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Analysis

1 / 183
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).

2.20250270575COMPOSITIONS AND METHODS FOR INDOOR AIR REMEDIATION
US - 28.08.2025
Int.Class C12N 15/82
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
82for plant cells
 Appl.No 18284959 Applicant Neoplants SAS Inventor Patrick Torbey

The present disclosure provides compositions, methods of use, and methods of creation for a population of transgenic plants derived from plant cells transformed with recombinant DNA for expression of heterologous proteins. In particular, the present disclosure provides compositions comprising indoor ornamental plants suited for the removal of volatile organic compounds such as formaldehyde, benzene, toluene, ethylbenzene and/or xylene from air. Also disclosed are transgenic seeds for growing a transgenic plant having the recombinant DNA in its genome and exhibiting enhanced VOC removal from air. Also disclosed are methods for generating seed and plants based on the transgenic events. Also disclosed are microbes selected for during directed evolution to have enhanced VOC removal from air capabilities. Also disclosed are methods and compositions for generating plant-microbiome pairings for enhanced VOC removal from air.

3.20250262235OLIGONUCLEOTIDE COMPOSITIONS AND METHODS RELATING THERETO
US - 21.08.2025
Int.Class A61K 31/712
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
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
 Appl.No 18856553 Applicant WAVE LIFE SCIENCES LTD. Inventor Genliang Lu

The present disclosure features useful oligonucleotide compositions and methods related thereto. The present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g. modifications of sugar, base and/or internucleotide linkages) or patterns thereof, can have a significant impact on oligonucleotide properties and activities. The present disclosure also provides methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

4.120504661ALKBH8小分子抑制剂及其制备方法和用途
CN - 19.08.2025
Int.Class C07D 401/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
401Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
02containing two hetero rings
04directly linked by a ring-member-to-ring- member bond
 Appl.No 202510962686.0 Applicant 复旦大学 Inventor 刘苹羽
本发明涉及ALKBH8小分子抑制剂及其制备方法和用途。具体地，本发明公开了一种式I所示化合物的ALKBH8小分子抑制剂，本发明还提供了所述化合物的制备方法，并通过实验证明了ALKBH8是一个潜在的衰老抑制剂新靶点，还公开了所述化合物在预防和/或治疗衰老相关疾病方面的用途。#imgabs0# (I)。
5.20250257359MUSK-TARGETING OLIGONUCLEOTIDES
US - 14.08.2025
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 18730084 Applicant Bolden Therapeutics, Inc. Inventor John Page

The present disclosure provides, among other things, MuSK-targeting oligonucleotide compositions that alter the splicing of MuSK transcripts through exon skipping, and methods of treating diseases with said compositions.

6.20250255897P-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.

7.WO/2025/167868MODIFIED NUCLEIC ACID AND USE THEREOF
WO - 14.08.2025
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 PCT/CN2025/075700 Applicant SHENZHEN SHENXIN BIOTECHNOLOGY CO., LTD. Inventor LI, Linxian
The present invention relates to a modified nucleic acid and use thereof. The modified nucleic acid is a non-natural nucleic acid, and comprises a 3'-UTR and one or more microRNA binding sites located downstream of the 3'-UTR.
8.1020250122469암 및 대사 질환의 치료를 위한 조성물 및 방법
KR - 13.08.2025
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 1020257020095 Applicant 프레첼 테라퓨틱스, 인크. Inventor 시, 용홍
다양한 질환, 예컨대 암 및 대사 장애의 치료에서 올리고뉴클레오타이드 조성물 및 이의 용도가 기재된다.
9.120456910寡核苷酸组合物及其方法
CN - 08.08.2025
Int.Class A61K 31/712
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
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
 Appl.No 202380067032.0 Applicant 艾美力克斯制药股份有限公司 Inventor J·B·科恩
除其它之外，本公开还提供了靶向SARM1的寡核苷酸及其组合物。在一些实施例中，本公开提供了用于预防或治疗各种病状、病症或疾病的方法。
10.WO/2025/162939NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF GPR146 IN A CELL
WO - 07.08.2025
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 PCT/EP2025/052139 Applicant SILENCE THERAPEUTICS GMBH Inventor MORRISON, Eliot
The invention relates to double-stranded nucleic acid molecules that interfere with or inhibit G Protein-Coupled Receptor 146 (GPR146) gene expression. It further relates to therapeutic uses of such inhibition such as for the treatment of GPR146-mediated diseases, disorders or syndromes, such as hyperlipidaemia, hypercholesterolaemia, Heterozygous familial hypercholesterolaemia (HeFH), Homozygous familial hypercholesterolaemia (HoFH), autosomal recessive hypercholesterolaemia, atherosclerosis, and/or atherosclerotic cardiovascular disease.
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