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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.

2.20250277256NUCLEIC 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.

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).
5.2025527583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。
6.20250262324Circular 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.

7.20250263765METHODS 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.

8.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.
9.2025121964脂質封入RNAナノ粒子の製造方法
JP 20.08.2025
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 2025076678 Applicant アークトゥラス・セラピューティクス・インコーポレイテッド Inventor カルマリ,プリヤ
【課題】粒子径および均一性を最適化し、大量生産を可能とする、脂質封入RNAナノ粒子を形成する方法を提供する。
【解決手段】a)RNAを含む水溶液を、0.1インチ~0.132インチの内径(ID)を有する第1チューブに流す工程;b)脂質を含むエタノール溶液を、0.005インチ~0.02インチのIDを有する第2チューブに、第1チューブを通る水溶液の1/3の流速にて流す工程であって、脂質がカチオン性脂質を含む工程;およびc)エタノール溶液および水溶液を、第1チューブに垂直に接合された第2チューブからなる混合モジュールへ流すことにより、エタノール溶液を水溶液と混合する工程を含む、脂質封入RNAナノ粒子を製造する方法であって、混合工程が、約10v/v%~75v/v%エタノール中のRNAおよび脂質の乱流を含む第1チューブ中を流れる排出溶液を生じ、脂質封入RNAナノ粒子が二層構造を有する、方法。
【選択図】なし
10.2025120300合成RIG-I様受容体アゴニスト
JP 15.08.2025
Int.Class C12N 15/117
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
117Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
Appl.No 2025094028 Applicant チェックメイト ファーマシューティカルズ, インコーポレイテッド Inventor アーサー エム. クリーグ
【課題】合成RIG-I様受容体アゴニストの提供。
【解決手段】本開示は、特に、RIG-I様受容体(RLR)に結合し、これをアゴナイズするRNA分子(例えば、RNAヘアピンアゴニスト)、およびある障害(例えば、癌)を治療するか、またはその1つ以上の症状を改良する方法における上述の分子の使用に関する。いくつかの実施形態において、本開示のRLRアゴニストは、以下:(i)GTリピートモチーフ、(ii)GAリピートモチーフ、(iii)AUCGリピートモチーフ、(iv)AUリピートモチーフ、(v)ジピリミジンモチーフ、(vi)ジプリンモチーフ、(vii)ピリミジントリプレットモチーフ、(viii)プリントリプレットモチーフ、(ix)回文配列モチーフ、および(x)(i)~(ix)のいずれかの組み合わせからなる群から選択される配列モチーフを含む。
【選択図】なし