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Analysis

1 / 179
1.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.
2.20250275999HIGH 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.

3.20250277202KNOCKOUT 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).

4.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).
5.2025528112ＲＮＡコードウイルス様粒子及びその使用
JP - 26.08.2025
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 2025507064 Applicant セキラス　インコーポレイテッド Inventor ベディ，スクマニ
本開示は、インフルエンザウイルスのウイルス様粒子（ＶＬＰ）形成エレメントをコードする１つ以上のリボ核酸（ＲＮＡ）を含む組成物に関する。本開示はさらに、組成物の使用を提供する。
【選択図】なし
6.2025527583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。
7.20250263678COMPOSITIONS AND METHODS FOR DELIVERY OF RNA
US - 21.08.2025
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 18270179 Applicant REJUVENATION TECHNOLOGIES, INC. Inventor John RAMUNAS

The disclosure relates to compositions and methods for the treatment of fibrotic diseases and disorders and/or liver diseases and disorders, with one or more synthetic messenger ribonucleic acids (mRNAs) encoding telomerase reverse transcriptase (TERT).

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.
10.20250263794RNA AND DNA ANALYSIS USING ENGINEERED SURFACES
US - 21.08.2025
Int.Class C12Q 1/6883
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
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6883for diseases caused by alterations of genetic material
 Appl.No 19184759 Applicant ALIDA BIOSCIENCES, INC. Inventor Gudrun STENGEL

Provided herein are compositions and methods for the multiplexed profiling of RNA and DNA modifications across transcriptomes and genomes, respectively. The methods combine molecular recognition of non-canonical features (e.g., base modifications, backbone modifications, lesions, and/or structural elements) of a target nucleic acid with a step of writing the information from this recognition event into the neighboring genetic sequence of the target nucleic acid using a barcode. The resultant barcoded nucleic acids are then converted into sequencing libraries and read by DNA/RNA sequencing methods. This step reveals the sequence of the barcode, which is correlated with the non-canonical feature in the target nucleic acid(s). The high throughput profiling methods described herein allow for identification and/or localization of one or more modifications in a target nucleic acid. The methods also allow for identification of the nature and location of several or all DNA/RNA modifications in parallel.

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