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Analysis

1 / 148
1.20250312376COMPOSITIONS AND METHODS FOR MODULATING A GENOME IN T CELLS, INDUCED PLURIPOTENT STEM CELLS, AND RESPIRATORY EPITHELIAL CELLS
US - 09.10.2025
Int.Class A61K 35/17
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
14Blood; Artificial blood
17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
 Appl.No 18860423 Applicant FLAGSHIP PIONEERING INNOVATIONS VI, LLC Inventor Barrett Ethan Steinberg

Methods and compositions for modulating a target genome are disclosed. For instance, gene modifying systems may be used to insert a heterologous object sequence (e.g., encoding a chimeric antigen receptor) into a target cell. The target cell may be, e.g., a T cell, induced pluripotent stem cell, or respiratory epithelial cell.

2.20250304994Circular RNA Compositions and Methods
US - 02.10.2025
Int.Class C12N 15/85
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
 Appl.No 19234626 Applicant Orna Therapeutics, Inc. Inventor Robert Alexander Wesselhoeft

Circular RNA, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises intron segments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, the disclosed methods and constructs result in improved translation when compared to existing RNA approaches. In some embodiments, the disclosed methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

3.20250302987Targeted Lipid Nanoparticles
US - 02.10.2025
Int.Class A61K 47/69
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
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
 Appl.No 19083329 Applicant Zitra Medicines Pty Ltd Inventor Denzil Furtado

The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention.

4.20250305002RECRUITMENT IN TRANS OF GENE EDITING SYSTEM COMPONENTS
US - 02.10.2025
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
 Appl.No 18690120 Applicant Flagship Pioneering Innovation VI, LLC Inventor Anne Helen Bothmer

The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject.

5.1020250141840혈색소병증의 치료를 위한 조성물 및 방법
KR - 29.09.2025
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 1020257030429 Applicant 노파르티스 아게 Inventor 보이타노, 앤소니 에드워드
본 발명은 혈색소병증(hemoglobinopathies) 치료를 위한 게놈 편집 시스템, 시약 및 방법에 관한 것이다.
6.2025138725分泌タンパク質の産生のための修飾ポリヌクレオチド
JP - 25.09.2025
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 2025106096 Applicant モデルナティエックス　インコーポレイテッド Inventor バンセル、ステファヌ
【課題】目的とするポリペプチド（例えば、修飾ｍＲＮＡまたはｍｍＲＮＡ）をコードし、当技術分野における課題のうちの１つ以上を回避する構造的および／または化学的特徴、例えば、構造的および機能的完全性を保持し、発現の閾値を克服し、発現速度、半減期、および／またはタンパク質濃度を改善し、タンパク質局在化を最適化し、かつ免疫応答等の有害な生物応答および／または分解経路を回避しながら、核酸系治療薬の製剤化および送達の最適化に有用な特徴を有する核酸系化合物またはポリヌクレオチドを提供する。
【解決手段】ポリヌクレオチド、一次構築物、および修飾ｍＲＮＡ分子（ｍｍＲＮＡ）の組成、これらの設計、調製、製造、および／または製剤化の方法、プロセス、キット、ならびにデバイスが提供される。
【選択図】なし
7.20250295788SILOXANE-BASED LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY
US - 25.09.2025
Int.Class A61K 47/54
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
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
 Appl.No 18862766 Applicant The Trustees of the University of Pennsylvania Inventor Michael J. Mitchell

The present disclosure relates, in part, to siloxane-based lipids or lipidoids, lipid nanoparticles (LNPs) comprising the same, and pharmaceutical compositions thereof. In certain embodiments, the LNPs of the present disclosure selectively target cells (e.g., hepatocytes, epithelial cells, endothelial cells, and immune cells, inter alia) and/or organs of interest (e.g., liver, spleen, heart, and lungs, inter alia). In another aspect, the present disclosure relates to methods of treating, preventing, and/or ameliorating one or more diseases and/or disorders in a subject, the method comprising administering to the subject at least one LNP of the present disclosure and/or at least one pharmaceutical composition of the present disclosure.

8.2025138811合成ＲＩＧ－Ｉ様受容体アゴニスト
JP - 25.09.2025
Int.Class C12N 7/01
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
7Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
01Viruses, e.g. bacteriophages, modified by introduction of foreign genetic material
 Appl.No 2025112246 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を提供すること。
【解決手段】いくつかの態様では、本開示は、（ａ）ウイルス様粒子と、（ｂ）ＲＩＧ－Ｉ様受容体（ＲＬＲ）に特異的に結合する少なくとも１つの合成ＲＩＧ－Ｉ様受容体（ＲＬＲ）アゴニストであって、１０～１００ヌクレオチドの長さのリボ核酸（ＲＮＡ）を含むＲＬＲアゴニストと、を含む、組成物であって、ＲＮＡの最も５’側のヌクレオチドが５’二リン酸もしくは三リン酸部分、またはその誘導体もしくは類似体を含み、少なくとも１つのＲＬＲアゴニストがウイルス様粒子にパッケージングされている、組成物を提供する。
【選択図】なし
9.20250295687TREM COMPOSITIONS AND METHODS OF USE FOR TREATING PROLIFERATIVE DISORDERS
US - 25.09.2025
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 18864142 Applicant Flagship Pioneering Innovations VI, LLC Inventor Theonie Anastassiadis

The disclosure relates generally to tRNA-based effector molecules (TREMs) and compositions thereof useful for the treatment or prevention of a proliferative disease or disorder (e.g., a cancer) in a subject.

10.20250295688COMPOSITIONS AND METHODS OF MODULATING XANTHINE DEHYDROGENASE
US - 25.09.2025
Int.Class A61K 31/713
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
713Double-stranded nucleic acids or oligonucleotides
 Appl.No 18572698 Applicant HORIZON THERAPEUTICS IRELAND DAC Inventor Duncan BROWN

Compositions, methods for making and using polynucleotide inhibitors modulating xanthine dehydrogenase expression or activity are provided.

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