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Analysis

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.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.
5.20250332244IMMUNOGENIC COMPOSITIONS AGAINST INFLUENZA
US 30.10.2025
Int.Class A61K 39/145
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
145Orthomyxoviridae, e.g. influenza virus
Appl.No 18882451 Applicant Pfizer Inc. Inventor Bogos Agianian

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use ribonucleic acid vaccines comprising polynucleotide molecules encoding one or more influenza antigens, such as hemagglutinin antigens.

6.WO/2025/226652NUCLEIC ACID SEQUENCES FOR REDUCING CHEMORESISTANCE AND TREATING DISEASE
WO 30.10.2025
Int.Class A61K 31/7115
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
7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
Appl.No PCT/US2025/025725 Applicant THE CLEVELAND CLINIC FOUNDATION Inventor LI, Xiaoxia
Provided herein are compositions, systems, kits, and methods for reducing chemoresistance (e.g., to platinum containing chemotherapeutics) and treating certain diseases, using nucleic acid sequences (e.g., aptamers and antisense sequences) that bind to certain mRNAs (e.g., WTAP), or bind a SEFIR domain of an ACT1 protein, or bind to or inhibit mRNA sequences that bind to Act1 protein.
7.20250327070COMPOSITIONS AND METHODS FOR INHIBITING COMPLEMENT FACTOR B
US 23.10.2025
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 18292187 Applicant ASTRAZENECA IRELAND LIMITED Inventor Melissa Lasaro

Described herein are oligonucleotides (e.g., RNAi oligonucleotides) containing sense and antisense strands for targeting complement factor B (CFB) mRNA. The RNAi oligonucleotide may be used to inhibit CFB expression, levels, and/or activity in a cell. Also, described herein are methods for using an oligonucleotide (e.g., an RNAi oligonucleotide) for the prophylaxis or treatment of a disease, disorder, or condition mediated by complement pathway activation or dysregulation.

8.120813692工程化指导RNA和多核苷酸
CN 17.10.2025
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 202380094407.2 Applicant 塑造治疗公司 Inventor 扬尼斯·萨瓦
本文公开了用于治疗受试者的疾病或病症的工程化指导RNA以及包含其的组合物。本文还公开了通过施用本文所述的工程化指导RNA或药物组合物来治疗受试者的疾病或病症的方法。
9.20250320519SYNTHETIC CIRCULAR RNA COMPOSITIONS AND METHODS OF USE THEREOF
US 16.10.2025
Int.Class C12N 15/85
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
Appl.No 18868935 Applicant SHANGHAI CIRCODE BIOMED CO., LTD. Inventor Yun YANG

The present disclosure relates to compositions, methods, processes, kits and devices for the selection, design, preparation, manufacture, formulation, and/or use of a polynucleotide having an Internal Ribosome Entry Site (IRES) sequence, an IRES-like sequence or a combination thereof. In particular, the present disclosure relates to compositions, methods, processes, kits and devices for the selection, design, preparation, manufacture, formulation, and/or use of a circular polynucleotide (e.g., a circular RNA). The present disclosure also relates to a method of improving expression, functional stability, immunogenicity, ease of manufacturing and/or half-life of a therapeutic product encoded by the circular RNA.

10.20250319036BISPHOSPHONATE LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY
US 16.10.2025
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 18865098 Applicant The Trustees of the University of Pennsylvania Inventor Michael J. Mitchell

Described herein, in some aspects, are bisphosphonate lipid compounds, lipid nanoparticles (LNPs) thereof, and methods of use thereof. In various embodiments, the LNP selectively targets a cell of interest (e.g., a bone cell and/or bone marrow cell, such as a stem cell, stroma cell, osteoblast, osteocyte, osteoclast, bone lining cell, local mesenchymal cell, progenitor cell, mononuclear blood-borne precursor cell, B cell, endothelial cell, granulocytes, T cell, monocytic lineage, B cell lineage, monocytes, cancer cell, tumor cell, tumor cell that metastasizes to bone, blood cancer cell, and multiple myeloma cell, inter alia). In other aspects, the present disclosure relates to methods for in vivo delivery of therapeutic agents to prevent or treat diseases, disorders, or conditions using the LNP compositions of the disclosure.