Resumen de: WO2025226846A1
Disclosed are substituted purine derivatives of formulae (I), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods involving the inventive compounds or compositions for treating and/or preventing cell proliferative diseases including certain cancers of breast, brain, ovarian, lung, colorectal cancer, leukemias, lymphoma, melanoma, multiple myeloma, Ewing's sarcoma, osteosarcoma and inflammatory and myotonic dystrophy type 1 diseases in a mammal. Treatment of a subject with a proliferative disease using a compound or composition of the invention may inhibit the aberrant activity of kinases, such as a cyclin-dependent kinases (CDK) (e.g., CDK12/13), and degrade some other proteins (including cycK) and therefore, induce potent antiproliferative and apoptotic effects and/or inhibit transcription in the subject.
Resumen de: US2025333445A1
The invention provides a CD16a-binding polypeptide which comprises at least one motif that binds to CD16a, wherein said polypeptide comprises the following structure: N-terminal portion-Helix 1-Separating portion-Helix 2-C-terminal portion the CD16a-binding motif being the portion Helix 1-Separating portion-Helix 2. The invention further provides pharmaceutical compositions comprising the CD16a-binding polypeptide, and the use of the CD16a-binding polypeptide or pharmaceutical compositions as a medicament, particularly for use in the treatment or prophylaxis of cancers such as multiple myeloma.
Resumen de: US2025333502A1
Methods are provided to manipulate phagocytosis of cells, including hematopoietic cells, e.g. circulating hematopoietic cells, bone marrow cells, acute leukemia cells, etc.; and solid tumor cells. In some embodiments of the invention the circulating cells are hematopoietic stem cells, or hematopoietic progenitor cells, particularly in a transplantation context, where protection from phagocytosis is desirable. In other embodiments the circulating cells are leukemia cells, particularly acute myeloid leukemia (AML), where increased phagocytosis is desirable.
Resumen de: US2025333530A1
The present invention relates to methods of treating B-cell proliferative disorders, e.g., primary refractory or relapsed diffuse large B-cell lymphoma (DLBCL), by administering glofitamab in combination with gemcitabine and oxaliplatin. Further the invention related to an optimized corticosteroid prophylaxis for glofitamab resulting in lower incidence of cytokine release syndrome (CRS).
Resumen de: US2025333421A1
The present invention relates to pharmaceutical agents useful for therapy and/or prophylaxis in a mammal, pharmaceutical composition comprising such compounds, and their use as menin/MLL protein/protein interaction inhibitors, useful for treating diseases such as cancer, including but not limited to leukemia, myelodysplastic syndrome (MDS), and myeloproiiferative neoplasms (MPN); and diabetes.
Resumen de: US2025333795A1
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.
Resumen de: US2025334578A1
The present invention relates to antigen binding molecules that are at least bispecific and specifically bind to CD200 and CD123 on the cellular surface of leukemic stem cells (LSCs). The present invention further relates to a method for identifying such antigen binding molecules and the use of the antigen binding molecules for the diagnosis and treatment of leukemia, such as AML or CML, and in particular pediatric forms thereof.
Resumen de: WO2025223509A1
Disclosed in the present invention are a class of dienyl long-chain compounds, and a preparation method therefor and the use thereof. The structure is as shown in formula I, and the definition of each substituent in the formula is as described in the description and the claims. The compound of the present invention has an ACLY inhibitory activity and a lipid synthesis inhibitory activity, and can be used in the preparation of a drug for treating metabolic diseases such as hyperlipidemia and atherosclerosis, or a variety of cancers such as lung cancer, pancreatic cancer, breast cancer, ovarian cancer, liver cancer, intestinal cancer, brain cancer and acute myeloid leukemia.
Resumen de: US2025332150A1
T-cell acute lymphoblastic leukemias (T-ALL) are aggressive hematological malignancies associated with poor clinical outcome. TP53 alterations (TP53Alt) were rarely identified in T-ALL at diagnosis and their prognostic impact remains unclear. In a cohort of 476 adults and pediatric T-ALL, TP53Alt were observed in 4% of cases and were associated with chemoresistance and poor prognosis. APR-246, a small compound which restores wild-type configuration to mutated p53, showed efficacy in T-ALL harboring TP53 mutations. More importantly, in TP53 germline T-ALL, Notch 1 pathway gain of function mutations were associated with substantial sensitivity to APR-246. Mechanistically, Notch 1 activation via p53 downregulation and subsequent ferroptosis induction led to preferential APR-246 sensitivity. Given that Notch 1 pathway oncogenic activation is present in more than 70% of T-ALLs, these observations pave the way for promising perspectives in T-ALL treatment which could benefit from the Achilles heel associated with Notch 1 activation sensitizing leukemia cells to APR-246-induced ferroptosis, thus extending the use of APR-246 in T-ALL beyond TP53 alterations.
Resumen de: WO2025227007A1
Disclosed herein are 4,6-substituted-2-(3'-1,1'-biphenyl-4-yl)quinoline analogs and pharmaceutically acceptable salts thereof that are inhibitors of dihydroorotate dehydrogenase (DHODH) with properties, including stability and bioavailability as disclosed herein. Also disclosed herein are methods of making the analogs and salts thereof. The disclosed analogs and salts thereof can be used in the treatment of a variety of disorders and diseases in which inhibition of DHODH can be clinically useful, including cancer, such as a hematological cancer, including acute myeloid leukemia (AML); graft-versus-host-diseases; autoimmune disorders; and disorders associated with T-cell proliferation.
Resumen de: US2025332216A1
A method of treating classic Hodgkin's lymphoma (cHL) in a subject in need thereof, comprising intravenously administering to the subject i) a recombinant fusion protein at the dose of about 2.0 mg/kg body weight, once a week, and ii) an anti-PD-1 antibody at the dose of about 200 mg, once every 3 weeks, wherein the recombinant fusion protein comprises a mutated SIRPα D1 domain and a functional IgG1 heavy chain constant region, wherein the mutated SIRPα D1 domain comprises the amino acid sequence of SEQ ID NO: 2.
Resumen de: WO2025226831A1
Disclosed are purine derivatives of formulae (I), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods involving the inventive compounds or compositions for treating and/or preventing cell proliferative diseases including certain cancers of breast, brain, ovarian, lung, colorectal cancer, leukemias, lymphoma, melanoma, multiple myeloma, Ewing's sarcoma, osteosarcoma and inflammatory and myotonic dystrophy type 1 diseases in a mammal. Treatment of a subject with a proliferative disease using a compound or composition of the invention may inhibit the aberrant activity of kinases, such as a cyclin-dependent kinases (CDK) (e.g., CDK12/13), and therefore, induce potent antiproliferative and apoptotic effects and/or inhibit transcription in the subject.
Resumen de: WO2025223394A1
The present application relates to a pharmaceutical composition comprising i) a recombinant fusion protein and ii) an anti-PD-1 antibody, wherein the recombinant fusion protein comprises a mutated SIRPα D1 domain linked to a functional IgG1 heavy chain constant region, wherein the mutated SIRPα D1 domain comprises the amino acid sequence of SEQ ID NO: 2. The present application also relates to use of the pharmaceutical composition in preparation of a medicament for treating classic Hodgkin's lymphoma (cHL), e.g., relapsed or refractory cHL.
Resumen de: WO2024137713A1
The present disclosure relates to the use of pelabresib, and pharmaceutically acceptable salts and hydrates thereof, for treating lower risk myelodysplastic syndrome (LR-MDS) and conditions associated therewith.
Resumen de: WO2025218293A1
The present invention relates to a hetero (aromatic) ring-substituted cyclic diamine compound, the preparation thereof and the use thereof in the preparation of a drug for treating and/or preventing tumors. The structural formula of the compound is as shown in (I). The compound has a significant inhibitory effect on the growth of cells of tumors such as leukemia, lymphoma, breast cancer, melanoma, ovarian cancer, colorectal cancer, cervical cancer, lung cancer, prostate cancer, esophageal cancer, glioma, kidney cancer, nasopharyngeal carcinoma, liver cancer, gastric cancer and pancreatic cancer. Compared with the prior art, the compound of the present invention has a broad-spectrum anti-tumor activity, has a good effect on inhibiting tumors, and has an effect of degrading PD-L1 proteins, and is thus a PD-L1 immunomodulator.
Resumen de: WO2025217743A1
Systemic lupus erythematosus (SLE) is an autoimmune disease with multisystem involvement and is associated with significant morbidity and mortality. The diagnosis of SLE is challenging because signs and symptoms vary considerably from person to person, may change over time, and may overlap with those of many other disorders. There is thus a need for better tools and assays for the diagnosis and assessment of SLE. The present application discloses novel methods for the diagnosis and follow-up of SLE based on the detection of the levels of Casitas B-lineage lymphoma (CBL) and/or CBL-B in T lymphocytes from a subject, as well as novel methods for treating a subject suffering from SLE or preventing the development of SLE in an at-risk subject through the administration of an agent that increases the expression or activity of CBL and/or CBL-B in T lymphocytes from the subject, and/or the depletion of ICOS+ T cells.
Resumen de: US2025326856A1
Novel chimeric and/or humanized forms of the anti-CD45 BC8 antibody are described. The disclosed chimeric or humanized antibodies can be used as research, diagnostic, or therapeutic tools against CD45-related disorders, such as hematologic malignancies including acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), other myeloid and lymphoid disorders, other cancers, as well as non-malignant disorders, such as autoimmune disorders, infections, inherited blood disorders, and metabolic disorders.
Resumen de: US2025325644A1
The invention features immunogenic compositions containing anaplastic lymphoma kinase (ALK) polypeptides and methods of use thereof. The immunogenic compositions and methods of the invention may be used to treat a disease associated with ALK in a subject, such as cancer (e.g., a solid tumor cancer or an ALK+ cancer).
Resumen de: US2025324955A1
Genetically modified zebrafish, in which mutation combinations frequently identified in human AML are stably expressed in the stem cell population of the fish, are provided. The combination of mutations result in morphologic, cytochemical and molecular changes of its blood cells that are remarkably similar to those in human AML. The zebrafish model provides a foundation for the study of AML initiation and progression and a high throughput in vivo drug screening platform to identify personalized therapies for AML based on specific mutation combinations. The method of drug screening includes contacting embryos or adult fish containing mutations as disclosed herein, with a test agent, at test concentrations and test intervals to determine the therapeutic effect if any, of the test agent.
Resumen de: AU2025202444A1
TTC-018 USE OF ANTI-HA-1 AND ANTI-HA-2 BINDING PROTEINS FOR TREATMENT OF AML, ALL, AND MDS The present disclosure encompasses, among other things, methods and compositions for use in the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic disorder (MDS) in subjects that received HCT. The present disclosure relates, at least in part, to T cells engineered to express particular T Cell Receptors (TCRs) and their use in the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic disorder (MDS) in subjects that received HCT. TTC-018 USE OF ANTI-HA-1 AND ANTI-HA-2 BINDING PROTEINS FOR TREATMENT OF AML, ALL, AND MDS The present disclosure encompasses, among other things, methods and compositions for use in the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic disorder (MDS) in subjects that received HCT. The present disclosure relates, at least in part, to T cells engineered to express particular T Cell Receptors (TCRs) and their use in the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic disorder (MDS) in subjects that received HCT. pr - - - - - , , p r h e p r e s e n t d i s c l o s u r e e n c o m p a s s e s , a m o n g o t h e r t h i n g s , m e t h o d s a n d c o m p o s i t i o n s f o r u s e i n t h e t r e a t m e n t o f a c u t e m y e l o i d l e u k e m i a ( ) , a c u t e l y m p h o c y t i
Resumen de: AU2024238830A1
The present disclosed invention provides pharmaceutical compositions and methods of use for bisbenzylisoquinolines such as 6,6',7,12-tetramethoxy-2,2'-dimethyl-berbaman, and analogs, derivatives, isomers, and modified forms such as crystalline, salt forms, or a salt of this compound with a pharmaceutically acceptable acid or in combination with other agents to treat acute, chronic and pre-leukemic conditions as well as lymphomas and solid tumors. These include preneoplastic and neoplastic diseases and solid tumors including but not limited to acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), atypical chronic myeloid leukemia (aCML), and acute myeloid leukemia (AML), polycythemia vera (PV), chronic lymphoblastic leukemia (CLL), myeloproliferative syndrome (MPS), myelodysplastic syndrome (MDS), myeloproliferative neoplasms (MPN), myelofibrosis (MF), and polycythemia vera (PV).
Resumen de: EP4635981A1
The present invention relates to an anti-CLL1 single-domain antibody and use thereof, specifically, to a single-domain antibody having an amino acid sequence of SEQ ID No. 1. The single-domain antibody has high affinity and can specifically target CLL1-positive cells, and can be applied to the detection of CLL1 expression in bone marrow cells of acute myeloid leukemia (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 in the preparation of CLL1-targeting chimeric antigen receptor (CAR) cells or diagnostic kits for CLL1 protein detection and the like. The single-domain antibody drug has a stable structure, small molecular size, ease of recombinant expression, and low production cost. It can be used alone or employed as a drug delivery system to carry related drugs, which has broad prospects and important significance in the fields of pharmaceutical application, clinical diagnosis, and related fields.
Resumen de: US2025320225A1
The present disclosure provides methods of inhibiting the interaction of menin with MLL1, MLL2 and MLL-fusion oncoproteins with compositions of Formula (II-A). The methods are useful for the treatment of leukemia, solid cancers, diabetes and other diseases dependent on activity of MLL1, MLL2, MLL fusion proteins, and/or menin. Compositions of Formula (II-A) for use in these methods are also provided.
Resumen de: US2025320229A1
The present disclosure provides methods of inhibiting the interaction of menin with MLL1, MLL2 and MLL-fusion oncoproteins. The methods are useful for the treatment of leukemia, solid cancers, diabetes and other diseases dependent on activity of MLL1, MLL2, MLL fusion proteins, and/or menin. Compositions for use in these methods are also provided.
Nº publicación: US2025320298A1 16/10/2025
Solicitante:
GENENTECH INC [US]
HOFFMANN LA ROCHE INC [US]
Genentech, Inc,
Hoffmann-La Roche Inc
Resumen de: US2025320298A1
The invention provides methods of dosing for the treatment of cancers, such as multiple myelomas, with anti-fragment crystallizable receptor-like 5 (FcRH5)/anti-cluster of differentiation 3 (CD3) bispecific antibodies.