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1.WO/2025/140549IMMUNE STIMULATING ANTI-HBSAG ANTIBODY CONJUGATES, PHARMACEUTICAL COMPOSITIONS, AND THERAPEUTIC APPLICATIONS
WO 03.07.2025
Int.Class A61K 47/68
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
68the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
Appl.No PCT/CN2024/143178 Applicant ETINPRO (BEIJING) CO. LTD. Inventor XU, Zheng
Provided herein are immune stimulating anti-HBsAg antibody conjugates, e.g., a compound of Formula (I), and pharmaceutical compositions thereof. Also provided herein are methods of their use for treating, preventing, or ameliorating one or more symptoms of an HBV infection.
2.WO/2025/141146ANTI-CCR9 ANTIBODY
WO 03.07.2025
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
Appl.No PCT/EP2024/088546 Applicant SUNROCK BIOPHARMA S.L. Inventor SIMÓN BUELA, Laureano
The present invention provides an anti-CCR9 antibody or antigen binding fragment thereof comprising a variable light chain comprising SEQ ID NO:1 or a variant thereof having at least 95% sequence identity to SEQ ID NO:1, wherein the variable light chain comprises a CDR-L1 comprising the amino acid sequence RSSQSLXHSNGXTFLH (SEQ ID NO: 2), a CDR-L2 comprising the amino acid sequence KVSNRFS (SEQ ID NO: 3) and a CDR-L3 comprising the amino acid sequence SQTTHXPWT (SEQ ID NO: 4), wherein X can be any amino acid. Also provided is the anti-CCR9 antibody or antigen binding fragment thereof for use in the treatment or prevention of a disease, as well as methods of diagnosis and antibody drug conjugates.
3.WO/2025/144736POLYNUCLEOTIDES AND METHODS FOR GENE EDITING
WO 03.07.2025
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Appl.No PCT/US2024/061421 Applicant ENCELADUS BIO, INC. Inventor KITA, Ryosuke
Compounds, polypeptides, compositions, and nucleic acid molecules are provided herein that may be used, for example, to edit the genome of a cell, such as a human cell.
4.WO/2025/143118PHARMACEUTICAL COMPOSITION FOR TREATING SPINAL CORD INJURY AND METHOD FOR PRODUCING NON-HUMAN PRIMATE ANIMAL MODEL FOR SPINAL CORD INJURY
WO 03.07.2025
Int.Class A61K 31/7105
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
Appl.No PCT/JP2024/046150 Applicant ASTELLAS PHARMA INC. Inventor NAKAHARA, Soichiro
The present invention pertains to: a pharmaceutical composition for treating spinal cord injury, the pharmaceutical composition containing an mRNA encoding NeuroD1 protein; a method for producing a non-human primate animal model with which it is possible to assess spinal cord injury (in particular, chronic-stage spinal cord injury); and a method for assessing, by using the model, the effect of a test substance as a therapeutic agent for spinal cord injury.
5.20250207139RNAi Agents for Inhibiting Expression of Activin Receptor-Like Kinase 7 (ALK7), Compositions Thereof, and Methods of Use
US 26.06.2025
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Appl.No 18988719 Applicant Arrowhead Pharmaceuticals, Inc. Inventor Michelle Ngai

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an Activin Receptor-Like Kinase 7 (ALK7) gene. The ALK7 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ALK7 gene. Pharmaceutical compositions that include one or more ALK7 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ALK7 RNAi agents to adipose tissue, in vivo, provides for inhibition of ALK7 gene expression and a reduction in ALK7 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including obesity, diabetes, or insulin resistance.

6.WO/2025/131119CANCER VACCINES AND USES THEREOF
WO 26.06.2025
Int.Class C12N 15/62
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
62DNA sequences coding for fusion proteins
Appl.No PCT/CN2024/141468 Applicant EVEREST MEDICINES (CHINA) CO., LTD. Inventor HU, Gang
Provided herein are nucleic acids, pharmaceutical compositions and vaccines that express a cancer antigen (e.g., PRAME). Also provided herein are methods for treating a cancer.
7.WO/2025/137390RNAI AGENTS FOR INHIBITING EXPRESSION OF ACTIVIN RECEPTOR-LIKE KINASE 7 (ALK7), COMPOSITIONS THEREOF, AND METHODS OF USE
WO 26.06.2025
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Appl.No PCT/US2024/061179 Applicant ARROWHEAD PHARMACEUTICALS, INC. Inventor NGAI, Michelle
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an Activin Receptor-Like Kinase 7 (ALK7) gene. The ALK7 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ALK7 gene. Pharmaceutical compositions that include one or more ALK7 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ALK7 RNAi agents to adipose tissue, in vivo, provides for inhibition of ALK7 gene expression and a reduction in ALK7 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including obesity, diabetes, or insulin resistance.
8.WO/2025/137275NOVEL CAS NUCLEASES AND POLYNUCLEOTIDES ENCODING THE SAME
WO 26.06.2025
Int.Class C12N 9/22
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
Appl.No PCT/US2024/061026 Applicant MODERNATX, INC. Inventor AZMI, Ishara
The present disclosure provides novel CRISPR-associated (Cas) nucleases, as well as polynucleotides (e.g., DNA and RNA polynucleotides) encoding the same, host cells containing such nucleases and polynucleotides, and methods of using the foregoing compositions to effectuate genome editing.
9.20250207163METHODS AND APPARATUS FOR SYNTHESIZING NUCLEIC ACIDS
US 26.06.2025
Int.Class C12P 19/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
Appl.No 18850185 Applicant Molecular Assemblies, Inc. Inventor Patrycja A. Hopkins

Methods for the synthesis of polynucleotides using polymerases nucleotide analogs with labile terminator groups that allow stepwise addition of nucleotides are described. These nucleotide analogs may have a modification on 3′, 2′, or 3′-2′-bridged hydroxyl groups or on nucleobase moieties and can be used in the synthesis of natural or modified polynucleotides suitable for use in biological systems. The labile terminator groups are removed under mild conditions or by enzymes. They are accepted as substrates by polymerases. The synthesis may be carried out on a surface.

10.WO/2025/137547MUSCLE TARGETING COMPLEXES AND FORMULATIONS FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
WO 26.06.2025
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
Appl.No PCT/US2024/061427 Applicant DYNE THERAPEUTICS, INC. Inventor WEEDEN, Timothy
Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., an RNAi oligonucleotide such as an siRNA, useful for targeting DUX4) covalently linked to an antibody (e.g., anti-TfRl antibody).