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Analysis

1 / 98,393
1.WO/2025/259747DYSTROPHY MYOTONIC PROTEIN KINASE (DMPK) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO - 18.12.2025
Int.Class A61K 31/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
 Appl.No PCT/US2025/033136 Applicant ALNYLAM PHARMACEUTICALS, INC. Inventor SCHMIDT, Karyn
The disclosure relates to double stranded ribonucleic acid (dsRNA) agents and compositions targeting a dystrophy myotonic protein kinase (DMPK) gene, as well as methods of inhibiting expression of a DMPK gene and methods of treating subjects having a DMPK-associated disease or disorder, e.g., myotonic dystrophy (DM), using such dsRNA agents and compositions.
2.WO/2025/259702METHOD FOR GENERATING CAR-T CELLS IN WHICH CAR EXPRESSION IS COUPLED WITH EXPRESSION OF ENDOGENOUS FOXP3, AND COMPOSITIONS AND METHODS OF USE THEREOF
WO - 18.12.2025
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
 Appl.No PCT/US2025/033055 Applicant ADOPTRACELL, INC. Inventor HARRINGTON, John, Joseph
Methods of generating CAR-Treg cells in which CAR expression is coupled to expression of FoxP3 are provided. Methods disclosed include site-specific modification of genomic FOXP3. Also disclosed are compositions and methods for the treatment of an autoimmune disease using CAR-Treg cells.
3.WO/2025/257301TREATMENT OF NEUROFIBROMATOSIS TYPE 2 BY INHIBITORS OF G6PD, ACSL3 AND/OR OXSM
WO - 18.12.2025
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/EP2025/066359 Applicant DEUTSCHES KREBSFORSCHUNGSZENTRUM STIFTUNG DES ÖFFENTLICHEN RECHTS Inventor TELEMAN, Aurelio
The present invention relates to an inhibitor of an enzyme being involved in promoting cellular reductive capacity, preferably glucose-6-phosphate-dehydrogenase (G6PD) or acyl-CoA synthetase long chain family member 3 (ACSL3) for use in treating neurofibromatosis type 2 or preventing tumors caused by neurofibromatosis type 2.
4.WO/2025/258996APTAMER-RBP-BASED ASSEMBLY FOR EXTRACELLULAR SECRETION OF TARGET CHEMICALS, SYSTEM FOR EXTRACELLULAR SECRETION OF TARGET CHEMICAL SUBSTANCE CONTAINING SAME, AND PRODUCTION METHOD OF TARGET CHEMICALS USING SAME
WO - 18.12.2025
Int.Class C12N 15/115
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
115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith
 Appl.No PCT/KR2025/007982 Applicant CHUNG ANG UNIVERSITY INDUSTRY ACADEMIC COOPERATION FOUNDATION Inventor YOO, Seung Min
The present invention relates to an assembly for extracellular secretion of a target chemical substance, which comprises a conjugate including an aptamer fused to RNA of the target chemical substance that can be produced intracellularly, and a transporter including an RNA-binding protein fused to a carrier protein; a gene construct encoding each of the transporter and the conjugate constituting the assembly; a vector carrying the gene construct; a host cell into having the vector introduced thereinto; a method for producing the target chemical substance, the method comprising a step of culturing the host cell; an extracellular secretion system of the target chemical substance constructed by introducing the gene construct or the vector into a microorganism; and a method for producing the target chemical substance by activating the system. By using the assembly and the system according to the present invention, it is possible to increase the intracellular production as well as extracellular secretion and excretion of the target chemical substance, and the invention can be advantageously applied to the production of microorganism-based useful chemical substances.
5.WO/2025/259761TRNA SEQUENCE ELEMENTS THAT REGULATE MRNA STABILITY
WO - 18.12.2025
Int.Class C12N 15/11
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
 Appl.No PCT/US2025/033162 Applicant THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM Inventor MENDELL, Joshua T.
Provided herein are methods and compositions for modulating CNOT3 recruitment in a cell ribosome. The methods and compositions can modulate a target mRNA stability and/or half-life. Also provided are pharmaceutical compositions for use in the treatment of an mRNA-decay associated disease, nonsense mutation associated disease, obesity, a mitochondrial- associated disorder, or a metabolic disorder.
6.WO/2025/259791COMPOSITIONS AND METHODS FOR TNPB AND ΩRNA MEDIATED TARGETING
WO - 18.12.2025
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/US2025/033213 Applicant THE REGENTS OF THE UNIVERSITY OF CALIFORNIA Inventor DOUDNA, Jennifer A.
Provided are variant TnpB proteins that include an amino acid sequence having one or more amino acid substitutions relative to a corresponding wild type TnpB protein. Also provided are variant TnpB ωRNAs that include: a first nucleotide sequence (that includes one or more mutations relative to a corresponding wild type TnpB ωRNA) that can form a complex with a TnpB protein, and a second nucleotide sequence that can hybridize to a target sequence of a target nucleic acid, thereby guiding the complex to the target sequence. Methods of use are also provided.
7.WO/2025/259836THERAPY FOR OCULAR NEOVASCULARIZATION
WO - 18.12.2025
Int.Class A61K 38/17
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
 Appl.No PCT/US2025/033283 Applicant BETH ISRAEL DEACONESS MEDICAL CENTER, INC. Inventor FERRAN, Christiane
Provided are molecules and compositions for eye-targeted gene therapy and methods of using the same. The eye-targeted gene therapy alleviates ischemic retinopathy by reducing pathologic neovascularization, gliosis and neuronal apoptosis.
8.WO/2025/260061COMPOSITIONS AND METHODS FOR CANCER THERAPY
WO - 18.12.2025
Int.Class A61K 38/17
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
 Appl.No PCT/US2025/033668 Applicant CREATE MEDICINES, INC. Inventor GETTS, Daniel
Compositions comprising a nucleic acid for in vivo engineered cells, such as myeloid cells. The nucleic acid encodes a chimeric fusion protein that can be expressed predominantly on myeloid cells and has a binding domain capable to binding surface molecules on target cells such as cancer cells. Also disclosed are pharmaceutical compositions and methods for treating cancer.
9.WO/2025/256587DOUBLE-STRANDED RNA COMPOSITION CAPABLE OF REGULATING EXPRESSION OF INHBE, CONJUGATE THEREOF, PHARMACEUTICAL COMPOSITION THEREOF AND USE THEREOF
WO - 18.12.2025
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/CN2025/100647 Applicant HANGZHOU ZHONGMEIHUADONG PHARMACEUTICAL CO., LTD. Inventor PAN, Huaye
Provided are a double-stranded RNA capable of regulating the expression of INHBE and a conjugate thereof. Further provided is a pharmaceutical composition comprising the double-stranded RNA or the conjugate thereof. The double-stranded RNA, the conjugate thereof and the pharmaceutical composition thereof have good stability and high INHBE mRNA inhibitory activity, and therefore can be used for the treatment of obesity.
10.WO/2025/258687PAN-CANCER NUCLEIC ACID THERAPEUTIC
WO - 18.12.2025
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
 Appl.No PCT/JP2025/021484 Applicant NATIONAL UNIVERSITY CORPORATION TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH SYSTEM Inventor KANDA Mitsuro
The present invention provides an inhibitor capable of inhibiting the expression of PCDHA11 and inhibiting the function of PCDHA11. Specifically, the present invention provides nucleic acid molecules such as siRNA or antisense nucleic acids that inhibit PCDHA11 expression. These inhibitors are effective against gastric cancer, pancreatic cancer, breast cancer, esophageal squamous cell carcinoma, lung adenocarcinoma, colon cancer, and the like, and can be used as pan-cancer therapeutics.
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