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Analysis

1 / 579
1.20250327070COMPOSITIONS 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.

2.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.
3.20250325469Inhibitors of atp synthase - cosmetic and therapeutic uses
US - 23.10.2025
Int.Class A61K 8/64
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
8Cosmetics or similar toiletry preparations
18characterised by the composition
30containing organic compounds
64Proteins; Peptides; Derivatives or degradation products thereof
 Appl.No 18271488 Applicant Michael David Forrest Inventor Michael David Forrest

With supporting experimental data, this disclosure teaches that IF1 protein activity is a molecular determinant of lifespan, therein explaining why different species have different maximal lifespans, and it teaches a IF1 protein/fragment (or sequence variant thereof), or a fusion protein thereof, optionally a fusion protein comprising a Cell Penetrating Peptide (CPP) sequence, as an agent to slow/delay/reduce aging in a subject, optionally as a component of a cosmetic, optionally to treat an age-correlated disease/disorder. Moreover it teaches other inhibitors of F1F0 ATP hydrolysis, including small molecules, of a number of different scaffolds, for this purpose. Furthermore, with supporting experimental data, it teaches that compounds that slow the ATP-hydrolysing mode of ATP synthase are useful for treating various diseases and disorders, including cancer, particularly cancers that utilise the Warburg effect.

4.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.
5.20250325494COMPOSITION 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.

6.20250327045PRIME EDITOR VARIANTS, CONSTRUCTS, AND METHODS FOR ENHANCING PRIME EDITING EFFICIENCY AND PRECISION
US - 23.10.2025
Int.Class C12N 9/12
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
10Transferases (2.)
12transferring phosphorus containing groups, e.g. kinases (2.7)
 Appl.No 18271656 Applicant THE BROAD INSTITUTE, INC. Inventor David R. Liu

The present disclosure provides compositions and methods for prime editing with improved editing efficiency and/or reduced indel formation by inhibiting the DNA mismatch repair path way while conducting prime editing of a target site. Accordingly, the present disclosure provides a method for editing a nucleic acid molecule by prime editing that involves contacting a nucleic acid molecule with a prime editor, a pegRNA, and an inhibitor of the DNA mismatch repair pathway, thereby installing one or more modifications to the nucleic acid molecule at a target site with increased editing efficiency and/or lower indel formation. The present disclosure further provides polynucleotides for editing a DNA target site by prime editing comprising a nucleic acid sequence encoding a napDNAbp, a polymerase, and an inhibitor of the DNA mismatch repair pathway, wherein the napDNAbp and polymerase is capable in the presence of a pegRNA of installing one or more modifications in the DNA target site with increased editing efficiency and/or lower indel formation. The disclosure further provides, vectors, cells, and kits comprising the compositions and polynucleotides of the disclosure. The present disclosure also provides compositions and methods for prime editing with improved editing efficiency and/or reduced indel formation with modified prime editor fusion proteins. The disclosure further provides, vectors, cells, and kits comprising the compositions and polynucleotides of the disclosure.

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.

8.20250326783DISCOVERY OF A URIDINE RNA MODIFICATION AND A PROPOSED BIOSYNTHESIS PATHWAY
US - 23.10.2025
Int.Class C07H 19/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
19Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro derivatives thereof
02sharing nitrogen
 Appl.No 18965274 Applicant New York Institute of Technology Inventor Shenglong Zhang

The present disclosure relates to the identification of a novel nucleoside modification, referred to herein as anhydride U, present in tRNA molecules within the cell. The present disclosure further relates to the enzymatic and chemical production of anhydride U as well as methods for detecting and regulating the presence of anhydride U within the cell.

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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