Resumen de: US2025235454A1
A compound derived from an aliphatic diamine including at least one pyridine or pyrimidine unit, for use as an anticancer agent, to a therapeutic composition including this compound, to a product including such a compound and another active agent, as well as to such a compound.
Resumen de: US2025236889A1
The present disclosure provides methods for genetically modifying lymphocytes and methods for performing adoptive cellular therapy that include transducing T cells and/or NK cells. The methods can include inhibitory RNA molecule(s) and/or engineered signaling polypeptides that can include a lymphoproliferative element, and/or a chimeric antigen receptor (CAR), for example a microenvironment restricted biologic CAR (MRB-CAR). Additional elements of such engineered signaling polypeptides are provided herein, such as those that drive proliferation and regulatory elements therefor, as well as replication incompetent recombinant retroviral particles and packaging cell lines and methods of making the same. Numerous elements and methods for regulating transduced and/or genetically modified T cells and/or NK cells are provided, such as, for example, those including riboswitches, MRB-CARS, recognition domains, and/or pH-modulating agents.
Resumen de: US2025235535A1
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: AU2024208091A1
This application pertains to the use of compounds, e.g., Compound A: or a pharmaceutically acceptable salt thereof, for the treatment of various diseases or disorders, including, for example, diffuse large B-cell lymphoma (DLBCL) and angioimmunoblastic T- cell lymphoma.
Resumen de: US2025235465A1
Methods and corresponding uses are provided for treating disease in a patient, involving the use of sodium trans-tetrachloridobis(1H-indazole)ruthenate(III) so as to ameliorate drug resistance, including resistance to proteasome inhibitors and immunomodulatory imide drugs. The disease may for example be relapsing/refractory multiple myeloma.
Resumen de: AU2024228130A1
The present invention relates to a nucleic acid encoding a recombinase capable of recombining asymmetric target sequences of SEQ ID NO:1 within the long terminal repeat of proviral DNA of a plurality of HTLV-1 strains, the recombinase having specific amino acid exchanges compared to the sequence of cre recombinase. The invention also provides expression vectors comprising said nucleic acids and cells comprising the same, the recombinase in protein form and pharmaceutical compositions comprising them. A method for provision of such recombinases is also provided. The invention also provides a method of treating a HTLV-1-infected subject, wherein the subject optionally has adult T-cell leukemia (ATL) and/or HTLV-1-associated myelopathy (HAM).
Resumen de: AU2023390460A1
The present disclosure provides methods of administering belumosudil to patients with multiple myeloma.
Resumen de: AU2025205154A1
CRYSTALLINE FORMS OF A BRUTON'S TYROSINE KINASE INHIBITOR Described herein is the Bruton's tyrosine kinase (Btk) inhibitor 1-((R)-3-(4-amino-3-(4- phenoxyphenyl)-1H-pyrazolo3,4-dpyrimidin-1-yl)piperidin-1-yl)prop-2-en-1-one, including crystalline forms, solvates and pharmaceutically acceptable salts thereof. Also disclosed are pharmaceutical compositions that include the Btk inhibitor, as well as methods of using the Btk in hibitor, 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. CRYSTALLINE FORMS OF A BRUTON'S TYROSINE KINASE INHIBITOR Described herein is the Bruton's tyrosine kinase (Btk) inhibitor 1-(R)-3-(4-amino-3-(4- phenoxyphenyl)-1H-pyrazolo3,4-dpyrimidin-1-yl)piperidin-1-yl)prop-2-en-1-one,1 including crystalline forms, solvates and pharmaceutically acceptable salts thereof. Also disclosed are pharmaceutical compositions that include the Btk inhibitor, as well as methods of using the Btk in hibitor, 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. ul ' u l e s c r i b e d h e r e i n i s t h e r u t o n ' s t y r o s i n e k i n a s e ( t k ) i n h i b i t o r - ( ) - - ( - a m i n o - - ( - p h e n o x y p h e n y l ) - - p y r a z o l
Resumen de: WO2025155775A1
Methods of detecting leukemia stem cells are provided herein. Further provided are methods of diagnosing and treating AML as well as minimal residual disease or medium- or high-risk AML.
Resumen de: WO2025153509A1
A first aspect of the invention relates to a variant prime editing polypeptide composed of a SpCas9 nickase fused to an engineered Moloney Murine leukaemia virus (M-MLV) reverse transcriptase, characterized by a mutation, relative to a reference sequence SEQ ID NO 001, selected from the group consisting of A277D; K1865T; and S237A. Another aspect of the invention relates to a combination of a first and second split PE variant polypeptide comprising split intein component at its C- and N-terminus, respectively, which, when both are present within a target cell, are capable of forming a fusion polypeptide comprising a functional prime editing polypeptide according to the first aspect of the invention. Other aspects of the invention relate to a combination medicaments and nucleic acid sequences facilitating the PE variant of the invention.
Resumen de: WO2025155780A1
Disclosed herein are novel compounds that are Mpsl/TTK inhibitors. Also disclosed herein are compositions comprising the compounds and methods of using the compounds in treating various diseases in a patient. In some embodiments, some such compounds, compositions, and their uses may be useful for the treatment of cancer. In some implementations, the cancer is brain cancer, glioblastoma multiforme, head and neck cancers, colorectal cancer, stomach or gastric cancer, pancreatic cancer, melanoma, bladder cancer, kidney cancer, renal cell carcinoma, breast cancer, ovarian cancer, lymphoma, thyroid cancer, mesothelioma, sarcoma, lung cancer, non-small cell lung cancer, small cell lung cancer, or endometrial cancer.
Resumen de: WO2025155777A1
Disclosed herein are novel compounds that are Mpsl/TTK inhibitors. Also disclosed are compositions comprising the compounds and methods of using the compounds in treating various diseases. In some embodiments, some such compounds, compositions, and their uses may be useful for the treatment of cancer. In some implementations, the cancer is brain cancer, glioblastoma multiforme, head and neck cancers, colorectal cancer, stomach or gastric cancer, pancreatic cancer, melanoma, bladder cancer, kidney cancer, renal cell carcinoma, breast cancer, ovarian cancer, lymphoma, thyroid cancer, mesothelioma, sarcoma, lung cancer, non-small cell lung cancer, small cell lung cancer, or endometrial cancer.
Resumen de: US2025236642A1
As a technique that can be used for cancer immunotherapy of adult T-cell leukemia (ATL), provided is a pharmaceutical composition for treatment or prevention of adult T-cell leukemia, the pharmaceutical composition containing a peptide consisting of an amino acid sequence of any one of SEQ ID NOs: 1 to 68.
Resumen de: US2025236621A1
The present disclosure relates to compounds and compositions for inhibition of Bcr-Abl tyrosine kinases, methods of preparing said compounds and compositions, and their use in the treatment of various cancers, such as chronic myeloid leukemia (CML).
Resumen de: US2025236600A1
The present invention is comprised in the field of medicinal chemistry, and it is related to substituted vinyl piperazine-piperidine urea compound which are well effective as antitumoral agents. In particular, they are suitable in methods of treatment of glioblastoma (GB), multiple myeloma (MM) or pancreatic cancer (PC).
Resumen de: US2025236607A1
Described herein are CDK9 degraders that include a CDK9 binding moiety, such as AT7519 or VIP152, conjugated to a E3 ubiquitin ligase binding moiety, such as thalidomide, lenalidomide, or pomalidomide. These degraders can induce the ubiquitination of CDK9 and promote its degradation in cells. The linker covalently tethering the CDK9 binding moiety to the E3 ubiquitin ligase binding moiety can be selected to tune the solubility profile and potency of the degrader. Accordingly, the present disclosure provides compounds, compositions, kits, uses, and methods for the treatment of cancer (e.g., blood cancers such as acute myeloid leukemia or acute lymphoblastic leukemia).
Resumen de: EP4588492A2
Compositions and methods are described for stabilizing a radio-iodinated monoclonal IgG antibody for up to 17 days against radiolytic decomposition. The stabilized radiolabeled murine antibody binding the CD45 antigen expressed on various forms of lymphomas is useful as a radio-therapeutic and diagnostic agent in the treatment of human malignancies of hematopoietic origin, including lymphomas.
Resumen de: EP4588484A2
The present disclosure provides methods for treating multiple myeloma (such as refractory multiple myeloma or relapsed and refractory multiple myeloma) in an individual who received at least two prior therapies for multiple myeloma. The methods comprise administering to the individual an anti-CD38 antibody, pomalidomide, and dexamethasone. Also provided are methods of improving renal impairment in an individual having multiple myeloma.
Resumen de: US2025228881A1
A novel multi-fused-ring compound and a composition for preventing or treating multiple myeloma comprising the same as an active ingredient is described herein. The compound is a derivative synthesized from the natural compound ginsenoside as a starting material, and shows no cytotoxicity to normal hematopoietic stem cells, while exhibiting a significant killing effect on various multiple myeloma cell lines, indicating that it may be administered for a long period of time without side effects. In addition, the compound exhibits a significant synergistic effect when co-administered with the immunomodulator thalidomide or its analog lenalidomide, and thus may be useful as an efficient therapeutic agent or therapeutic aid agent composition for multiple myeloma, an incurable disease.
Resumen de: US2025230202A1
The present disclosure provides use of a promyelocytic leukemia 1 (PML-1) protein in preparation of a drug for inhibiting a cytokine storm, and belongs to the technical field of biomedicine. The present disclosure further provides use of a PML-1 protein and/or a product expressing the PML-1 protein in preparation of a drug for inhibiting a cytokine storm. In the present disclosure, the PML-1 protein and/or the product expressing the PML-1 protein can significantly inhibit the expression of proteins TAB1, TAK1, and p-TAK1 and inhibit inflammatory cytokines TNF-α, IL-1β, MIP-1α, IL-6, IL-8, and MCP-1 in an inflammatory signaling pathway, thereby inhibiting the cytokine storm. The PML-1 protein can be directly used for the treatment of inflammations and related diseases. Compared with traditional chemical anti-inflammatory drugs, the protein shows more significant curative effect, stronger specificity, lower toxicity, smaller side effects, and clearer biological functions, exhibiting broad application prospects.
Resumen de: US2025230234A1
The present disclosure provides antigen-binding proteins specifically binding GPRC5D, as well as respective antibodies in enhanced ADCC formats, and methods of using them to treat cancers such as multiple myeloma.
Resumen de: US2025231193A1
T-cell malignancies are a broad, heterogenous group of diseases and include T-cell lymphomas and T-cell leukemias. T-cell lymphomas are a heterogeneous group of malignancies involving T lymphocytes and generally characterized by a poor prognosis. Among them, cutaneous T-cell lymphomas involve primarily the skin. Mycosis fungoides and Sézary syndrome are the most frequent cutaneous T-cell lymphomas. The inventors showed that both circulating malignant and non-malignant T cells express CD51 in patients with Sézary syndrome. CD51 therefore appears as a useful diagnostic, prognostic and follow-up marker, and as a potential therapeutic target in T-cell lymphomas.
Resumen de: US2025228783A1
Described herein are pharmaceutical formulations of Bruton's tyrosine kinase (Btk) inhibitor 1-((R)-3-(4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo 3,4-dpyrimidin-1-yl)piperidin-1-yl)prop-2-en-1-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.
Resumen de: US2025230252A1
Methods of treating acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) are provided, as are compositions and combinations suitable for use in said methods.
Nº publicación: US2025228918A1 17/07/2025
Solicitante:
YALE UNIV [US]
Yale University
Resumen de: US2025228918A1
Described herein are a method of increasing energy expenditure level in a subject, a method of reducing body weight in a subject, a method of decreasing the amount of white adipose (WAT) tissue and/or promoting browning of WAT in a subject, and/or a method of improving glucose tolerance and/or insulin sensitivity in a subject. Each of the methods includes downregulating the level and/or activity of Augmentor α (Augα), or downregulating the level and/or activity of anaplastic lymphoma kinase (ALK) in the subject. Also described herein are a method of decreasing energy expenditure level in a subject, and/or a method of increasing body weight in a subject. Each of the methods includes upregulating the level and/or activity of Augα, or upregulating the level and/or activity of ALK in the subject.