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Analysis

1 / 110
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/2025/217126POLYNUCLEOTIDES, 3' CHAIN TERMINATING TAILS, LIGATION AND USES THEREOF
WO - 16.10.2025
Int.Class C12N 15/11
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
 Appl.No PCT/US2025/023599 Applicant CONVERGENCE BIO, INC. Inventor WANG, Chong
The present disclosure provides constructs and methods for RNA ligation of 3' chain terminating tails and uses for improving nucleic acid stability and protein expression.
3.20250320491CRISPR-CPF1-RELATED METHODS, COMPOSITIONS AND COMPONENTS FOR CANCER IMMUNOTHERAPY
US - 16.10.2025
Int.Class C12N 15/11
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
 Appl.No 19200416 Applicant EDITAS MEDICINE, INC. Inventor Gordon Grant Welstead

CRISPR/Cpf1-related compositions and methods for treatment of cancer.

4.20250319160USE OF IFN-LAMBDA MRNA FOR TREATING VIRAL INFECTIONS
US - 16.10.2025
Int.Class A61K 38/21
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
19Cytokines; Lymphokines; Interferons
21Interferons
 Appl.No 19248327 Applicant ethris GmbH Inventor Carsten Rudolph

The present invention relates to pharmaceutical compositions comprising an mRNA encoding an IFN-λ polypeptide for use in treating a viral-induced disorder, preferably a viral-induced respiratory disorder, such as COVID-19.

5.WO/2025/217135RNA LIGATION METHODS AND PRODUCTS
WO - 16.10.2025
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 PCT/US2025/023609 Applicant CONVERGENCE BIO, INC. Inventor WANG, Chong
The present disclosure provides products of and methods comprising RNA ligation. In some embodiments, the method comprises contacting an RNA ligase enzyme with (i) an acceptor RNA polynucleotide comprising a 3' hydroxyl group and (ii) a donor RNA polynucleotide.
6.WO/2025/212831METHODS OF INDUCING AN ANTI-HSV B-CELL IMMUNE RESPONSE
WO - 09.10.2025
Int.Class A61K 39/245
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
245Herpetoviridae, e.g. herpes simplex virus
 Appl.No PCT/US2025/022857 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor FRIEDMAN, Harvey
The present disclosure provides a combination comprising ribonucleotide sequences that encode herpes simplex virus (HSV) glycoproteins, including those involved in virus entry and immune evasion and related methods of treating, suppressing, inhibiting, or preventing an HSV infection. The present disclosure further provides a related formulation comprising ribonucleotide sequences that encode HSV glycoproteins, wherein one or more of the ribonucleotides are fully or partially encapsulated within a lipid nanoparticle, wherein the lipid nanoparticle comprises (a) a polymer-conjugated lipid; (b) a cationic lipid; and (c) one or more neutral lipids. Finally, the present disclosure provides methods of inducing an HSV glycoprotein-specific B-cell response in a subject comprising administering to the subject a composition comprising the HSV glycoprotein.
7.WO/2025/212851mRNA COMPOSITIONS AND USES THEREOF IN VARICELLA ZOSTER VIRUS VACCINES
WO - 09.10.2025
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
 Appl.No PCT/US2025/022899 Applicant ORBITAL THERAPEUTICS, INC. Inventor BESIN, Gilles
The present disclosure relates to mRNA sequences encoding an antigen from varicella zoster virus. The mRNA sequences described herein are used as vaccines and compositions for preventing or treating diseases resulting from varicella zoster virus, including shingles. Methods for formulating and preparing the vaccines are also described.
8.20250302987Targeted Lipid Nanoparticles
US - 02.10.2025
Int.Class A61K 47/69
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
 Appl.No 19083329 Applicant Zitra Medicines Pty Ltd Inventor Denzil Furtado

The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention.

9.1020250141840혈색소병증의 치료를 위한 조성물 및 방법
KR - 29.09.2025
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 1020257030429 Applicant 노파르티스 아게 Inventor 보이타노, 앤소니 에드워드
본 발명은 혈색소병증(hemoglobinopathies) 치료를 위한 게놈 편집 시스템, 시약 및 방법에 관한 것이다.
10.WO/2025/199339MECP2-TARGETING ENGINEERED GUIDE RNAS AND POLYNUCLEOTIDES
WO - 25.09.2025
Int.Class C12N 15/11
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
 Appl.No PCT/US2025/020730 Applicant SHAPE THERAPEUTICS INC. Inventor SAVVA, Yiannis
Disclosed herein are engineered guide RNAs and compositions comprising the same for treatment of diseases or conditions in a subject. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
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