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PHARMACEUTICAL COMPOSITION

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.

POLYNUCLEOTIDE, CHIMERIC ANTIGEN RECEPTOR, VECTOR, COMPOSITION, METHOD FOR PRODUCING A MODIFIED IMMUNE EFFECTOR CELL AND USE OF THE POLYNUCLEOTIDE

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.

CHIMERIC ANTIGEN RECEPTOR AND CAR-T CELLS THAT BIND CXCR5

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

ANTI-CD86 CAR EXPRESSING LYMPHOCYTES FOR TARGETED TUMOR THERAPY

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.

DETERMINING A PATIENT’S RESPONSE TO A TREATMENT IN MULTIPLE MYELOMA

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.

METHODS FOR TREATING LYMPHOMA

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.

MEDICAMENT FOR TREATING HUMAN TUMOR BASED ON ERF3A-TARGETING PROTEOLYSIS MECHANISM

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.

NOVEL ANTI-CD38 ANTIBODIES FOR THE TREATMENT OF CANCER

Resumen de: US2025216392A1

Antibodies, humanized antibodies, resurfaced antibodies, antibody fragments, derivatized antibodies, and conjugates of same with cytotoxic agents, which specifically bind to CD38, are capable of killing CD38′ cells by apoptosis, antibody-dependent cell-mediated cytotoxicity (ADCC), and/or complement-dependent cytotoxicity (CDC). Said antibodies and fragments thereof may be used in the treatment of tumors that express CD38 protein, such as multiple myeloma, chronic lymphocytic leukemia, chronic myelogenous leukemia, acute myelogenous leukemia, or acute lymphocytic leukemia, or the treatment of autoimmune and inflammatory diseases such as systemic lupus, rheumatoid arthritis, multiple sclerosis, erythematosus, and asthma. Said derivatized antibodies may be used in the diagnosis and imaging of tumors that express elevated levels of CD38. Also provided are cytotoxic conjugates comprising a cell binding agent and a cytotoxic agent, therapeutic compositions comprising the conjugate, methods for using the conjugates in the inhibition of cell growth and the treatment of disease, and a kit comprising the cytotoxic conjugate. In particular, the cell binding agent is a monoclonal antibody, and epitope-binding fragments thereof, that recognizes and binds the CD38 protein.

METHODS OF TREATING AML BY IDENTIFYING AND TARGETING A MYELOID/LYMPHOID LEUKEMIA STEM CELL

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.

METHODS FOR IDENTIFYING RESISTANCE OR RESPONSE TO VENETOCLAX/HYPOMETHYLATING AGENT/ANTI-CD70 AGENT TREATMENT FOR ACUTE MYELOID LEUKEMIA

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.

NATURAL KILLER CELL ENGAGERS

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.

ANTI-CLL1 SINGLE-DOMAIN ANTIBODY AND USE THEREOF

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.

CIRCULAR RNA ENCODING CARS AND THE USE THEREOF

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.

PHARMACEUTICAL COMPOSITION OF ANAPLASTIC LYMPHOMA KINASE INHIBITOR AND PREPARATION METHOD THEREFOR

Resumen de: WO2025140560A1

A pharmaceutical composition of a polycyclic anaplastic lymphoma kinase inhibitor, a preparation method therefor, and use thereof. In particular, the present disclosure relates to a pharmaceutical preparation of 5-chloro-N4-(2-(isopropylsulfonyl)phenyl)-N2-(7-methyl-8-(piperidin-4-yl)-2,3-dihydrobenzob1,4dioxin-5-yl)pyrimidine-2,4-diamine, a preparation method therefor, and use thereof.

COMPOSITION FOR AMPLIFYING FLT3 GENE, AND USES THEREOF

Resumen de: EP4578962A1

The present invention relates to a composition for amplifying a FLT3 gene, and uses thereof, and, more particularly, to a composition comprising a primer set capable of simultaneously amplifying an ITD detection region and a TKD mutation region of the FLT3 gene, and uses thereof. The composition for gene amplification, according to the present invention, enables the simultaneous performance of: diagnosis of acute myeloid leukemia (AML) in patients having FLT3-ITD mutations; determination of targeted anticancer treatment prescription for AML patients having FLT3-ITD mutations; detection of minimal residual disease (MRD) in AML patients; prognosis prediction in AML patients; and identification of drug resistance to AML tyrosine kinase inhibitors, and thus, shortens the time to derive analysis results from samples and enables efficient testing. The present invention enables the selection of correct and rapid diagnosis and treatment methods in the treatment of patients with acute myeloid leukemia, and thus is useful for early treatment and recurrence prevention.

METHODS OF TREATING CHRONIC MYELOID LEUKEMIA USING THE TYROSINE KINASE INHIBITOR VODOBATINIB

Resumen de: AU2023331249A1

The present invention relates to methods of treating leukemia using Tyrosine Kinase inhibitors. The invention particularly relates to methods of treating CML and ALL using a compound of Formula I or a pharmaceutically acceptable salt thereof. The compound of Formula 1 has been shown to be efficacious safe and tolerable at a dose from 10 mg to 210 mg.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

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.

COMBINATIONS OF IL15/IL15R ALPHA HETERODIMERIC FC-FUSION PROTEINS AND FCRH5XCD3 BISPECIFIC ANTIBODIES FOR THE TREATMENT OF BLOOD CANCERS

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.

REPURPOSING VARENICLINE AS AN ANTI-INFLAMMATORY AGENT WITH ITS SUPPRESSOR EFFECTS ON INFLAMMATORY CYTOKINES

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.

DOSING FOR COMBINATION TREATMENT WITH ANTI-CD20/ANTI-CD3 BISPECIFIC ANTIBODY AND ANTI-CD79B ANTIBODY DRUG CONJUGATE

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.

METHODS AND COMPOSITIONS FOR IDENTIFYING HOX GENE SIGNATURES TO ASSIGN SPECIFIC AND EFFECTIVE THERAPIES IN ACUTE MYELOID LEUKEMIA AND OTHER CANCERS

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.

METHODS OF TREATING MYELODYSPLASTIC SYNDROME AND MONITORING THE TREATMENT

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.

METHODS FOR DETECTING CELLULAR TRANSITIONS

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.

Pharmaceutical formulations of Bruton's tyrosine kinase inhibitor

Resumen de: AU2025204331A1

PHARMACEUTICAL FORMULATIONS OF BRUTON'S TYROSINE KINASE INHIBITOR Abstract Described herein are pharmaceutical formulations of Bruton's tyrosine kinase (Btk) inhibitor I-((R)-3-(4-amino-3-(4-phenoxyphenyl)-IH-pyrazolo 3,4-dpyrimidin- l-yl)piperidin-l-yl)prop-2-en-I-one. Also disclosed are methods of using the Btk inhibitor, alone or in combination with other therapeutic agents, for the treatment of autoimmune diseases or conditions, heteroimmune diseases or conditions, cancer, including lymphoma, and inflammatory diseases or conditions. PHARMACEUTICAL FORMULATIONS OF BRUTON'S TYROSINE KINASE INHIBITOR Abstract Formulation A Formulation B Formulation C Formulation D 0 6 12 18 24 Nominal Time Post-Dose (h) Described herein are pharmaceutical formulations of Bruton's tyrosine kinase (Btk) inhibitor I-((R)-3-(4-amino-3-(4-phenoxyphenyl)-IH-pyrazole 3,4-dpyrimidin- 1-yl)piperidin-l-yl)prop-2-en-I-one. Also disclosed are methods of using the Btk inhibitor, alone or in combination with other therapeutic agents, for the treatment of autoimmune diseases or conditions, heteroimmune diseases or conditions, cancer, including lymphoma, and inflammatory diseases or conditions. un ' u n b s t r a c t o r m u l a t i o n o r m u l a t i o n Mean Ibrutinib Plasma Concentration (ng/mL) o r m u l a t i o n o r m u l a t i o n o m i n a l i m e o s t - o s e ( h ) e s c r i b e d h e r e i n a r e p h a r m a c e u t i c a l f o r m u l a t i o n s o f r u t o n ' s t y r o s i n e k i n a s e (

DISEASE DETECTION SYSTEMS AND METHODS

Nº publicación: WO2025137368A1 26/06/2025

Solicitante:

CELLARITY INC [US]
CELLARITY, INC