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: WO2025134050A1
Embodiments of the present disclosure relate to methods of treating multiple myeloma in a subject in need thereof, comprising administering a therapeutically effective amount of a BCMA x GPRC5D x CD3 trispecific antibody or trispecific binding fragment thereof, to the subject to the subject to treat the multiple myeloma.
Resumen de: WO2025132479A1
The role of pro- and anti-inflammatory macrophages in disease depends on the specific disease and the balance between the two types of macrophages. Imbalances can lead to the development or progression of diseases, and targeting macrophage function may be a potential therapeutic approach for certain diseases. For example, by altering the phenotype and function of TAM-M2, it is possible to shift the immune response from an anti-inflammatory to a pro-inflammatory state, which may help reduce tumor progression. Here, the inventors demonstrate the ability of Gilteritinib to reprogram primary human macrophages. Gilteritinib is a targeted therapy used in the treatment of acute myeloid leukemia (AML) with a specific genetic mutation called FLT3-internal tandem duplication (FLT3-ITD) or a tyrosine kinase domain (TKD) mutation. Accordingly, the present invention relates to an FLT3 inhibitor to modulate the immune environment and enhance existing therapies by blocking immunosuppressive immune cells.
Resumen de: WO2025137368A1
A myelodysplastic syndrome-related detection system obtains single-nucleus or single-cell transcriptome data for genes for each of a plurality of nuclei or cells. Each nucleus or cell is obtained from a sample from a different subject in a cohort. Metadata for each respective subject is obtained that indicates whether the respective subject has myelodysplastic syndrome or is healthy. The nuclei or cells are clustered into clusters by computing distances using the transcriptome data for the genes for each unique pair of nuclei or cells in the plurality of nuclei or cells and through evaluation of these distances with a criterion function. Each cluster has a subset of nuclei or cells. The metadata identifies a cluster associated with the myelodysplastic syndrome on the basis that the cluster is enriched for nuclei or cells from subjects having the myelodysplastic syndrome.
Resumen de: US2025207202A1
The present disclosure provides kits and/or methods of detecting and identifying epigenetic patterns associated with acute myeloid leukemia and other cancers. The present disclosure also relates to treating, preventing, ameliorating, or reducing acute myeloid leukemia and other cancers.
Resumen de: WO2025137567A1
Some embodiments of the disclosure include compounds of Formula (I) and Formula (la), and compositions thereof, 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 compounds (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 IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
Resumen de: US2025205309A1
The disclosure provides methods of treating a blood cancer, such as multiple myeloma, by administering a combination of a heterodimeric protein comprising a first monomer comprising an IL15 protein-Fc domain fusion and a second monomer comprising an IL15Rα protein-Fc domain fusion, such as XmAb24306, and a FcRH5xCD3 bispecific antibody, such as cevostamab.
Resumen de: US2025205248A1
A method of a clinical use of varenicline apart from a smoking cessation is provided, including supressing inflammatory cytokines with the varenicline and repositioning the varenicline as an anti-inflammatory drug. The varenicline is repositioned in an LPS-induced in-vitro inflammation model by using a commercially available and widely used immortalized macrophage cell line obtained from male adult Balb/c mice and transformed by Abelson murine leukemia virus.
Resumen de: US2025206835A1
The present invention relates to the treatment of subjects having CD20-positive cell proliferative disorders (e.g., B cell proliferative disorders, such as non-Hodgkin's lymphomas). More specifically, the invention pertains to the treatment of subjects having a CD20-positive cell proliferative disorder (e.g., B cell proliferative disorder) by administering a combination of an anti-CD20/anti-CD3 bispecific antibody and an anti-CD79b antibody drug conjugate.
Resumen de: WO2025137363A1
The disclosure relates, inter alia, to methods for monitoring cellular transitions associated with or indicative of a presence or a stage of a myelodysplastic syndrome-related disease.
Resumen de: US2025197495A1
Provided are methods for clinical treatment of hematological malignancies, such as relapsed or refractory chronic lymphocytic leukemia or lymphoma using an anti-LAG-3 antibody. Particular malignancies include, e.g., chronic lymphocytic leukemia (CLL), Hodgkin lymphoma (HL), or non-Hodgkin lymphoma (NHL).
Resumen de: WO2025128434A1
The disclosure describes T cells that express chimeric antigen receptors (CARs), as well as pharmaceutical compositions comprising T cells and methods of making and using such T cells. Particularly, this disclosure describes T cells expressing a CAR that binds to CD64, and methods of use in treating acute myeloid leukemia.
Resumen de: US2025197471A1
Provided are adoptive cell therapy methods involving the administration of doses of cells for treating B cell malignancies. The cells generally express recombinant receptors such as chimeric antigen receptors (CARs). In some embodiments, the methods are for treating subjects with chronic lymphocytic leukemia (CLL). In some embodiments, the methods are for treating subjects with non-Hodgkin lymphoma (NHL). In some embodiments, the methods involve prior administration of a lymphodepleting therapy, such as prior administration of fludaribine and/or another lymphodepleting chemotherapeutic agent, for example cyclophosphamide. In some embodiments, features of the methods include an increase in complete remission, overall survival and/or progression free survival of subjects treated in accord with the provided methods.
Resumen de: US2025195653A1
The present disclosure relates to methods for treating or preventing a leukemia or a myelodysplastic syndrome in a subject in need thereof, said method comprising administering to the subject an effective amount of a combination comprising: (i) a binding protein comprising a first antigen binding domain (ABD) with binding specificity to CD123 and a second (ABD) with binding specificity to NKp46; and one or both of: (ii) a BCL-2 inhibitor, and (iii) a DNA hypomethylating agent.
Resumen de: US2025195696A1
In some aspects, the disclosure relates to compositions and methods useful for maintaining or improving retinal function and/or morphology. The disclosure is based, in part, on isolated nucleic acids encoding certain neurotrophic factors (e.g., leukemia inhibitory factor (LIF), etc.) and gene therapy vectors (e.g., recombinant adeno-associated virus (rAAV) vectors) encoding the same. In some embodiments, isolated nucleic acids and gene therapy vectors described by the disclosure are useful for treatment of certain diseases or disorders of the eye, for example retinal degeneration, retinitis pigmentosa (RP), age-related macular degeneration (AMD), glaucoma, etc.
Resumen de: US2025195666A1
The disclosure provides, inter alia, compounds comprising Toll-like receptor 9-binding nucleic acid sequences and nucleic acid sequences comprising a microRNA-142 passenger strand sequence hybridized to a microRNA-142 guide strand sequence; pharmaceutical compositions comprising the compounds; and the use of the compounds and pharmaceutical compositions to treat myeloid leukemia.
Resumen de: US2025195616A1
Disclosed in a method of treating a myeloid neoplasm, acute leukemia, or infectious disease in a subject, the method including administering to a subject in need thereof a pegylated interferon-α at a regular interval of every 2 to 8 weeks at a first dose of 250 to 500 μg.
Resumen de: US2025195498A1
The present invention relates to an inhibitor of the Helicobacter pylori (H. pylori) cell purine nucleotide salvage pathway (reserve pathway) for use in the treatment of H. pylori infection and/or the treatment or prevention of a disease associated with this infection, such as gastritis and duodenitis, gastric and duodenal ulcers, as well as gastric cancer and MALT-type lymphoma, as well as pancreatic cancer or neurodegenerative diseases or disorders. The present invention also provides a pharmaceutical composition comprising such an inhibitor of the H. pylori cell purine nucleotide salvage pathway and at least one pharmaceutically acceptable excipient. The present invention further provides a pharmaceutical composition comprising such an inhibitor of the H. pylori cell purine nucleotide salvage pathway, at least one pharmaceutically acceptable excipient and at least one additional drug for the treatment of H. pylori infection, selected in particular from an antibiotic, a proton pump inhibitor and a bismuth salt.
Resumen de: US2025195472A1
An indole alkaloid, specifically the alkaloid (3S,3aR,8aS)-3-butyl-5-hydroxy-3,3a,8a-trimethyl-3,3a,8,8a-tetrahydro-2H-furo2,3-bindole-2-one, with the generic name Andranone, as an antineoplastic agent against neoplastic cells of colon cancer, breast cancer, leukaemia, central nervous system cancer, prostate cancer, lung cancer and cervical cancer.
Resumen de: US2025197528A1
The present disclosure relates to multifunctional binding proteins comprising a first and a second antigen binding domains (ABDs) and all or part of an immunoglobulin Fc region or variant thereof, wherein the first ABD binds specifically to human BCMA and the second ABD binds specifically to human NKp46 and wherein all or part of the immunoglobulin Fc region or variant thereof bind to a human Fc-γ receptor.The disclosure also relates to methods for making said binding proteins, compositions thereof, and their uses, including the treatment or prevention of proliferative disorders, including multiple myeloma (MM).
Resumen de: US2025197510A1
The present invention provides novel human fms related tyrosine kinase 3 (FLT3) antigen binding proteins, such as antibodies, having improved FLT3 binding affinity, and/or anti-tumor activity. The FLT3 antibodies of the invention were generated by mutation of a parent FLT3 antibody and tested in in vitro in binding assays as well as in vivo in a mouse tumor model and in human patient tumor samples. The antibodies of the invention are provided as monospecific constructs or in a bispecific FLT3xCD3 antibody format and show excellent target affinity and/or tumor cell killing. The present invention also relates methods for producing the antigen binding proteins of the invention as well as nucleic acids encoding them, vectors for and host cells for their expression. The invention further relates to methods of treating or diagnosing a disease such as leukemia using an FLT3 antigen binding protein (ABP) of the invention.
Resumen de: US2025195678A1
The present application relates to a conjugate comprising an amatoxin, a target-binding moiety wherein the target is CD20, i.e., a CD20-binding moiety, and optionally a linker linking said amatoxin and said CD20-binding moiety. The invention further relates to the synthesis of said conjugate. In addition, the invention relates to a pharmaceutical composition comprising such conjugate, particularly for use in the treatment of B-cell and/or lymphoma associated diseases and/or malignancies.
Resumen de: US2025195646A1
The present disclosure describes a pharmaceutical combination of an anti-CD19 antibody and a phosphoinositide 3-kinase inhibitor for the treatment of non-Hodgkin's lymphoma, chronic lymphocytic leukemia and/or acute lymphoblastic leukemia.
Resumen de: US2025197392A1
The present invention relates to crystalline forms of an inhibitor of menin/mixed lineage leukemia (MLL) protein-protein interaction. The present invention also relates to pharmaceutical compositions comprising crystalline forms of an inhibitor of menin/mixed lineage leukemia (MLL) protein-protein interaction. These crystalline forms and pharmaceutical compositions comprising said crystalline forms may be useful for treating diseases such as cancer.
Nº publicación: AU2023405289A1 19/06/2025
Solicitante:
UNIV DE BARCELONA
FUNDACIO DE RECERCA CLINIC BARCELONA INST DINVESTIGACIONS BIOMEDIQUES AUGUST PI I SUNYER
HOSPITAL CLINIC DE BARCELONA
UNIVERSITAT DE BARCELONA,
FUNDACI\u00D3 DE RECERCA CL\u00CDNIC BARCELONA-INSTITUT D'INVESTIGACIONS BIOMEDIQUES AUGUST PI I SUNYER,
HOSPITAL CLINIC DE BARCELONA
Resumen de: AU2023405289A1
The present invention relates to a CD229 and a BCMA targeting moiety, wherein the CD229 targeting moiety is an antibody, F(ab')2, Fab, scFab or scFv. The present invention further provides CARs, nucleic acid, cells, pharmaceutical compositions and kits comprising the CD229 and BCMA targeting moiety. Methods of treatment of a CD229-positive cancer, preferably Multiple Myeloma, are also provided.