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Analysis

1.20250275999HIGH 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.

2.20250277202KNOCKOUT 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).

3.WO/2025/184356VACCINES AND COMPOSITIONS AGAINST GAMMA HERPESVIRUSES
WO 04.09.2025
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/US2025/017607 Applicant HDT BIO CORP. Inventor ERASMUS, Jesse
The disclosure provides compositions, methods of treatment, and methods of making and using compositions to deliver a nucleic acid to a subject. Compositions described herein include lipid carriers, optionally including an inorganic particle, capable of admixing with nucleic acids. Methods of using these compositions as a vaccine for treatment of a gamma herpesvirus or a cancer are also provided.
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).
5.20250268828Lipid 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.

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.

9.20250263765METHODS FOR PRODUCING NUCLEIC ACIDS
US 21.08.2025
Int.Class C12P 19/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
Appl.No 18856566 Applicant PFIZER INC. Inventor Amer Al-Lozi

Described are methods for producing RNA molecules in an in vitro transcription reaction. Also described are methods for producing an mRNA molecule from a circular double-stranded DNA template using an in vitro transcription reaction system wherein the in vitro transcription reaction system lacks a polyamine. Also described are in vitro transcription reaction systems comprising enzymatic 5′ capping and oligo (dT) purification.

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.