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Analysis

1 / 194
1.WO/2025/186726MODULATING EXPRESSION OF AGT (ANGIOTENSINOGEN) GENE
WO - 11.09.2025
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 PCT/IB2025/052358 Applicant CRISPR THERAPEUTICS AG Inventor DEQUEANT, Mary-Lee
The present disclosure relates to methods, compositions and kits for treating conditions that are related to the modulation of expression of angiotensinogen (AGT) gene by gene editing.
2.WO/2025/189068MRNA-LIPID NANOPARTICLE IMMUNE MODULATORS AGAINST ALLERGIC AND INFLAMMATORY DISEASES
WO - 11.09.2025
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/US2025/018859 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor WEISSMAN, Drew
Provided are compositions comprising mRNA molecules encoding antigens, such as allergens and autoantigens, and methods of use thereof, optionally in combination with an mTOR inhibitor, to prevent or reduce allergic responses or to promote tolerance to the encoded antigens.
3.WO/2025/186719IMMUNOGENIC COMPOSITIONS AND USES THEREOF
WO - 11.09.2025
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/IB2025/052342 Applicant PFIZER INC. Inventor ALLEN, Pirada Suphaphiphat
The present disclosure relates to compositions comprising RNA molecules encoding an antigen derived from influenza, wherein the RNA may be formulated in a lipid nanoparticle (LNP), and wherein the composition further includes a polypeptide antigen derived from respiratory syncytial virus (RSV) and/or RNA molecules encoding an antigen derived from RSV, wherein the RSV RNA may be formulated in an LNP. The present disclosure further relates to the use of the RNA molecules, RNA-LNPs and compositions for the prevention of influenza and RSV infection-induced illness.
4.20250277202KNOCKOUT OF A MUTANT ALLELE OF AN ELANE GENE
US - 04.09.2025
Int.Class C12N 9/64
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.)
48acting on peptide bonds, e.g. thromboplastin, leucine aminopeptidase (3.4)
50Proteinases
64derived from animal tissue, e.g. rennin
 Appl.No 19059992 Applicant EmendoBio Inc. Inventor David BARAM

Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN).

5.20250277256NUCLEIC ACID DETECTION AND ANALYSIS SYSTEMS
US - 04.09.2025
Int.Class C12Q 1/6827
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6813Hybridisation assays
6827for detection of mutation or polymorphism
 Appl.No 18280164 Applicant The Johns Hopkins University Inventor Taekjip Ha

In one embodiment, methods for detecting a specific nucleic acid sequence in a genome are provided that may include: a) inducing a nick in genomic nucleic acid sequences by a gene editing complex; b) denaturing the genomic nucleic acid sequences by contacting the genomic nucleic acid sequences with a helicase enzyme at the nicked genomic nucleic acid sequences; c) contacting the denatured genome with a detectably labeled probe, wherein the detectably labeled probe is complementary to the specific nucleic acid sequence of interest; and, d) detecting the specific nucleic acid sequence of interest.

6.20250277223METHODS OF TREATING OR PREVENTING AUTOIMMUNE DISEASES
US - 04.09.2025
Int.Class C12N 15/62
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
62DNA sequences coding for fusion proteins
 Appl.No 18563288 Applicant IMCYSE SA Inventor Geoffrey GLOIRE

The invention relates to non-immunogenic mRNA encoding immunogenic peptides comprising a T-cell epitope and an oxidoreductase motifs, and their use in the treatment and/or prevention of e.g. type-1 diabetes (T1D), multiple sclerosis (MS), neuromyelitis optica (NMO), or rheumatoid arthritis (RA) in subjects.

7.20250268828Lipid Nanoparticle Compositions for Delivering Circular Polynucleotides
US - 28.08.2025
Int.Class A61K 9/1272
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
1272comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
 Appl.No 18697259 Applicant Orna Therapeutics, Inc. Inventor Allen T. Horhota

Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.

8.20250262324Circular RNA Compositions
US - 21.08.2025
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
 Appl.No 19200761 Applicant Orna Therapeutics, Inc. Inventor Amy M. Becker

Provided herein are circular RNA constructs comprising an IRES, and at least one expression sequence encoding binding molecule, compositions thereof, and methods of treatment, including for cancer and autoimmune disease. In particular, circular RNA comprising an IRES and a CD19 binder, a HER2 binder, or a BCMA binder are provided, optionally formulated with a delivery vehicle. Precursor polynucleotides comprising an IRES, and at least one expression sequence encoding a CAR construct are also described herein.

9.WO/2025/175176SYNTHETIC NUCLEIC ACIDS AND METHODS OF USING THE SAME FOR IMPROVED POLYPEPTIDE PRODUCTION
WO - 21.08.2025
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/US2025/016041 Applicant MODERNATX, INC. Inventor MIRACCO, Edward, John
This disclosure relates to synthetic nucleic acid molecules, such as synthetic mRNA molecules, containing chemical modifications that improve nucleic acid stability while simultaneously preserving the ability of the nucleic acid to be processed by endogenous translational machinery and to furnish protein product.
10.20250263691POTENCY ASSAY FOR THERAPEUTIC POTENTIAL OF CODING NUCLEIC ACID
US - 21.08.2025
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 18256510 Applicant BioNTech SE Inventor Heinrich HAAS

The invention provides potency assays for measuring, determining, identifying, quantifying, confirming, and/or validating the therapeutic potential of nucleic acid such as RNA encoding a pharmaceutically active peptide or polypeptide. The potency assays may be performed with nucleic acid such as RNA encoding various types of peptides or polypeptides, including pharmaceutically active peptides or polypeptides comprising one or more antigens or one or more epitopes. Nucleic acid such as RNA having therapeutic potential may be useful in downstream clinical applications, e.g., for eliciting an immune response against one or more antigens or one or more epitopes encoded by the nucleic acid in a subject which immune response may be therapeutic or partially or fully protective. Thus, the nucleic having therapeutic potential may be useful for vaccination.

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