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Analysis

1 / 169
1.WO/2025/187767ANTISENSE OLIGONUCLEOTIDE FOR SUPPRESSING PROLIFERATION OF CORONAVIRUS
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/JP2025/008156 Applicant DAIICHI SANKYO COMPANY, LIMITED Inventor ONISHI Yoshiyuki
One problem to be addressed by the present invention is to provide a drug which is capable of suppressing the proliferation of SARS-CoV-2 with high efficiency. Another problem to be addressed by the present invention is to provide a drug which exhibits an inhibitory effect on a wide range of coronaviruses and thereby can immediately respond to the pandemic of unknown SARS-CoV-2 mutants and new types of coronaviruses which could happen in the future.　Provided is an oligonucleotide or a pharmaceutically acceptable salt thereof, wherein: the oligonucleotide comprises an oligonucleotide that is composed of 17-30 nucleotides and comprises a nucleotide sequence and is complementary to a region lying between a nucleotide located at position-13520 to a nucleotide located at position-13550 in SARS-CoV-2 RNA genome comprising the nucleotide sequence represented by SEQ ID NO: 1; and the oligonucleotide is a mixmer and is capable of inhibiting the viral proliferation of coronaviruses, wherein the 5'-end and/or the 3'-end of the oligonucleotide may be chemically modified. Also provided is a drug comprising the oligonucleotide or a pharmaceutically acceptable salt thereof.
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.
3.20250275999HIGH PURITY RNA COMPOSITIONS AND METHODS FOR PREPARATION THEREOF
US - 04.09.2025
Int.Class A61K 31/7105
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
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
 Appl.No 19057378 Applicant ModernaTX, Inc. Inventor Stephen Hoge

The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

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.WO/2025/179029MODIFIED IMMUNE CELLS WITH A GENETIC MODULATION
WO - 28.08.2025
Int.Class C12N 5/0783
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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
078Cells from blood or from the immune system
0783T cells; NK cells; Progenitors of T or NK cells
 Appl.No PCT/US2025/016616 Applicant THE CLEVELAND CLINIC FOUNDATION Inventor MELENHORST, Jan Joseph
Provided herein are systems, kits, compositions, and methods for treating a subject with cancer or other disease with a CAR T-cell (or other immune cell) that has at least one endogenous gene that is modulated, and/or wherein mRNA from the gene is over-expressed or under-expressed, or that has exogenous mRNA that is expressed (e.g., expressed by an expression vector, or comprising modified bases). In certain embodiments, the endogenous gene, or mRNA therefrom, is inhibited or silenced and is selected from Table 2 (e.g., selected from PCNX1, PDCD10, MYC, ASXL1, RPTOR, BCAP31, ANKRD11, TCF3, IQCB1, CTLA4, ZZEF1, SRCAP, CARD8, DNMT1 and HSF2). In some embodiments, the endogenous gene, or mRNA therefrom, or mRNA in an expression vector, is over-expressed and is selected from Table 1 (e.g., NOSIP, FADD, RUNX1, AQR, ATE1, ATP9B, LSM2, CD3D, WNK1, EIF2B3, and TAL2).
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.

8.2025527583脂質ナノ粒子の組成物
JP - 22.08.2025
Int.Class A61K 47/18
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
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
16containing nitrogen
18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
 Appl.No 2025509162 Applicant スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド Inventor ユハン・ジアン
本明細書では、治療目的または予防目的での治療用ペイロード（例えば、ｍＲＮＡ、ｓｉＲＮＡ、ＤＮＡ）の送達に使用され得る、カチオン性脂質、ステロイド、ポリマーコンジュゲート脂質、及び０．５モルパーセント以下のリン脂質を含む、脂質ナノ粒子組成物が提供される。
9.20250262324Circular 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.

10.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.
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