Resumen de: WO2025080924A1
Provided herein is a recombinant adeno-associated virus (rAAV) comprising an AAV capsid and an expression cassette comprising an engineered nucleic acid sequence encoding an anti-hCD20 antibody (i.e., rituximab) comprising heavy chain and light chain operably linked to regulatory control sequences which direct expression of rituximab in a target cell. Also provided are a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, nucleic acid molecule, packaging host cells, rAAV production system, and a method of treating CD20-positive central nervous system lymphomas
Resumen de: WO2025077315A1
Provided are an anti-BCMA single-domain antibody, and a preparation method therefor and the use thereof. Specifically, provided is a single-domain antibody having an amino acid sequence of SEQ ID No. 1. The single-domain antibody has high affinity, can thoroughly specifically target BCMA-positive cells, and can be applied to the detection of BCMA expression in bone marrow cells of MM patients. The single-domain antibody can be prepared into a specific antibody drug clinically used for preventing and treating BCMA-target-related diseases (such as multiple myeloma, B-cell acute lymphoblastic leukemia, non-Hodgkin's lymphoma and Hodgkin's lymphoma); or a BCMA protein detection kit, etc. The single-domain antibody has a stable structure, a small molecular size, is easily recombinantly expressed and has a low production cost, can be used alone or as a drug delivery system to carry relevant drugs, and has very wide prospects and important significance in fields such as drug application and clinical diagnosis.
Resumen de: WO2025080753A1
The present invention relates to bifunctional compounds, which find utility to degrade and (inhibit) one or more of the following kinases: DYRK1A, DYRK1B, DYRK2, DYRK3, DYRK4, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, HIPKs, and/or CMGC kinases leading to inhibition of WNT signaling. In particular, the present invention is directed to compounds, which contain on one end an E3 ubiquitin ligase binding moiety which binds to an E3 ubiquitin ligase and on the other end a moiety which binds one or more of the following kinases: DYRK1A, DYRK1B, DYRK2, DYRK3, DYRK4, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, HIPKs, and/or CMGC kinases leading to inhibition of WNT signaling, such that the one or more kinases is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of the one or more kinases. The bifunctional compounds serve as therapeutics for the treatment of Alzheimer's disease, down syndrome, diabetes, an autoimmune disease, an inflammatory disorder (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer, acute myeloid leukemia, myelodysplastic syndrome), a viral infection (e.g., SARS-CoV-2 infection (e.g., COVID-19)), and other diseases.
Resumen de: EP4538294A1
The present application relates to a multi-specific binding molecule targeting BCMA, GPRC5D and a T cell receptor. In particular, the present application discloses a multi-specific antibody against BCMA, GPRC5D and CD3, which can bind to a tumor surface antigen while activating T cells, thereby promoting the specific killing of tumor cells, in particular BCMA-positive or GPRC5D-positive multiple myeloma, by T cells. The present application further provides a preparation method for and the application of the multi-specific binding molecule.
Resumen de: WO2025076428A1
The present disclosure relates to a method for classifying a subject diagnosed with follicular lymphoma (FL) into 7 FL subtypes (FL1-FL7) and related methods of treating a subject diagnosed with FL. The present disclosure further includes related compositions, e.g., isolated probes and primers, as well as kits and arrays comprising same. A therapy guidance software tool is also disclosed.
Resumen de: WO2025076471A2
Disclosed are chimeric antigen receptors that target glycoprotein A repetitions predominant (GARP) and methods of their use in the treatment of cancer including, but not limited to breast cancer, bladder cancer, glioblastoma, or leukemia or other malignancies characterized with GARP+ Tregs.
Resumen de: WO2025076472A1
Provided herein are methods and uses of combination therapies involving GPRC5D- targeted cell therapy comprising chimeric antigen receptors (CARs), which contain extracellular antigen-binding domains that bind to G Protein-Coupled Receptor Class C Group 5 Member D (GPRC5D), and a combination agent, e.g. an immunomodulatory compound, for treating subjects with cancers such as multiple myeloma, and related methods, uses, and articles of manufacture.
Resumen de: WO2025076501A1
Disclosed herein are high throughput synthetic methods for the deliberate and prospective discovery of molecular glues which can be used to form composite protein-ligand surfaces that facilitate interfacial binding to other proteins over dispersed surfaces. In particular, this application discloses a high throughput approach using sulfur(VI) fluoride exchange (SuFEx) transformations and N-hydroxysuccinimide (NHS)-ester derived amide couplings to prospectively repurpose known ligands for a prolein-of-interest into degraders and compounds capable of inducing proximity to other proteins. Disclosed herein are methods of developing known ligands of a target protein into degraders of the target proteins. Further disclosed are methods of developing novel small molecule chromatin-competitive inhibitors of the eleven nineteen leukemia (ENL) YEATS domain into effective degraders of ENL.
Resumen de: WO2025073216A1
Provided are a novel DNA methylation marker TAGMe-5 for tumor identification and a use thereof. The tumor marker TAGMe-5 shows a significant DNA methylation difference in para-carcinoma tissues and carcinoma tissues, the difference has remarkable statistical significance, and the difference is shown in a plurality of tumors such as solid tumors and non-solid tumors. The solid tumors comprise lung cancer, liver cancer, prostate cancer, cervical cancer, endometrial cancer, urothelial carcinoma, a biliary tract tumor, etc. The non-solid tumors comprise a blood system tumor, lymphoma, etc. Therefore, the tumor marker can be used for screening, molecular diagnosis, prognosis, and therapeutic effect evaluation for clinical multi-tumors (Pan-cancer).
Resumen de: US2025115665A1
Provided are methods of clinical treatment of Diffuse Large B-cell Lymphoma (for example, relapsed and/or refractory Diffuse Large B-cell Lymphoma) in human subjects using a bispecific antibody which binds to CD3 and CD20 in combination with lenalidomide or ibrutinib and lenalidomide.
Resumen de: WO2025068340A1
The present invention relates to a method for predicting the survival time of a patient suffering from an Acute Myeloid Leukemia (AML). In this study, the inventors used conditional Jam-3-deficient mice crossed with iMLL-AF9 leukemia model. They found that Jam-3-deficiency rewired the transcriptional program of leukemia initiating cells (LIC) with upregulation of genes belonging to AP-l/TNF-α pathways. Transposition of results to human allowed to determine a new prognosis score called ATIC for AP-l/TNF-α Initiating Cells, complementary and distinct from the LSC17 score. Thus, the invention relates to a method for predicting the survival time of a patient suffering from an Acute Myeloid Leukemia (AML) based on the determination in a sample obtained from the patient of the expression levels of at least 7 genes selected in the group consisting in: JAM3, DUSP1, JUN, IER2, DUSP2, RGS1, H2BC8, PTGS2, NFKBID, PPP1R15A, NFKBIZ, ZFP36, SNORA28, TPT1, KLF2, BTG2, JUNB, JUNE), ATF3, UBC, SKIL, TAF7, SLFN12L, NR4A1, CHST2, GASS, SNORA31, HES1, EGR3, RPS13, PMAIP1, RHOB, MYL9, ZNF699, ZNF101, FOS, FJX1, RPP25L, HEY1, PTMA, GIMAP4, EFCAB11, FOSE, CD14, CCL4, CCL3, PF4, OSM, CD69, ITGA2B, VWF, MYCN and F2RL2.
Resumen de: WO2025071406A1
The invention relates to the field of leukemia/lymphoma diagnosis, more specifically to the detection of MRD in bone marrow, blood and other fluids and tissues from patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma (T-ALL/T-LBL). Provided is a reagent composition for the cytometric detection of minimal residual disease (MRD) in T-ALL and/or T-LBL, the reagent composition comprising a panel of at least four antibodies conjugated to a detectable label, the panel comprising antibodies against markers NKp80, CD16, cyCD3 and smCD3.
Resumen de: AU2021239828A1
This invention relates to compounds for treating acute myeloid leukemia or inhibiting recurrence of acute myeloid leukemia and for inhibiting growth of and/or killing leukemic stem cells.
Resumen de: US2025110131A1
Breast implant-associated anaplastic large cell lymphoma (BIA-ALCL) is a rare T-cell lymphoma that can develop around breast implants. The disclosure is directed towards devices and methods for diagnose BIA-ALCL from the seroma (fluid) surrounding the implant using a lateral flow assay (LFA) detecting CD30 and/or one or more cytokines known to be produced by tumor cells in BIA-ALCL.
Resumen de: US2025108043A1
Provided are systems and methods for continuously administering to a subject in need of treatment a formulation comprising an immunomodulatory imide compound. In some embodiments, the method are for use in treating multiple myeloma, transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes, mantle cell lymphoma, hematologic cancers, or solid tumor cancers.
Resumen de: US2025109198A1
Described herein are approved products and methods of using approved products for treating relapsed or refractory multiple myeloma in a patient. Also described herein are methods of selling or offering for sale an approved product.
Resumen de: US2025109211A1
Disclosed are methods of treating cancers and enhancing efficacy of BCMAxCD3 bispecific antibodies. In particular, methods are disclosed of using a BCMAxCD3 bispecific antibody, an anti-CD38 antibody and/or pomalidomide to treat cancers, particularly relapsed or refractory multiple myeloma.
Resumen de: AU2025201932A1
Abstract The present disclosure provides compositions and methods useful for treating Glioblastoma Multiforme (GBM), e.g., compositions comprising virus-like particles (VLPs) comprising Moloney Murine leukemia virus (MMLV) core proteins and the human cytomegalovirus epitopes, gB and pp65, formulated with GM-CSF, which, at dose of at least 10 pg gB/pp65Gag, reverse dysregulation of anti-HCMV immunity in GBM patients. 21570040_1 (GHMatters) P117888.AU.1
Resumen de: WO2025071306A1
The present invention relates to a pharmaceutical composition for treating cancer in which an elF4A inhibitor and at least one of a BCL-2 inhibitor or a selective export protein (XPO-1) inhibitor are used in a combination regimen. The present invention relates to a pharmaceutical composition for treating cancer, wherein the elF4A inhibitor is one selected from the group consisting of silvestrol, CR-1-3B, zotatifin, rohinitib, didesmethylrocaglamide, and episylvestrol, and used in a combination regimen with at least one of a BCL-2 inhibitor or a selective nuclear export protein inhibitor.
Resumen de: WO2025072081A1
Provided herein are systems, kits, and methods for generating an enriched T cell population from an initial peripheral blood mononuclear cell (PBMC) population using at least two types of cell-binding reagents: (e.g., particles conjugated to CD32, CD19, CD244, or CD25 binding agents), where the enriched T cell population is: i) enriched for desired T-cells (e.g., early memory T cells and naïve T cells), and ii) depleted in normal and malignant non-desired cells selected from: CD25hi regulatory T-cells (Tregs), CD25hi CLL cells, CD244 T-cells, CD32+ monocytes, CD32+ myeloid leukemia cells, CD32 basophils, CD19+ and/or CD32+ B cells, CD244+ natural killer (NK) cells, and myeloid cells). In certain embodiments, the enriched T cell populations are used for generating a population of chimeric antigen receptor (CAR) T-cells, T-cell receptor-engineered T cells, or Tumor-infiltrating T lymphocyte (ITL) products.
Resumen de: WO2025072692A1
Compositions for treating hematological conditions such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) are provided. In particular, a population of CD8+ T lymphocytes with cytotoxic activity against leukemia cells is provided, as well as methods of isolating and enriching such cells for therapeutic applications.
Resumen de: WO2025072637A1
Disclosed herein is a class of small-molecules having oxygenated heterocyclic ring structure. Compounds disclosed herein are lysine demthylase-1 (LSD-1) inhibitors, and accordingly, also disclosed herein is the use the compounds as therapeutics for the treatment of hematological disorders (e.g., sickle cell disease (SCD), β-thalassemia), cancer (e.g., acute myeloid leukemia (AML), multiple myeloma, biliary tract cancer, non-small cell lung cancer (NSCLC), chronic lymphocytic leukemia, advanced solid tumor, advanced malignancies), and/or a neurological disorder (e.g., Alzheimer's disease (AD), Amyotrophic Lateral Sclerosis (ALS), Parkinson disease (PD), Schizophrenia, Huntington disease (HD)), a metabolic disorder (e.g., type-2 diabetes (T2D), obesity) and other conditions related to LSD-1 activity (e.g., mild to moderate Alzheimer's disease, myocardial fibrosis, autism, complex neurodevelopmental diseases).
Resumen de: WO2025067468A1
Described herein are Casitas B-lineage lymphoma (Cbl) inhibitors and pharmaceutical compositions comprising said inhibitors. The subject compounds and compositions are useful for the treatment of a disease or condition associated with Cbl-b activity, such as cancers.
Resumen de: WO2025065747A1
Disclosed is an application of a CSF1R inhibitor in the preparation of an NK/T cell lymphoma treatment drug. Further disclosed are a use as an NK/T cell lymphoma marker, a use of a CSF1 detection reagent in the preparation of an NK/T cell lymphoma prognosis kit, and a use of the CSF1 detection reagent in the preparation of an NK/T cell lymphoma primary screening kit. The present invention can be promoted and applied to primary screening, prognosis and treatment of clinical NK/T cell lymphoma patients.
Nº publicación: EP4530630A2 02/04/2025
Solicitante:
ELANCO TIERGESUNDHEIT AG [CH]
Elanco Tiergesundheit AG
Resumen de: EP4530630A2
This disclosure relates to immunogens and monoclonal antibodies useful in the identification and/or treatment of cancer cells, including those of the dog. In one example, chimeric anti-canine CD20 antibodies are provided. The antibodies can be used therapeutically to treat lymphoma in dogs.