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Analysis

1 / 42
1.20250277202KNOCKOUT 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).

2.20250255897P-ETHOXY NUCLEIC ACIDS FOR LIPOSOMAL FORMULATION
US - 14.08.2025
Int.Class A61K 31/7125
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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
 Appl.No 18890384 Applicant Bio-Path Holdings, Inc. Inventor Peter NIELSEN

Provided herein are therapeutic oligonucleotides that comprise at least one p-ethoxy backbone linkage but no more than 80% p-ethoxy backbone linkages. Provided herein are improved delivery systems for therapeutic oligonucleotides comprising a liposome that comprises neutral phospholipids and a p-ethoxy oligonucleotide that is entrapped in the liposome.

3.120457204用于转录因子4（TCF4）重复扩增切除的组合物和方法
CN - 08.08.2025
Int.Class C12N 15/10
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
10Processes for the isolation, preparation or purification of DNA or RNA
 Appl.No 202380089726.4 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
包含具有17‑50个连续核苷酸的向导序列部分的RNA分子，所述17‑50个连续核苷酸含有SEQ ID NO：1‑2325中任一项所示序列的核苷酸及其组合物、方法和用途。
4.2025524813ＡＰＬＰ２セーフハーバー部位におけるノックイン戦略
JP - 01.08.2025
Int.Class C12N 5/0789
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
0789Stem cells; Multipotent progenitor cells
 Appl.No 2025501882 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１～１５９６４１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。
5.2025523975ＡＮＧＰＴＬ３の両アレルノックアウト
JP - 25.07.2025
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 2025502919 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
１７～５０ヌクレオチドからなり、配列番号１～２０３４７のいずれか１つに示す配列中の１７～２２ヌクレオチドを含有するガイド配列部分を含むＲＮＡ分子並びにその組成物、方法及び使用。
6.120344666CIITA双等位基因敲除
CN - 18.07.2025
Int.Class C12N 15/74
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
74Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
 Appl.No 202380075638.9 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
一种包含RNA分子的组合物，及其方法和用途，所述RNA分子包含具有17‑50个连续核苷酸的向导序列部分，所述17‑50个连续核苷酸含有SEQ ID NO:1‑34194中任一项所示序列的核苷酸。
7.120265768PDCD1双等位基因敲除
CN - 04.07.2025
Int.Class C12N 15/10
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
10Processes for the isolation, preparation or purification of DNA or RNA
 Appl.No 202380075648.2 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
一种包含RNA分子的组合物，及其方法和用途，所述RNA分子包含具有含有SEQ ID NO:1‑6215中任一个所示序列中的核苷酸的17‑50个连续核苷酸的向导序列部分，或使细胞中的程序性细胞死亡蛋白1(PDCD1)基因的等位基因失活，使得细胞具有改善的活性、保留和/或扩增品质，以用于过继性癌症免疫治疗。
8.120225689HLA-E双等位基因敲除
CN - 27.06.2025
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 202380079148.6 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
一种包含RNA分子的组合物，及其方法和用途，所述RNA分子包含具有17‑50个连续核苷酸的向导序列部分，所述17‑50个连续核苷酸含有SEQ ID NO:1‑7830中任一项所示序列的核苷酸。
9.120202302CTLA4双等位基因敲除
CN - 24.06.2025
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 202380079156.0 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
一种包含RNA分子的组合物，及其方法和用途所述RNA分子包含具有17‑50个连续核苷酸的向导序列部分，所述17‑50个连续核苷酸含有SEQ ID NO:1‑4886中任一项所示序列的核苷酸。
10.120187858CISH双等位基因敲除
CN - 20.06.2025
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 202380075646.3 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
一种包含RNA分子的组合物，及其方法和用途所述RNA分子包含具有17‑50个连续核苷酸的向导序列部分，所述17‑50个连续核苷酸含有SEQ ID NO:1‑6833中任一项所示序列的核苷酸。
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