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81
results.
Updated on
05/06/2025 [06:45:00]
Absstract of: WO2024054564A2
MicroOrganoSpheres (MOS) generated using cells from multiple myeloma bone marrow biopsies are provided herein, as are methods and materials for making and using such MOS.
Absstract of: WO2024026019A1
This disclosure relates to methods for treating cancer in a subject identified as having chronic myelomonocytic leukemia (CMML), comprising administering an anti-ILT3 antigen binding protein, or antigen binding fragment to the patient every three weeks (Q3W).
Absstract of: CN118338900A
The present disclosure relates to compositions comprising nirogastat or a pharmaceutically acceptable salt thereof and methods of treatment.
Absstract of: WO2024023313A1
The invention relates to genetically engineered mouse models for multiple myeloma (MM) and their uses thereof for the development of multiple myeloma models as well as for the screening of compounds suitable for the treatment of multiple myeloma.
Absstract of: WO2025108429A1
A chimeric antigen receptors (CARs) and CAR-NK cells targeting both BCMA and GPRC5D and use thereof in treating multiple myeloma (MM) is provided. In particular, the disclosure relates to bi-specific CARs comprising: (a) a B-cell maturation antigen (BCMA) targeting domain; and (b) a G protein-coupled receptor of family C, group 5, member D (GPRC5D) targeting domain, preferably armored with such as IL15, and the corresponding coding polynucleotides for the CARs and vectors comprising the polynucleotides. Also provided is CAR-NK cell modified with the CAR (s), and use of the CAR and CAR-NK cell in the treatment of MM.
Absstract of: WO2025111214A1
The present invention relates to the use of roxadustat in the manufacture of a medicament for treating anemia in a subject having anemia associated with myelodysplastic syndrome (MDS). Methods for treating anemia in a subject having anemia associated with myelodysplastic syndrome comprising administering to the subject a therapeutically effective amount of roxadustat, thereby treating the anemia are also described.
Absstract of: WO2025111247A1
The present disclosure relates to methods of treating a subject having multiple myeloma, wherein the multiple myeloma has been determined to be refractory multiple myeloma, comprising administering to the subject a pharmaceutically acceptable form of Compound 1 and wherein the pharmaceutically acceptable from is selected from a gentisate form and a succinate form.
Absstract of: WO2025111533A1
This disclosure describes, in part, CAR-T cells comprising CDKN1B loss of function mutations, and method of using a BCMA binding CAR-T cell comprising a CDKN1B loss of function mutation to treat BMCA-expressing cancers (e.g., multiple myeloma).
Absstract of: US2025171753A1
The present invention relates to a peptide comprising an amino acid sequence EX1EAX2SGSS according to SEQ ID No. 1, or a pharmaceutically acceptable salt thereof, wherein X1 and X2 independently represent a linker which links the adjoining amino acids or an amino acid deletion, and wherein the peptide has an overall length in the range of from 7 to 30 amino acids. The present invention further relates to a pharmaceutical composition comprising the peptide, and the peptide for use in the preventive treatment of acute lymphoblastic leukemia, myelodysplastic syndrome or acute myeloid leukemia, particularly therapy-related acute lymphoblastic leukemia, therapy-related myelodysplastic syndrome or therapy-related acute myeloid leukemia.
Absstract of: US2025171535A1
Provided herein are compositions and methods related to depletion of diseased hematopoietic stem cells (HSC) using an anti-c-kit antibody. The compositions and methods described herein may be used to treat a subject in need of diseased HSC depletion due to a variety of diseases or disorders, such as myelodysplastic syndrome and acute myeloid leukemia.
Absstract of: AU2025203224A1
The disclosure is directed to an antibody-drug conjugate (ADC) comprising a monoclonal antibody, or an antigen-binding fragment thereof, directed against B-cell maturation antigen (BCMA) conjugated to a cytotoxin. The disclosure also provides compositions comprising the antibody-drug conjugate and methods of killing multiple myeloma cells (including multiple myeloma stems cells) that express BCMA by contacting multiple myeloma cells with the ADC.
Absstract of: US2025170160A1
Methods of monitoring therapeutic efficacy in a subject with MDS are provided. Also provided is a method of identifying a subject with myelodysplastic syndrome (MDS) for treatment with a telomerase inhibitor, and methods of treating MDS. The subject methods can include administering to the subject an effective amount of a telomerase inhibitor and assessing the hTERT expression levels in a biological sample obtained from the subject. In some cases, a 50% or greater reduction in hTERT expression level identifies a subject who has an increased likelihood of benefiting from treatment with the telomerase inhibitor. The subject can be naive to treatment with a HMA, lenalidomide, or both. In some cases, the subject is classified as having low or intermediate-1 IPSS risk MDS and/or MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA). In some instances, the telomerase inhibitor is imetelstat sodium.
Absstract of: US2025170125A1
This invention discloses use of (3-((1H-pyrazol3,4-bpyridine-5-substituted)ethinyl)-4-methyl-N-(4-((4-methylpiperazine-1-substituted)methyl)-3-(trifluoromethyl)phenyl)benzamide) and a pharmaceutically acceptable salt thereof in the manufacture of a medicament for treating acute lymphoblastic leukemia in particular precursor B-cell lymphoblastic leukemia.
Absstract of: US2025170174A1
Methods for treating leukemia are disclosed, comprising administering to a subject in need thereof therapeutically effective amounts of natural killer (NK)-derived exosomes. One non-limiting practical utilization of the NK-derived exosomes is induction therapy of acute myeloid leukemia (AML), to thereby immediately and effectively induce remission of the disease in newly diagnosed AML patients.
Absstract of: EP4559477A2
The present application provides compositions and methods for treating cancers, including follicular lymphoma, T cell lymphoma and adenoid cystic carcinoma, using an anti-CD137 antibody that specifically binds to an extracellular domain of human CD137. In some embodiment, combination therapies including the anti-CD137 antibody and an immune checkpoint inhibitor, and/or a chemotherapeutic agent are provided. Biomarkers such as total CD137, membrane bound CD137 (mCD137), soluble CD137 (sCD137), CD137 ligand, Ki67, CD8+ effector memory T (T<sub>em</sub>) cells, regulatory T (T<sub>reg</sub>) cells, and natural killer (NK) cell levels for the methods of treatment described herein are also provided.
Absstract of: CN119497710A
Compounds, compositions, and methods for treating cancers characterized by aberrant B cell lymphoma 6 (BCL6) activity are described.
Absstract of: US2025161279A1
The present disclosure provides methods of treating multiple myeloma in a subject with a Bcl-2 inhibitor, in particularly 2-((1H-pyrrolo 2,3-bpyridin-5-yl)oxy)-N-((4-((((1r,4r)-4-hydroxy-4-methylcyclohexyl)methyl)amino)-3-nitrophenyl)sulfonyl)-4-(2-((S)-2-(2-isopropylphenyl)pyrrolidin-1-yl)-7-azaspiro3.5nonan-7-yl)benzamide or a pharmaceutically acceptable salt thereof, or in combination with dexamethasone.
Absstract of: EP4559902A2
Disclosed herein are new compounds and compositions and their application as pharmaceuticals for the treatment of diseases. Methods of inhibition of KDM1A, methods of increasing gamma globin gene expression, and methods to induce differentiation of cancer cells in a human or animal subject are also provided for the treatment of diseases such as acute myelogenous leukemia.
Absstract of: WO2025104184A1
The present invention provides an in vitro method of generating a haematopoietic cell, the method comprising: a) Providing a cell genetically modified to comprise a nucleotide sequence(s) encoding exogenous transcription factors, the exogenous transcription factors comprising an ETS family transcription factor, T-cell acute lymphocytic leukaemia protein 1 (Tal1) and a GATA family transcription factor, wherein expression of the exogenous transcription factors from the nucleotide sequence(s) is inducible by culture with an inducer, and wherein the cell comprises a detectable expression level of the exogenous transcription factors; and b) Culturing the genetically modified cell in a differentiation medium which does not comprise the inducer, such that the expression level of the exogenous transcription factors in the cell is reduced to a level whereby the cell differentiates into a haematopoietic cell. Also provided are genetically modified haematopoietic cells, genetically modified cells and therapeutic uses thereof.
Absstract of: WO2025104045A1
The present invention relates to a method of treating an anaplastic lymphoma kinase (ALK) fusion-positive solid tumour or central nervous system tumour, comprising administering to a subject in need of such treatment a therapeutically effective amount of alectinib, or a pharmaceutically acceptable salt thereof, wherein the subject is aged <18 years.
Absstract of: WO2025106732A1
Single-nucleotide variants (SNVs) in key T cell genes can drive clinical pathologies and could be repurposed to improve cellular cancer immunotherapies. Here, we perform massively parallel base editing screens to generate thousands of variants at gene loci annotated with known or potential clinical significance. We discover a broad landscape of putative gain- (GOF) and loss-of-function (LOF) mutations, including in PIK3CD and its regulatory subunit PIK3R1, LCK, SOS, AKT1, and RHOA. Base editing of PIK3CD and PIK3R1 variants in T cells with an engineered T cell receptor specific against a melanoma epitope or in different generations of CD19 chimeric antigen receptor T (CAR-T) cells demonstrates that discovered GOF variants, but not LOF or silent mutation controls, enhanced signaling, cytokine production and lysis of cognate melanoma and leukemia cell models, respectively. Additionally, we show that generations of CD19 CAR-T cells engineered with PIK3CD GOF mutations demonstrate enhanced antigen-specific signaling, cytokine production, and leukemia cell killing, including when benchmarked against other recent strategies.
Absstract of: WO2025106552A1
Several embodiments of the methods and compositions disclosed herein relate to immune cells that are engineered to express chimeric receptors and various dosing regimens for administering such cells. In several embodiments, the immune cells express a chimeric receptor that targets ligands of NKG2D. In several embodiments, the cancer is a leukemia, such as acute myeloid leukemia (AML).
Absstract of: AU2025203054A1
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.
Absstract of: AU2023361041A1
The invention is directed to pharmaceutical compositions for oral administration comprising deferiprone. In particular the invention is directed to a modified-release formulation in form of mini-tablets suitable for twice-a-day oral administration for the treatment of diseases which cause an overload of iron for example, thalassemia, sickle cell anemia, hemochromatosis, and myelodysplasia, or for the prevention and/or treatment of diseases which are caused by an overload of iron. The invention is also directed to methods of making said formulation.
Nº publicación: AU2023385479A1 22/05/2025
Applicant:
JJR&D LLC
JJR&D, LLC
Absstract of: AU2023385479A1
Compositions and methods for treating cancer, particularly leukemia, using a cytotoxic composition comprising monocytes activated by 3-glucan. The monocytes are preferably incubated with the β-glucan and then processed to extract particles, such as microvesicles and exosomes from the treated monocytes to produce the cytotoxic composition. Preferably the cytotoxic composition comprises at least 50% exosomes having a size of 150 nm or less that are activated with β-glucan. Zymosan is the preferred β-glucan. The cytotoxic composition has an apoptosis effect. When a subject having cancer is treated according to preferred embodiments, the cytotoxic composition preferably induces a cytokine response in the subject's immune system. The combination of the cytotoxic composition and cytokine response are synergistic.
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