Alerta

METTL3 INHIBITOR

Resumen de: EP4800016A1

The present invention discloses a METTL3 inhibitor represented by general formula (I), its pharmaceutical composition, preparation method, and the use thereof in the preparation of drugs, prevention and/or treatment of drugs for indications associated with METTL3. The compound of the present invention is an ideal high-activity METTL3 inhibitor, which can be used for the treatment and/or prevention of diseases, including AML, myeloid leukemia, solid tumors such as hepatocellular carcinoma, colorectal cancer, and prostate cancer.

USE OF HYPOXIA-INDUCIBLE FACTOR-PROLYL HYDROXYLASE INHIBITOR (HIF-PHI) IN RARE ANEMIA

Resumen de: EP4799642A1

0001 The present application relates to use of a hypoxia-inducible factor-prolyl hydroxylase inhibitors (HIF-PHI) in rare anemia. Specifically disclosed in the present application is use of certain hypoxia-inducible factor-prolyl hydroxylase inhibitors (HIF-PHIs) in the treatment of anemia of myelodysplastic syndromes (MDS anemia), beta-thalassemia (β-thalassemia), and/or sickle cell disease (sickle cell anemia, SCD anemia).

TREATMENT OF MYELOID DISORDERS AND ACUTE LEUKEMIAS TARGETING NOVEL TUMOR SPECIFIC ANTIGENS

Resumen de: US20260250409A1

0000 The present disclosure relates to a method of diagnosing or treating myeloid disorders and acute leukemias by using a tumor specific antigen selected from CD63, CD151, CD72, CD84, CD69, and CD109. Further provided are an antigen binding protein (ABP), an ABP-drug conjugate, and a CAR targeting the tumor specific antigen, and methods for their use.

INHIBITION OF APELIN SIGNALING TO TREAT ACUTE MYELOID LEUKEMIA

Resumen de: WO2026178316A1

Disclosed herein are methods of inhibiting apelin, including but not limited to blocking the apelin ligand and/or the apelin receptors and/or signaling thereof, to prevent or reduce endothelial cell clonal expansion and transcriptional remodeling which promotes hematopoietic disorder progression, including but not limited to acute myeloid leukemia (AML).

TREATMENT OF HEMATOLOGICAL MALIGNANCIES WITH ANTIBODIES INHIBITING GALECTIN-9

Resumen de: US20260250394A1

0000 Disclosed herein are methods for treating a hematologic malignancy (e.g., acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or acute lymphoblastic leukemia (ALL)), using an anti-Galectin-9 antibody (e.g., G9.2-17(IgG4). In some embodiments, the anti-Galectin-9 antibody is used as a monotherapy.

A Vaccine Composition of Cells Expressing a Lentiviral Vector and Methods of Using

Resumen de: US20260250717A1

0000 A vector construct is described that is a lentiviral construct including DNA encoding for GM-CSF. A vaccine composition is also described that is includes K562 cells transfected with this vector construct, and also possibly including the U266 and H929. Methods are described for using the vaccine composition in methods of immunizing against plasma cell disorders, including multiple myeloma and related disorders.

METHOD AND DRUG FOR TREATING CANCER BY JOINT USE OF NKG2D CHIMERIC ANTIGEN RECEPTOR AND PD1 INHIBITOR

Resumen de: US20260248853A1

0000 The present disclosure provides a novel chimeric antigen receptor and a combination thereof with a PD1 inhibitor. Further provided are expression vectors and host cells expressing the chimeric antigen receptor and the composition of said receptor with the PD1 inhibitor. Further provided are uses of the chimeric antigen receptor and the composition of said receptor with the PD1 inhibitor in treating cancer or preparing a drug for treating cancer. The drug and the method provided by the present invention can effectively treat cancer, particularly myeloma and solid tumors.

Pharmaceutical formulations of Bruton's tyrosine kinase inhibitor

Resumen de: AU2026214088A1

#48183531_1 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 Mean Ibrutinib Plasma Concentration (ng/mL) Formulation C Formulation D 0 6 12 18 24 Nominal Time Post-Dose (h) ug ' u g 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)

Substituted straight chain spiro derivatives

Resumen de: AU2026205213A1

The present invention relates to pharmaceutical agents useful for therapy and/or prophylaxis in 5 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 myeloproliferative neoplasms (MPN); and diabetes. ul u l

Compounds

Resumen de: GB2704140A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein X-Y is -NHSO2-, v is 1 or 2; A is selected from C4-C8-cycloalkyl, fused or bridged bicyclic C6-C12- cycloalkyl, or C4-C8-cycloalkyl fused to an aryl or heteroaryl group, each optionally substituted by one or more R4 groups. R1 is haloalkyl or OR3; R2 is H or halo; R3 is alkyl or benzyl; and each R4 is independently alkyl or halo. R5a is selected from COOH, CONR8R9, CONHSO2R10 and heteroaryl; R5b is H, alkyl or halo. R6a and R6b are each independently H or alkyl, or together form a CO group with the carbon atom to which they are attached. R7a and R7b are each independently H or alkyl, may together form a cycloalkyl group, or R7a is H and R7b and R5b together form a 1- or 2-carbon alkylene bridge. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include (R)-4-(N-(2-(cyclohexylamino)-5-(trifluoromethyl)phenyl)sulfamoyl)morpholine-2-carboxylic acid Formula (I)

Compounds

Resumen de: GB2704114A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein X-Y is -NHSO₂-. A is selected from C₄-C₈ cycloalkyl, bicyclic C₅-C₁₂ cycloalkyl, or a C₄-C₈ cycloalkyl fused to an aryl or heteroaryl group, each optionally substituted by one or more R₄ groups. R₁ is haloalkyl or OR₃; R₂ is H or halo; R₃ is alkyl or benzyl; and each R₄ is independently alkyl or halo. B is an azetidine group substituted by (R₅)ₘ, wherein R₅ is independently selected from COOH, (CR₁ₐR1b)ₐNR₆R₇, alkyl, haloalkyl, halo, alkoxy, hydroxyalkyl, cycloalkyl, heteroaryl or heterocycloalkyl, optionally substituted. R1a and R1b are independently H, OH, alkyl or hydroxyalkyl; a is 0-3; R₆ and R₇ are independently H, alkyl, SO-alkyl, haloalkyl or heterocycloalkyl; and m is 0-6. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions comprising such cells are also disclosed. Exemplified compounds include N-(2-(cyclohexylamino)-4-fluoro-5-(trifluoromethyl)phenyl)-3-(pyridin-4-yl)azetidine-1-sulfonamide. Formula (I)

Compounds

Resumen de: GB2704272A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein X-Y is -NHSO2-, A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; B is NR6R7, wherein R6 and R7 together with the nitrogen to which they are attached form a 5 to 8-membered heterocycloalkyl group containing at least one further group selected from NR38, CO, SO2 and SONH, optionally wherein two non-adjacent carbons in the heterocycloalkyl group are linked by a one or two carbon alkylene bridge or an ether bridge, and wherein the said hetrocycloalkyl group is optionally substituted by one or more groups selected from R5 and R35, R5 is selected from COOH, CONR8R9, CONHSO2R10, tetrazolyl and hydroxyalkyl; R38 is selected from H, alkyl and CO-alkyl. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include 4-(N-(2-(cyclohexylamino)-5-(trifluoromethyl)phenyl)sulfamoyl)-1-methylpiperazine-2-carboxylic acid Formula (I)

Compounds

Resumen de: GB2704197A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein X-Y is -NHSO2-, A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; B is NR6R7, wherein R6 and R7 together with the nitrogen to which they are attached form a 5 to 7-membered heterocycloalkyl group optionally containing one or more further groups selected from S, SO and SO2, optionally wherein two non-adjacent carbons in the heterocycloalkyl group are linked by a one or two carbon alkylene bridge, and wherein the said hetrocycloalkyl group is substituted by one or more R5 groups, and is optionally further substituted by one or more R35 groups; R5 is selected from (CR1aR1b)aCOOH, CONR8R9, CONHSO2R10, SO(N=H)-R10 and tetrazolyl. Rxx groups are as defined herein. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include (S)-1-(N-(2-(cyclohexylamino)-5-(trifluoromethyl)phenyl)sulfamoyl)piperidine-3-carboxylic acid. Formula (I)

Compounds

Resumen de: GB2704195A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein X-Y is -NHSO2-, A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; B is NR6R7, wherein R6 and R7 together with the nitrogen to which they are attached form a 5-, 6- or 7-membered heterocycloalkyl group that is substituted by one or more R5 groups, and is optionally further substituted by one or more R13 groups, optionally wherein two non-adjacent carbons in the heterocycloalkyl group are linked by a one or two carbon alkylene bridge; each R5 is independently selected from (CR1aR1b)aNR8R9, OR10, NR8CO2R11 and NR8COR12. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include (R)(N-(2-(cyclohexylamino)-4-fluoro-5-(trifluoromethyl)phenyl)-3-hydroxypiperidine-1-sulfonamide Formula (I)

Compounds

Resumen de: GB2704145A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein R groups are as defined herein, X-Y is -NHSO2-, A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; B is a fused bicyclic group selected from the hetero groups as defined herein, the group is further substituted by zero or more R5 groups; each R5 is independently selected from COOH, CONR8R9, CONHSO2R10, SO2R35, NR36R37, alkyl, OH, halo, haloalkyl, alkoxy, and hydroxyalkyl. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include N-(2-(cyclohexylamino)-4-fluoro-5-(trifluoromethyl)phenyl)-5,6-dihydro-1,2,4triazolo1,5-apyrazine-7(8H)-sulfonamide No figure

Compounds

Resumen de: GB2704209A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein R groups are as defined herein, X-Y is -NHSO2- or -SO2NH; A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; R1 is haloalkyl or OR3; R2 is H or halo; R3 is alkyl or benzyl; and each R4 is independently alkyl or halo. R5 is selected from CO2H, CONHSO2R10, CONR11R12 and tetrazolyl. R6 and R7 are independently H, alkoxy or halo. R8 is selected from halo, alkoxy, haloalkoxy, alkyl, haloalkyl, (CH2)k-cycloalkyl, hydroxyalkyl, CN, OH, SOR9, SO2R9 and heteroaryl. R9 is alkyl, R10 is alkyl, cycloalkyl, aryl or heteroaryl optionally substituted, R11 is H or alkyl, R12 is H, CN, alkyl or CONH2, and k is 0, 1, 2 or 3. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include 3-(N-(2-(cyclobutylamino)-5-(trifluoromethyl)phenyl)sulfamoyl)-4-methylbenzoic acid. No Figure

Compounds

Resumen de: GB2704162A

A compound of formula (I), or a pharmaceutically acceptable salt or hydrate thereof, is provided, wherein R groups are as defined herein, X-Y is -NHSO2-, A is selected from: a C4-C8-cycloalkyl group; a bicyclic C5-C12-cycloalkyl group; and a C4-C8-cycloalkyl group fused to an aryl or heteroaryl group; each of which is optionally substituted by one or more R4 groups; R1 is haloalkyl or OR3; R2 is H or halo; R3 is alkyl or benzyl; and each R4 is independently alkyl or halo. B is NR6R7, wherein R6 and R7, together with the nitrogen to which they are attached, form a heterocycloalkyl group, wherein said heterocycloalkyl group is fused to at least one saturated cyclic group to form a fused polycyclic group wherein one or more carbons in the fused polycyclic group is optionally replaced by a group indepependetly selected from NR10, CO, N, O, S, SO and SO2, and is optionally further substituted by one or more R5 groups; each R5 is independently selected from NR8R9, OH, alkyl, halo, haloalkyl, alkoxy, and hydroxyalkyl. The compounds modulate ERAP2 and are useful in treating proliferative, viral, immune or inflammatory disorders, particularly cancer or leukaemia. The compounds stimulate a neo-antigen directed immune response. Methods of producing antigen-presenting cells and immunogenic compositions are also disclosed. Exemplified compounds include N-(2-(cyclohexylamino)-4-fluoro-5-(trifluoromethyl)phenyl)-2-methyl-3-oxooctahydropyrrolo3,4-cpyridine-5-sulfonamide. No Figure

Methods of Treating High Risk Multiple Myeloma

Resumen de: US20260242503A1

Disclosed are methods of treating a subject having high-risk multiple myeloma, methods of achieving negative minimal residual disease status in a subject having multiple myeloma, and methods of predicting a likelihood of, or decreasing a risk of, relapse and/or disease progression in a subject having multiple myeloma.

Bispecific anti-CD28 x anti-CD22 antibodies and uses thereof

Resumen de: AU2026210818A1

The present invention provides bispecific antigen-binding molecules comprising a first antigen-binding domain that specifically binds human CD28, and a second antigen- binding molecule that specifically binds human CD-22. In certain embodiments, the bispecific antigen- binding molecules of the present invention are capable of inhibiting the growth of tumors expressing CD-22, such as B-cell lymphomas. The antibodies and bispecific antigen-binding molecules of the invention are useful for the treatment of diseases and disorders in which an up-regulated or induced targeted immune response is desired and/or therapeutically beneficial. ul u l

DENOSUMAB FORMULATION

Resumen de: US20260242496A1

0000 An aqueous pharmaceutical formulation having improved stability includes denosumab and a poloxamer, and preferably a histidine buffer and/or sugar or sugar alcohol. The formulation is for use in treating or preventing osteoporosis, loss of bone mass, skeletal-related events associated with multiple myeloma, solid tumor bone metastases, giant cell tumors of the bone or hypercalcemia.

USE OF ANTI-CD20 ANTIBODY DRUG CONJUGATE IN PREPARATION OF DRUG FOR TREATING NON-HODGKIN LYMPHOMA

Resumen de: US20260241027A1

0000 The present disclosure discloses a use of anti-CD20 ADC in the preparation of a drug for treating NHL, and the drug has excellent clinical efficacy and safety in treating a R/R NHL patient after receiving at least two standard treatments.

CRISPR-RELATED METHODS AND COMPOSITIONS TARGETING FL1-1 EXPRESSION

Resumen de: US20260241033A1

The present disclosure is directed to CRISPR-related systems and components for targeting, editing, and/or modulating expression of a FLI-1 (Friend virus leukemia integration 1 transcription factor; Fli-1 Proto-Oncogene, ETS Transcription Factor) gene. The present disclosure also relates to methods and applications thereof in connection with engineered cells including T cells or T cell precursors.

ANTIMALARIAL ENDOPEROXIDE DERIVATIVES FOR THE TREATMENT OF MYELODYPLASTIC SYNDROME

Resumen de: US20260242391A1

Disclosed herein are methods for using an antimalarial endoperoxide compound, such as artemisinin, intreating a subject suffering from myelodysplastic syndromes (MDS), and in slowing or preventing the progression of MDS in the subject to development of acute myeloid leukemia (AML).

OTUD7A INHIBITOR COMPOUNDS AND METHODS

Resumen de: WO2026174182A1

Pyrazolopyrimidines and imidazopyrazine compounds of Formulas (I) and (II) are provided. The compounds can be used to target OTU domain-containing protein 7A (OTUD7A), e.g., to block and/or reduce OTUD7A-mediated deubiquitination of Ewing sarcoma breakpoint region 1-Friend leukemia virus integration 1 transcription factor (EWS-FLI1). Methods of treating conditions dependent on FLI1, including Ewing sarcoma, leukemia, and other cancers, by administering the compounds, are also described.

SUPER RESOLUTION MICROSCOPIC IMAGING OF T LYMPHOCYTE CLONALITY COMBINING MULTIPLEXED SEQUENTIAL FLUORESCENCE IN SITU HYBRIDIZATION WITH BULK GENOMIC DNA SEQUENCING

Nº publicación: US20260242870A1 20/08/2026

Solicitante:

CALIFORNIA INST OF TECHN [US]
CALIFORNIA INSTITUTE OF TECHNOLOGY

Resumen de: US20260242870A1

This disclosure provides methods for imaging of samples for diagnosis of lymphoid neoplasms, lymphocytic autoimmune disease, and other conditions related to clonal expansion of lymphocytes. Using these methods, individual lymphocytes can be imaged in situ to determine tissue-wide clonality based on rearrangement of T cell receptor gene segments. Post hoc bulk sequencing allows for complementarity determining sequences to be mapped back to individual lymphocyte clones in super resolution images. In additional to its clinical applications in histopathology, these methods are also useful for the study of cellular and immunologic processes related to lymphoproliferative and autoimmune disorders.