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Analysis

1 / 420
1.WO/2025/223684METHODS AND COMPOSITIONS FOR STIMULATING IMMUNE RESPONSE
WO - 30.10.2025
Int.Class A61K 39/39
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
39characterised by the immunostimulating additives, e.g. chemical adjuvants
 Appl.No PCT/EP2024/071234 Applicant BIONTECH SE Inventor SAHIN, Ugur
The present invention relates to methods and compositions for stimulating an immune response. In particular, the present invention relates to compositions comprising an immunostimulatory RNA comprising sequences derived from an Influenza A virus nudeoprotein-encoding RNA molecule that act as adjuvants and/or immunostimulatory agents to enhance host immune responses, in particular as adjuvants for cancer vaccines.
2.WO/2025/223309LIPID COMPOUND, LIPID NANOPARTICLE CONTAINING LIPID COMPOUND, AND USE THEREOF
WO - 30.10.2025
Int.Class C07C 235/10
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
235Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
02having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
04the carbon skeleton being acyclic and saturated
10having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by nitrogen atoms not being part of nitro or nitroso groups
 Appl.No PCT/CN2025/089754 Applicant THEMEDIUM THERAPEUTICS CO., LTD Inventor ZHANG, Lei
The present application provides a lipid compound, a lipid nanoparticle containing the lipid compound, and a use thereof in the preparation of a drug delivery carrier. The lipid nanoparticle of the present application can deliver a therapeutic agent or prophylactic agent (especially a nucleic acid therapeutic agent or prophylactic agent) to a site of injection by means of intramuscular injection. The therapeutic agent or the prophylactic agent has a high expression level in the site of injection, but shows a relatively low expression level in viscera, so that the drug can efficiently exert activity and has a relatively low visceral toxicity. The present application is of great significance to the development and application of nucleic acid therapeutic agents or prophylactic agents.
3.20250327070COMPOSITIONS 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.

4.WO/2025/221850REGULATORY SEQUENCES
WO - 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 PCT/US2025/024902 Applicant GINKGO BIOWORKS, INC. Inventor FLYNN, Elise, Duboscq
The present disclosure provides modular regulatory elements useful for transcription and gene expression.
5.WO/2025/222086NOVEL INTRON SEQUENCES FOR CIRCULARIZING RNA MOLECULES
WO - 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 PCT/US2025/025308 Applicant GINKGO BIOWORKS, INC. Inventor SHERMAN, Mark
In certain aspects, the present disclosure provides nucleic acid molecules containing an intron pair for production of linear and/or circularized nucleic acid molecules, as well as methods of preparation and methods of use.
6.20250325494COMPOSITION FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACID
US - 23.10.2025
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 19008633 Applicant THEMEDIUM THERAPEUTICS CO., LTD. Inventor Lei ZHANG

The present application provides a composition for organ-specific delivery of nucleic acids, which comprises a guided on-target lipid delivery lipid; further, the composition may comprise a helper lipid; still further, the composition may comprise a cationic lipid. The guided on-target lipid delivery lipid may be selected from one or more of an ionizable anionic steroid and/or an ionizable anionic polymer conjugated lipid; the helper lipid is optionally one or more of a phospholipid, a steroid, a polymer conjugated lipid, and a modifiable lipid; and the cationic lipid may be selected from one or more of a permanently cationic lipid and/or an ionizable cationic lipid. The delivery composition is capable of specifically delivering a prophylactic/therapeutic agent, particularly a nucleic acid component, to a target organ.

7.20250327049CBLB ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE
US - 23.10.2025
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 19185520 Applicant REGENERON PHARMACEUTICALS, INC. Inventor Jordan JARJOUR

The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.

8.WO/2025/222093ENGINEERED CELLS AND USES THEREOF
WO - 23.10.2025
Int.Class C07K 14/05
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
01DNA viruses
03Herpetoviridae, e.g. pseudorabies virus
05Epstein-Barr virus
 Appl.No PCT/US2025/025324 Applicant FACTOR BIOSCIENCE INC. Inventor ANGEL, Matthew
The present disclosure provides engineered induced pluripotent stem cells (iPSCs) expressing B2M-HLA fusion constructs. The present disclosure also provides iPSC-derived macrophages which overexpress IL-12. The present disclosure further provides engineered immune cells having persistent cytokine expression.
9.WO/2025/218812NOVEL INTERNAL RIBOSOME ENTRY SITES AND USES THEREOF
WO - 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 PCT/CN2025/090211 Applicant SHANGHAI CIRCODE BIOMED CO., LTD. Inventor
Provided are novel Internal Ribosome Entry Site (IRES) sequences that improve cap-independent translation in eukaryotic cells. Provides herein are nucleic acids incorporating such sequences, vectors and host cells comprising these sequences, methods for their use in producing proteins of interest, and a method of improving expression, functional stability, immunogenicity, ease of manufacturing and/or half-life of a therapeutic protein encoded by the circular RNA.
10.20250325490LIPID COMPOUND, LIPID NANOPARTICLE COMPRISING SAME AND USE THEREOF
US - 23.10.2025
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 18810352 Applicant TheMedium Therapeutics Co., Ltd Inventor Lei Zhang

The present disclosure provides a lipid compound, a lipid nanoparticle comprising the same and use thereof in the preparation of a drug delivery carrier. The lipid nanoparticle of the present disclosure can deliver a therapeutic or prophylactic agent (particularly a nucleic acid substance) to an injection site by intramuscular injection, and the therapeutic or prophylactic agent has high expression at the injection site but low expression in the viscera, which enables a drug to not only exert high activity but also have low visceral toxicity, and is of great significance to development and application of nucleic acid therapeutic or prophylactic agents.

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