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IC:C12N15/00

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1 / 98,551
1.WO/2025/264140TRINUCLEOTIDE CAPPING REAGENT, METHOD FOR PRODUCING SAME AND USE
WO - 26.12.2025
Int.Class C07H 21/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
 Appl.No PCT/RU2025/000171 Applicant FEDERAL STATE BUDGETARY INSTITUTION "NATIONAL RESEARCH CENTRE FOR EPIDEMIOLOGY AND MICROBIOLOGY NAMED AFTER THE HONORARY ACADEMICIAN N.F. GAMALEYA" OF THE MINISTRY OF HEALTH OF THE RUSSIAN FEDERATION Inventor GINTSBURG, Aleksandr Leonidovich
The group of inventions relates to the field of molecular biology, virology and medicine, and more particularly to novel reagents for the cotranscriptional capping of synthetic mRNA and to a method for producing same. The proposed reagent can be used to produce functional mRNA containing a 5' cap structure analog. The group of inventions makes it possible to create an mRNA cap structure analog that is cotranscriptionally incorporated into the structure of the mRNA and provides for more efficient mRNA translation.
2.WO/2025/262460LIPID COMPOSITIONS FOR NUCLEIC ACID DELIVERY
WO - 26.12.2025
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No PCT/IB2024/056108 Applicant BIONTECH SE Inventor PANZNER, Steffen
The present disclosure provides, among other things, composition and uses of said compositions comprising a cationic lipid, an anionically ionizable lipid, and a nucleic acid, wherein: the cationic lipid comprises a cationic head group, a first bridge group, and a first lipid tail group; the anionically ionizable lipid comprises an anionically ionizable head group, a second bridge group, and a second lipid tail group; the cationic lipid has a lipidic volume from about 700 Å3 to about 1500 Å3, and/or the anionically ionizable lipid has a lipidic volume from about 700 Å3 to about 1500 Å3; or the cationic lipid and the anionically ionizable lipid have a combined lipidic volume greater than 1400 Å3. In some embodiments, the present disclosure provides new cationic lipids and new anionically ionizable lipids.
3.WO/2025/264965INTEGRIN LIGANDS FOR EXTRAHEPATIC DELIVERY
WO - 26.12.2025
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/US2025/034444 Applicant ALNYLAM PHARMACEUTICALS, INC. Inventor PANARESE, Joe
The present invention provides double stranded ribonucleic acid (dsRNA) agents for inhibiting expression of a target gene, comprising an antisense strand which is complementary to the target gene; a sense strand which is complementary to the antisense strand and forms a double stranded region with the antisense strand; and at least one alpha-v-beta-6 (αvβ6) integrin targeting ligand that mediates delivery to an extrahepatic tissues, e.g., muscle tissue, e.g., skeletal muscle tissue and/or cardiac muscle tissue, or lung tissue, conjugated to at least one strand, compositions comprising such dsRNA agents, and methods of use thereof for treating a subject having a disorder that would benefit from reduction in expression of the target gene. The dsRNA may optionally contain an in vivo delivery enhancing agent.
4.WO/2025/261065MONOCLONAL ANTIBODY BINDING TO MICROTUBULE ASSOCIATED PROTEIN TAU WITH 231THR PHOSPHORYLATION AND USE THEREOF
WO - 26.12.2025
Int.Class C07K 16/18
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
 Appl.No PCT/CN2025/096371 Applicant HKIG IMMUNE TECHNOLOGY (HANGZHOU) LTD. Inventor BIAN, Chao
Disclosed in the present invention is a monoclonal antibody binding to microtubule associated protein tau with 231Thr phosphorylation (pTau231). The present invention provides an amino acid sequence of a variable domain of the monoclonal antibody and an amino acid sequence of a complementarity determining region (CDR) comprised therein. The monoclonal antibody of the present invention has the ability to specifically recognize and significantly bind to the pTau231, and can be used as a reagent raw material for the development and production of pTau231 clinical in vitro diagnostic (IVD) medical device products based on the principle of immunological test.
5.WO/2025/260495GENETICALLY ENGINEERED BACTERIUM, PREPARATION METHOD THEREFOR AND USE THEREOF IN DE NOVO SYNTHESIS OF SALIDROSIDE
WO - 26.12.2025
Int.Class C12N 1/21
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
1Microorganisms, e.g. protozoa; Compositions thereof; Processes of propagating, maintaining or preserving microorganisms or compositions thereof; Processes of preparing or isolating a composition containing a microorganism; Culture media therefor
20Bacteria; Culture media therefor
21modified by introduction of foreign genetic material
 Appl.No PCT/CN2024/113711 Applicant ASYMCHEM LABORATORIES (TIANJIN) CO., LTD. Inventor HONG, Hao
Provided are a genetically engineered bacterium, a preparation method therefor and a use thereof in de novo synthesis of salidroside. The genetically engineered bacterium comprises an exogenously introduced phenylpyruvate decarboxylase gene ARO10 and a glycosyltransferase gene OfT8GT1, wherein the phenylpyruvate decarboxylase gene ARO10 has a nucleotide sequence as shown in SEQ ID NO: 3, and the glycosyltransferase gene OfT8GT1 has a nucleotide sequence as shown in SEQ ID NO: 4. The method for synthesizing salidroside from the genetically engineered bacterium by catalysis greatly reduces synthesis costs, simplifies subsequent separation and purification steps, and exhibits high efficiency of salidroside synthesis, thereby laying a foundation for industrial production of salidroside by using a microbial fermentation method.
6.WO/2025/260975METHOD FOR GENE EDITING OF HEMATOPOIETIC STEM CELL, COMPOSITION AND USE THEREOF
WO - 26.12.2025
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/CN2025/091695 Applicant CARBIOGENE THERAPEUTICS CO., LTD. Inventor ZHU, Jiangao
Disclosed in the present invention are a method for the gene editing of a hematopoietic stem cell, a composition, and a use thereof. Specifically disclosed are a sequence of sgRNA and a use thereof. The present invention establishes a method for using the sgRNA to efficiently edit a hematopoietic stem cell in vitro. The gene editing efficiency can reach 77% or more. The hematopoietic stem cell with CLL1 gene knocked out by using the sgRNA has a good myeloid differentiation function without affecting the function of a differentiated myeloid cell derived therefrom, and both the hematopoietic stem cell with CLL1 gene knocked out and the differentiated myeloid cell derived therefrom can effectively avoid the killing effect of a CLL1-CAR-T cell. The method of the present invention can be used in an application of CAR-T cell therapy combined with hematopoietic stem cell transplantation therapy, can solve the problem of off-target toxicity caused by a CLL1-targeting CAR-T cell recognizing and attacking a normal myeloid cell, and has important clinical value and application prospects for AML treatment.
7.WO/2025/261380TRIMERIZATION REGULATORY ELEMENT, METHOD FOR CONSTRUCTING SAME, AND USE THEREOF
WO - 26.12.2025
Int.Class C12N 9/50
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.)
48acting on peptide bonds, e.g. thromboplastin, leucine aminopeptidase (3.4)
50Proteinases
 Appl.No PCT/CN2025/101638 Applicant SHANGHAITECH UNIVERSITY Inventor WANG, Zhizhi
Disclosed are a trimerization regulatory element, a method for constructing same, and use thereof. In addition to a protein A and a protein B that are obtained by splitting the hepatitis C virus protease NS3a, the trimerization regulatory element comprises a protein C. The trimerization of the trimerization regulatory element can be mediated by means of a targeting drug specific to the protease NS3a (grazoprevir/danoprevir). The trimerization regulatory element provided by the present invention has an excellent small-molecule-mediated trimerization effect, and a drug approved by the National Medical Products Administration is used. The protein used does not exist in the human body; therefore, the protein can result in better biological orthogonality. Moreover, the protein can be used as a clinically effective drug regulatory switch, effectively avoiding problems such as serious cytotoxicity and off-target effects.
8.WO/2025/261230ENGINEERED CELLS AND USE THEREOF
WO - 26.12.2025
Int.Class C12N 5/10
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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
 Appl.No PCT/CN2025/100326 Applicant TIANHAI YUANQI BIOTECHNOLOGY (TIANJIN) CO., LTD. Inventor CHENG, Tao
Disclosed are engineered cells and use thereof. Compared with the wild type, the engineered cells lack or reduce the expression of the HLA-A protein and/or the HLA-B protein. The disclosed engineered cells can significantly reduce the immune rejection response after artificial blood cell infusion, and significantly improve the implantation rate and long-term reconstruction ability in vivo. The cells will significantly expand the use of existing cord blood and bone marrow donor libraries, reduce the need to recruit a large number of donors to match receptors, and increase the probability of HLA matching donors, thereby expanding the infusion of clinical-grade allogeneic cells. As a source of universal cells, the engineered cells can enable HLA-matching off-the-shelf cell therapy, showing great promise in the area of clinical transplantation treatment.
9.WO/2025/262205NUCLEIC ACID SEQUENCES
WO - 26.12.2025
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/EP2025/067232 Applicant UCB BIOPHARMA SRL Inventor KAS, Onur Yusuf
The invention relates to the field of recombinant polynucleotides comprising adenoviral genome sequences. It also relates to methods and processes comprising such nucleic acid sequences.
10.WO/2025/264980AMBIENT STABILIZATION OF MRNA-HYBRID LIPID NANOCAPSULES IN SUGAR GLASS
WO - 26.12.2025
Int.Class A61K 9/1271
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
 Appl.No PCT/US2025/034475 Applicant GEORGIA TECH RESEARCH CORPORATION Inventor YADAVA, Sunil Kumar
An exemplary embodiment of the present disclosure provides nucleic acid delivery system comprising a nucleic acid therapeutic agent, a hybrid lipid nanocapsule comprising a fatty acid-polyamine conjugate or fatty acid-polypeptide conjugate capable of condensing with the nucleic acid therapeutic agent, and a sugar glass. Also disclosed herein is a hybrid lipid nanocapsule for delivery of a nucleic acid into a cell and a method for delivering a nucleic acid into a cell.
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