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1 / 355
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/2025/186726MODULATING 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.
3.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.
4.20250275999HIGH 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.

5.WO/2025/184301RNAI AGENTS FOR DUAL INHIBITION OF EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) AND PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9), COMPOSITIONS THEREOF, AND METHODS OF USE
WO - 04.09.2025
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/US2025/017533 Applicant ARROWHEAD PHARMACEUTICALS, INC. Inventor VAN DYKE, Jonathan
Described are RNAi agents, compositions that include RNAi agents, and methods for dual inhibition of an Apolipoprotein C-III (APOC3) and Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene. The APOC3-PCSK9 RNAi agents disclosed herein inhibit the expression of an APOC3 and a PCSK9 gene. Pharmaceutical compositions that include one or more APOC3-PCSK9 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described TSLP RNAi agents to hepatic cells, in vivo, provides for inhibition of APOC3 and/or PCSK9 gene expression, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including hypertriglyceridemia and hypercholesterolemia.
6.20250277202KNOCKOUT 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).

7.20250270561FUNCTIONAL NUCLEIC ACID MOLECULES
US - 28.08.2025
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 18246046 Applicant TRANSINE THERAPEUTICS LIMITED Inventor Stefano GUSTINCICH

The present invention relates to functional nucleic acid molecules for use in upregulating OPA1 expression. The functional nucleic acid molecules typically comprise at least one target binding sequence reverse complementary to an OPA1 mRNA sequence and at least one regulatory sequence which comprises a SINE B2 element or an internal ribosome entry site (IRES) sequence. Also described are therapeutic methods of using the functional nucleic acids.

8.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).
9.20250268828Lipid 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.

10.2025527670ＡＮＧＰＴＬ３関連疾患及び障害の治療方法
JP - 22.08.2025
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 2025511500 Applicant アローヘッド　ファーマシューティカルズ　インコーポレイテッド Inventor ハビエル　サン　マルティン
ＡＮＧＰＴＬ３ ＲＮＡｉ剤を含む医薬組成物を使用して、治療を必要とするヒト被験者におけるＡＮＧＰＴＬ３遺伝子発現の低下によって部分的に媒介され得る疾患及び障害を治療する方法が記載される。ＡＮＧＰＴＬ３ ＲＮＡｉ剤を含む開示される医薬組成物は、本明細書に開示される方法に従って、治療を必要とするヒト被験者に投与された場合、トリグリセリド（ＴＧ）レベルの上昇及び／又は低密度リポタンパク質コレステロール（ＬＤＬ－Ｃ）の上昇に関連する疾患及び障害、例えば、高トリグリセリド血症（重度の高トリグリセリド血症（ＳＨＴＧ）を含む）、ホモ接合性家族性高コレステロール血症（ＨｏＦＨ）、高トリグリセリド血症誘発性膵炎、メタボリック症候群、肥満、高脂血症、混合型脂質異常症、脂質及び／又はコレステロール代謝異常、アテローム性動脈硬化、心血管疾患、II型糖尿病、冠動脈疾患、非アルコール性脂肪性肝炎、非アルコール性脂肪肝疾患、ヘテロ接合性家族性高コレステロール血症（ＨｅＦＨ）、スタチン抵抗性高コレステロール血症、他の脂質異常症、及び他の代謝関連の障害及び疾患を治療する。
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