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Analysis

1 / 43
1.2025531296ＰＤＣＤ１の両アレルノックアウト
JP - 19.09.2025
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 2025516217 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～６２１５のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
2.2025131687アンチコドンループを延長した合成転移ＲＮＡ
JP - 09.09.2025
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 2025092679 Applicant アークタラス　テラピュウティクス、インク Inventor イグナトヴァ、ゾヤ
【課題】フレームシフト変異に関連する遺伝子疾患、例えば神経線維腫症を治療するためのフレームシフト変異抑制因子を提供する。
【解決手段】４ヌクレオチドアンチコドンまたは５ヌクレオチドアンチコドンを有する延長アンチコドンループを含むことを特徴とした合成転移ＲＮＡであって、コード配列に変異を有するｍＲＮＡ上のコドン、及び後続のコドン（－１フレームシフトの場合）または先行するコドン（＋１フレームシフトの場合）の残りの部分と塩基対合し、担持されるアミノ酸が成長アミノ酸鎖に組み込まれ、リーディングフレームが修正されるフレームシフト変異抑制因子。
【選択図】図１
3.2025529546ＣＩＳＨの両アレルノックアウト
JP - 04.09.2025
Int.Class C12N 15/12
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
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
12Genes encoding animal proteins
 Appl.No 2025516206 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～６８３３のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
4.2025529547ＣＩＩＴＡの両アレルノックアウト
JP - 04.09.2025
Int.Class C12N 15/55
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
52Genes encoding for enzymes or proenzymes
55Hydrolases (3)
 Appl.No 2025516207 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～３４１９４のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
5.20250277202KNOCKOUT 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).

6.2025529223Ｂ２Ｍセーフハーバー部位でのノックイン戦略
JP - 04.09.2025
Int.Class A61K 38/17
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
 Appl.No 2025513061 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～２８９９８のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子及び組成物、方法及びその使用。
7.2025529544ＣＴＬＡ４の両アレルノックアウト
JP - 04.09.2025
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 2025516204 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～４８８６のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
8.2025529545ＦＡＳＬＧの両アレルノックアウト
JP - 04.09.2025
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 2025516205 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～１１１２１のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
9.2025529540ＴＥＴ２の両アレルノックアウト
JP - 04.09.2025
Int.Class C12N 9/02
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
02Oxidoreductases (1.), e.g. luciferase
 Appl.No 2025516187 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～３７４２１のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
10.2025529542ＨＬＡ－Ｅの両アレルノックアウト
JP - 04.09.2025
Int.Class C12N 15/09
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
 Appl.No 2025516199 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～７８３０のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子を含有する組成物、方法及びその使用。
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