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Analysis

1 / 366
1.20250340907METHODS AND COMPOSITIONS FOR MODULATING A GENOME
US - 06.11.2025
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 19272711 Applicant Flagship Pioneering Innovations VI, LLC Inventor Anne Helen Bothmer

Methods and compositions for modulating a target genome are disclosed. This disclosure relates to novel compositions, systems and methods for altering a genome at one or more locations in a host cell, tissue or subject, in vivo or in vitro. In particular, the invention features compositions, systems and methods for inserting, altering, or deleting sequences of interest in a host genome.

2.WO/2025/228326RECOMBINANT RNA MOLECULE AND USE THEREOF
WO - 06.11.2025
Int.Class C12N 15/63
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
 Appl.No PCT/CN2025/091753 Applicant SHENZHEN SHENXIN BIOTECHNOLOGY CO., LTD. Inventor LI, Linxian
Provided are a recombinant RNA molecule and a use thereof. The recombinant RNA molecule comprises a polynucleotide encoding a polypeptide of interest and 5'-UTR and/or 3'-UTR. The recombinant RNA molecule has improved translation efficiency and/or stability; and the expression level of the polypeptide and/or protein is increased. Also provided are a vector encoding the recombinant RNA molecule, a pharmaceutical composition comprising the recombinant RNA molecule, and a method for treating or preventing a disease by using the recombinant RNA molecule.
3.WO/2025/231360NOVEL CAS NUCLEASES AND POLYNUCLEOTIDES ENCODING THE SAME
WO - 06.11.2025
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/US2025/027494 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 genetic editing.
4.20250340863POLYPEPTIDE-ENCODED LIBRARY AND SCREENING METHOD USING SAME
US - 06.11.2025
Int.Class C12N 15/10
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
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
10Processes for the isolation, preparation or purification of DNA or RNA
 Appl.No 18870952 Applicant WHOLESOME FUTURE LIMITED Inventor Haifeng ZHAO

A polypeptide-encoded nucleic acid or small molecule compound library includes polypeptide barcode tags and nucleic acid molecules or small molecule compounds corresponding to the polypeptide barcode tags. Further provided is a polypeptide-encoded nucleic acid or small molecule compound library, the library comprising a plurality of beads, and each bead comprising a polypeptide barcode tag connected to the bead and a nucleic acid molecule or a small molecule compound corresponding to the polypeptide barcode tag. A method is for screening a target nucleic acid or small molecule via the polypeptide-encoded nucleic acid or small molecule compound library. A screened nucleic acid molecule or small molecule compound is identified by decoding a polypeptide barcode tag associated therewith.

5.20250340877RNAi Agents for Inhibiting Expression of Complement Component C3 (C3), Pharmaceutical Compositions Thereof, and Methods of Use
US - 06.11.2025
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 19188473 Applicant Arrowhead Pharmaceuticals, Inc. Inventor Jeffrey Carlson

The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents or siRNAs, able to inhibit Complement Component C3 (C3) gene expression. Also disclosed are pharmaceutical compositions that include C3 RNAi agents and methods of use thereof. The C3 RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that comprise N-acetyl-galactosamine, to facilitate the delivery to hepatocyte cells. Delivery of the C3 RNAi agents in vivo provides for inhibition of C3 gene expression. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by C3 gene expression, including IgA nephropathy, C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria, and/or other complement-mediated renal diseases.

6.WO/2025/229953METHOD FOR PRODUCING OLIGONUCLEOTIDE
WO - 06.11.2025
Int.Class C07H 21/02
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
02with ribosyl as saccharide radical
 Appl.No PCT/JP2025/016225 Applicant SUMITOMO CHEMICAL COMPANY, LIMITED Inventor MIYAGAWA, Takuya
The purpose of the present invention is to provide a method for efficiently producing an oligonucleotide by a solid-phase synthesis approach. The present invention provides a method for producing an oligonucleotide by a solid phase synthesis approach, the method comprising a step for reacting an oligonucleotide in which a hydroxyl group at a chain extendable end is protected with a protective group that can be removed under acidic conditions and a deblocking solution to remove a protective group for a hydroxyl group at the chain extendable end, wherein the deblocking solution is a deblocking solution containing a base, an acid, and an aprotic solvent, and the base is a base having an acid dissociation constant (pKa) of a conjugate acid of 5-12, and an oligonucleotide in which the content ratio of the 2', 3'transfer body is a certain amount or less.
7.WO/2025/231484METHODS OF PREPARING CAPPED MRNA WITH SITE-SPECIFIC MODIFICATIONS
WO - 06.11.2025
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 PCT/US2025/027793 Applicant GENSCRIPT USA INC. Inventor PI, Fengmei
A method for producing a site-specific modified capped recombinant RNA includes (a) obtaining a first RNA that is monophosphorylated at the 5' end and contains the site-specific modification, (b) reacting an activated capping compound of Formula (I), Formular (II), or Formula (III) with the 5' monophosphorylated first RNA obtained from (a) in the presence of a heteroaromatic compound, a metal salt, and a solvent, hereby producing a 5' capped first RNA, (c) obtaining a second RNA that is monophosphorylated at the 5' end, and (d) ligating the 3' end of the 5' capped first RNA obtained from (b) to the 5' end of the second RNA obtained from (c), thereby producing the site-specific modified capped recombinant RNA.
8.WO/2025/228409LIPID NANOPARTICLE AND USE THEREOF
WO - 06.11.2025
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/CN2025/092313 Applicant SHENZHEN SHENXIN BIOTECHNOLOGY CO., LTD. Inventor LI, Linxian
Provided is a lipid nanoparticle, which comprises an active component, an ionizable lipid, an auxiliary lipid, a structural lipid, and a polymer lipid. The polymer lipid accounts for 0-0.8 mol% of the total lipid existing in the lipid nanoparticle. The reduction in the content of the polymer lipid in the lipid nanoparticle can change the delivery specificity of the lipid nanoparticle, such that the lipid nanoparticle can target the spleen or immune cells.
9.WO/2025/227005RNAI AGENTS FOR INHIBITING EXPRESSION OF MYOCILIN (MYOC), COMPOSITIONS THEREOF, AND METHODS OF USE
WO - 30.10.2025
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/US2025/026321 Applicant ARROWHEAD PHARMACEUTICALS, INC. Inventor LI, Xiaokai
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Myocilin (MYOC) gene. The MYOC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an MYOC gene. Pharmaceutical compositions that include one or more MYOC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MYOC RNAi agents to ocular tissue, in vivo, provides for inhibition of MYOC gene expression and a reduction in MYOC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including glaucoma, e.g., primary open-angle glaucoma (POAG).
10.20250333473CELLS LACKING B2M SURFACE EXPRESSION AND METHODS FOR ALLOGENEIC ADMINISTRATION OF SUCH CELLS
US - 30.10.2025
Int.Class C07K 14/74
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
74Major histocompatibility complex
 Appl.No 18949619 Applicant President and Fellows of Harvard College Inventor Chad A. Cowan

Disclosed herein are cells and populations of cells comprising a genome in which the B2M gene has been edited to eliminate surface expression of MHC Class I protein in the cells or population of cells, and methods for allogeneic administration of such cells to reduce the likelihood that the cells will trigger a host immune response when the cells are administered to a subject in need of such cells.

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