenalidomide, a commercially available immunomodulator widely used in the treatment of multiple myeloma and myelodysplastic syndromes. In addition, the compounds of the invention exhibit a significant synergistic effect when co-administered with the thalidomide or its analog lenalidomide, and thus are useful as an efficient therapeutic agent or therapeutic aid agent composition for multiple myeloma which is an incurable disease.
Resumen de: EP4582087A1
A method for treating lymphoma is provided. A pharmaceutical composition for treatment of lymphoma, including BCV, a pharmaceutically acceptable salt thereof, or a solvate thereof is used. The lymphoma may be EBV-positive lymphoma. The lymphoma may be MYC-positive lymphoma. The pharmaceutical composition may be used in combination with a chemotherapeutic agent.
Resumen de: EP4582096A1
The present invention relates to a composition for preventing or treating lupus or glomerulonephritis comprising a recombinant stabilized Galectin-9 protein. Specifically, the recombinant stabilized Galectin-9 protein of the present invention has been confirmed to exhibit safety in a systemic lupus erythematosus (SLE) animal model, reduce skin lesions, lymphadenopathy, and proteinuria caused by lupus, ameliorate lupus nephritis and glomerulonephritis, and decrease the concentration of anti-dsDNA antibodies in plasma. Accordingly, the recombinant stabilized Galectin-9 protein of the present invention can be effectively used as an active ingredient in a composition for preventing or treating lupus or glomerulonephritis.
Resumen de: AU2022476674A1
The present disclosure relates generally to methods of treating T-cell lymphomas with combination therapies.
Resumen de: WO2025137751A1
The present invention relates to a polynucleotide encoding an anti-BCMA chimeric antigen receptor (CAR) and to the polypeptide corresponding to the anti-BCMA chimeric antigen receptor (CAR) itself. The present invention also relates to a vector and a composition, comprising an immune effector cell, comprising the polynucleotide, as well as a method for producing the modified immune effector cell and the use of the polynucleotide, the vector, the composition or the immune effector cell produced by the method for the manufacture of a drug for the treatment of multiple myeloma.
Resumen de: AU2025204455A1
- 55 - The invention relates to an isolated chimeric antigen receptor polypeptide (CAR), wherein the CAR comprises an extracellular antigen-binding domain, comprising an antibody or antibody fragment that binds a human CXC chemokine receptor type 5 (CXCR5) protein. The invention further relates to a nucleic acid molecule encoding the CAR of the invention, a genetically modified immune cell, preferably a T cell, expressing the CAR of the invention and the use of said cell in the treatment of a medical disorder associated with the presence of pathogenic cells expressing CXCR5, preferably pathogenic mature B cells and/or memory B cells, and/or pathogenic T cells and/or T follicular helper cells, in particular mature B cell non-Hodgkin’s lymphoma (B-NHL), T cell non-Hodgkin's lymphoma, or autoantibody-dependent autoimmune disease, preferably selected from systemic lupus erythematosus (SLE) or rheumatoid arthritis. - 55 The invention relates to an isolated chimeric antigen receptor polypeptide (CAR), wherein the CAR comprises an extracellular antigen-binding domain, comprising an antibody or antibody fragment that binds a human CXC chemokine receptor type 5 (CXCR5) protein. The invention further relates to a nucleic acid molecule encoding the CAR of the invention, a genetically modified immune cell, preferably a T cell, expressing the CAR of the invention and the use of said cell in the treatment of a medical disorder associated with the presence of pathogenic cells expressing C
Resumen de: AU2024213852A1
New PCT-application based on EP 23 154 047.7 Ludwig-Maximilians-Universität München, Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt Vossius Ref.: AF3867 PCT S3The present invention relates to the recognition of CD86 as a marker of hematological cancer and thus relates to CD86 targeting agents for the treatment of such cancers, in particular, acute myeloid leukemia (AML), Hodgkin's lymphoma (HL) and non-Hodgkin's lymphoma (NHL). The invention in particular encompasses a lymphocyte recombinantly expressing a chimeric antigen T cell receptor (CAR) comprising an antigen binding domain that specifically binds CD86 for use in the treatment of such cancers, as well as also encompassing the CAR construct, i.e., comprising an antigen binding domain that specifically binds CD86.
Resumen de: US2025213185A1
The present disclosure relates to a computer-implemented method of a computer-implemented method of determining a patient's response to a treatment in multiple myeloma. The method comprises:providing results of a series of predefined consecutive tests on the patient,determining a response at time t as a function of a test result of the time t and a subsequent test result of a time t+1.
Resumen de: US2025213562A1
Compositions and methods for treating lymphoma, in particular. T-cell lymphoma and follicular lymphoma. in a human patient are provided. The methods entail administering to the patient an effective amount of cerdulatinib.
Resumen de: US2025213701A1
The present disclosure provides a proteolysis-targeting compound TPB-L-E3B, a method for synthesizing the same, and use thereof. The compound can treat human tumor diseases through the eRF3a-targeting proteolysis mechanism, and exhibits great potential in treating such diseases in in-vitro studies, particularly, in treating diseases such as prostate cancer, ovarian cancer, liver cancer, cervical cancer, leukemia, breast cancer, and the like.
Resumen de: WO2025140579A1
The present application relates to a pharmaceutical composition, comprising an active ingredient compound 1, a pharmaceutically acceptable polymer carrier, a surfactant and other pharmaceutically acceptable excipients, wherein the active ingredient, the polymer carrier and optionally at least a portion of the surfactant are present in the form of a solid dispersion. Disclosed in the present application is the pharmaceutical composition containing the compound 1 for the first time. The composition has stable product quality and good bioavailability. On the basis of the characteristic that the compound 1 is a JAK inhibitor, the composition has wide application prospects for alopecia areata, vitiligo, atopic dermatitis, psoriasis, membranous nephropathy, ankylosing spondylitis, peripheral T cell lymphoma, ulcerative colitis, rheumatoid arthritis, systemic lupus erythematosus, Crohn's disease, etc.
Resumen de: AU2023390484A1
The present disclosure provides methods of treating acute myeloid leukemia (AML) and methods of determining responsiveness to AML treatment regimens, the methods comprising identifying the presence or absence of Myeloid/Lymphoid leukemia stem cells (M/L LSC) in a sample from a subject.
Resumen de: AU2023390486A1
The present disclosure provides methods of treating acute myeloid leukemia (AML) and methods of determining responsiveness to AML treatment regimens, including regimens comprising the administration of a BCL-2 inhibitor, a hypomethylating agent, a CD70-targeting agent, or any combination thereof, the methods comprising identifying the presence or absence of one or more biomarkers described herein.
Resumen de: AU2022489190A1
The present invention relates to an anti-CLL1 single-domain antibody and use thereof. Specifically, the present invention relates to a single-domain antibody having an amino acid sequence of SEQ ID No. 1. The single-domain antibody has high affinity, can specifically target a CLL1-positive cell, and can be applied to the detection of CLL1 expression in bone marrow cells of AML patients. The single-domain antibody can be prepared into a specific antibody drug clinically used for preventing and treating CLL1 target-related diseases (such as acute myeloid leukemia, myelodysplastic syndromes, or chronic myeloid leukemia), and can also be used for preparing CAR cells targeting CLL1, a detection kit for a CLL1 protein, or the like. The single-domain antibody drug is stable in structure, small in molecule, easy to recombinantly express, and low in production cost, and can be used alone or as a drug loading system to carry related drugs, which has very wide prospects and important significance in the fields of drug application, clinical diagnosis, and the like.
Resumen de: AU2023399623A1
This document relates to methods and materials involved in treating cancer. For example, this document provides cell engagers that bind to natural killer (NK) cells and bind to cancer cells. In some cases, a cell engager provided herein can include a first antigen binding domain having the ability to bind to a NK cell Group 2 isoform C (NKG2C) polypeptide and a second antigen binding domain having the ability to bind to a polypeptide present on the surface of a cancer cell. In some cases, a mammal (e.g., a human) having cancer (e.g., a leukemia such as acute myeloid leukemia (AML)) can be administered one or more cell engagers provided herein to treat the cancer.
Nº publicación: WO2025139121A1 03/07/2025
Solicitante:
THERORNA SHANGHAI CO LTD [CN]
THERORNA SHANGHAI CO., LTD
Resumen de: WO2025139121A1
Provided is a circular RNA encoding CARs and the use thereof to create immune cells that target specific diseases, e. g., lymphoma, multiple myeloma and leukemia and auntoimmune diseases, such as systemic lupus erythematousus, lupus nephritis and myasthenia gravis.
BOPI
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