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IC:C12N15/00

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Analysis

1 / 99,265
1.WO/2026/026712SIRNA FOR INHIBITING MASP2 GENE EXPRESSION, AND MODIFIER THEREOF AND USE THEREOF
WO - 05.02.2026
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/CN2025/110900 Applicant FRONTIER BIOTECHNOLOGIES INC. Inventor WANG, Michael Zhiyan
Provided in the present invention is a double-stranded MASP2-targeting RNA molecule, specifically an siRNA. The siRNA provided in the present invention is capable of effectively inhibiting MASP2 expression, and can be used for preventing and treating diseases associated with MASP2 high expression or abnormal activation.
2.WO/2026/026847B7-H3-SPECIFIC ANTIBODY AND ANTI-TUMOR USE THEREOF
WO - 05.02.2026
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/CN2025/111475 Applicant ZHENGZHOU UNIVERSITY Inventor LIU, Kangdong
The present invention provides an anti-B7-H3 (CD276) antibody or an antigen-binding fragment thereof, comprising CDR-L1, CDR-L2 and CDR-L3 as shown in SEQ ID NOs: 1-3, respectively, and CDR-H1, CDR-H2 and CDR-H3 as shown in SEQ ID NOs: 4-6, respectively, and a use thereof in treating tumors.
3.WO/2026/026937SIRNA FOR INHIBITING HSD17B13 GENE EXPRESSION, AND MODIFICATION AND USE THEREOF
WO - 05.02.2026
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/CN2025/112029 Applicant FRONTIER BIOTECHNOLOGIES INC. Inventor WANG, Michael Zhiyan
Provided are an siRNA for inhibiting HSD17B13 gene expression, and a modification and a use thereof, belonging to the technical field of biopharmaceuticals. The double-stranded RNA molecule is an siRNA, and the siRNA comprises a sense strand and an antisense strand at least partially forming a double-stranded region, the sense strand comprising a nucleotide sequence that is any odd-numbered sequence from sequences 1-394. The aforementioned siRNA can efficiently degrade HSD17B13 mRNA and significantly reduce HSD17B13 protein levels, and can be used to prepare a novel drug for treating diseases caused by abnormal HSD17B13 expression, including but not limited to fatty liver, NAFLD, and NASH.
4.WO/2026/026952AGT-TARGETING DOUBLE-STRANDED SIRNA, CONJUGATE THEREOF, AND USE THEREOF
WO - 05.02.2026
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/CN2025/112133 Applicant SUNSHINE LAKE PHARMA CO., LTD. Inventor SHU, Zhiyu
Provided are an AGT-targeting double-stranded siRNA, a conjugate thereof, and a use thereof. The double-stranded siRNA and the conjugate thereof can inhibit the expression of angiotensinogen (AGT), and can be used for preparing a drug for treating and/or preventing AGT-related diseases.
5.WO/2026/026982SIRNA TARGETING MAPT GENE EXPRESSION, AND CONJUGATE THEREOF AND USE THEREOF
WO - 05.02.2026
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/CN2025/112493 Applicant LEADERNA THERAPEUTICS, LTD. Inventor ZHANG, Shuai
Provided are an siRNA targeting microtubule-associated protein tau (MAPT) gene expression, and a conjugate thereof and the use thereof, wherein the siRNA contains a sense strand and an antisense strand. The provided siRNA, siRNA conjugate and pharmaceutical composition have good stability and good MAPT gene inhibitory activity.
6.WO/2026/025197NON-AQUEOUS COMPOSITIONS FOR STORAGE AND/OR STABILIZATION OF NUCLEIC ACID AND METHODS OF USE THEREOF
WO - 05.02.2026
Int.Class C12Q 1/6806
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction assay
 Appl.No PCT/CA2025/051032 Applicant DNA GENOTEK INC. Inventor WOOD, Cameron Gary
The present application provides a non-aqueous storage composition for use in storage or stabilization of nucleic acid from or in biological samples. The non-aqueous storage composition comprises a non-aqueous solvent that comprises: dimethylsulfoxide, a glycol, glycerol or a mixture of two or more thereof; one or more surfactant; a buffer to provide a pH in the range of from about 4 to about 8; and, optionally, a chelator. Also provided herein are methods and kits for storage or stabilization of nucleic acid from or in biological samples. Further provided herein are methods and kits for concentration of nucleic acid or other analyte(s) of interest from a liquid biological sample using the non-aqueous storage composition.
7.WO/2026/029847METHODS AND COMPOSITIONS FOR PANDEMIC INFLUENZA VACCINE
WO - 05.02.2026
Int.Class C12N 15/86
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
 Appl.No PCT/US2025/031490 Applicant ARCTURUS THERAPEUTICS, INC. Inventor SULLIVAN, Brian
Provided herein are RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof. Also provided herein are compositions that include RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof, and lipids. RNA molecules and compositions including them are useful for inducing immune responses.
8.WO/2026/026592SIRNA FOR INHIBITING EXPRESSION OF MAPT GENE, CONJUGATE THEREOF AND USE THEREOF
WO - 05.02.2026
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/CN2025/109616 Applicant BEIJING YOUNGEN BIOTECHNOLOGY CO., LTD. Inventor ZHONG, Hui
The present invention relates to the field of biomedicine, and in particular to an siRNA for inhibiting expression of an MAPT gene, a conjugate thereof and a use thereof. The siRNA of the present invention comprises a sense strand and an antisense strand. The sense strand comprises nucleotide sequence I, and the antisense strand comprises nucleotide sequence II. Each nucleotide in the nucleotide sequence I and the nucleotide sequence II is a modified or unmodified nucleotide. The nucleotide sequence I and the nucleotide sequence II are at least partially reverse complementary to form a double-stranded region. The nucleotide sequence I is substantially identical to a first segment of nucleotide sequence. The first segment of nucleotide sequence is a segment of nucleotide sequence having a length of at least 15 nucleotides in an mRNA expressed by the MAPT gene. The siRNA and the conjugate thereof provided by the present invention can specifically induce the degradation of MAPT mRNA and thus inhibit the synthesis of MAPT, resulting in the reduction of Tau protein, thereby effectively preventing and/or treating neurodegenerative diseases.
9.WO/2026/028137FIBROBLAST GROWTH FACTOR 21 (FGF21) GENE THERAPY
WO - 05.02.2026
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/IB2025/057770 Applicant UNIVERSITAT AUTÒNOMA DE BARCELONA Inventor BOSCH TUBERT, Maria Fàtima
According to various aspects of this disclosure, the present disclosure relates to methods for reducing kidney inflammation, reducing kidney fibrosis, reducing oxidative stress in the kidney, preventing or reducing the likelihood of chronic kidney disease (CKD), treating or preventing acute kidney injury (AKI) or treating a kidney disease in a subject in need thereof comprising administering, e.g., intramuscularly, to the subject a recombinant adeno-associated virus (rAAV) vector, wherein the rAAV vector comprises a vector genome comprising a nucleotide sequence encoding a Fibroblast growth factor 21 (FGF21) or a functional fragment thereof and an AAV capsid (e.g., AAV1 serotype), optionally, wherein the subject does not suffer from diabetes.
10.WO/2026/030432PROCESSES FOR THE PURIFICATION OF VIRAL PARTICLES AND FORMULATIONS THEREOF
WO - 05.02.2026
Int.Class C12N 15/867
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
867Retroviral vectors
 Appl.No PCT/US2025/039850 Applicant INTERIUS BIOTHERAPEUTICS, INC. Inventor MEDI, Muneeswara, Babu
Provided for herein are pharmaceutical compositions comprising a virus, such as lentiviruses, including pseudotyped lentiviruses and uses thereof. Also provided for herein are methods of purifying and/or concentrating viral vectors and methods of producing sterile compositions, such as, sterile pharmaceutical compositions, wherein the methods can perform steps after passing the viral vector through a protein coated sterile filter. Also provided for herein are methods of using the pharmaceutical compositions.
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