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1.WO/2025/132845GALNAC PHOSPHORAMIDITES AND THEIR USES
WO 26.06.2025
Int.Class C07H 15/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
02Acyclic radicals, not substituted by cyclic structures
04attached to an oxygen atom of a saccharide radical
Appl.No PCT/EP2024/087484 Applicant SILENCE THERAPEUTICS GMBH Inventor BETHGE, Lucas
The invention generally relates to the field of phosphoramidite derivatives. In particular, the invention relates to N-Acetylgalactosamine phosphoramidite molecules and to conjugates of nucleic acid molecules with N-Acetylgalactosamine phosphoramidite containing molecules, and more specifically, to variants of N-Acetylgalactosamine phosphoramidite molecules and to conjugates of nucleic acid molecules with said variants of N-Acetylgalactosamine phosphoramidite containing molecules. Further, the invention provides associated methods of producing and using these molecules, in particular, in the field of medicine. More precisely, the N-Acetylgalactosamine derivatives of the present invention bear in the position 6' a protecting group selected from TMTr, DMTr, MMTr, Fmoc, Px, DMPx.
2.WO/2025/125630METHOD FOR BIOPROTAC DESIGN
WO 19.06.2025
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 PCT/EP2024/086358 Applicant MEDIMMUNE LIMITED Inventor HUNT, James
The present disclosure provides methods for identifying bioPROTACs capable of inducing degradation of target proteins. The disclosure further provides BIOPROTACs which have been generated according to these methods, including bioPROTACs capable of specifically causing degradation of c-Myc or K-Ras.
3.WO/2025/128846OLIGONUCLEOTIDES TARGETING SAMHD1
WO 19.06.2025
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/US2024/059808 Applicant PRETZEL THERAPEUTICS, INC. Inventor XIE, Xie
Oligonucleotides and their use in modulating (e.g., inhibiting) SAMHD1 expression are provided. Compositions and pharmaceutical compositions comprising such oligonucleotides are also provided.
4.2025518487オリゴヌクレオチド組成物及びその方法
JP 17.06.2025
Int.Class C12Q 1/68
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
Appl.No 2024566322 Applicant アミーリクス・ファーマシューティカルズ・インコーポレイテッド Inventor コーエン、ジョシュア・バリー
この開示は、特に、糖残基の2’位で修飾された、カルパイン2を標的とするオリゴヌクレオチド及びその組成物を提供する。一部の態様では、この開示は、神経変性状態を含むさまざまなの状態、障害又は疾患を予防又は治療する方法を提供する。
5.WO/2025/119162METAPNEUMOVIRUS (MPV) VACCINE
WO 12.06.2025
Int.Class C12N 15/45
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
31Genes encoding microbial proteins, e.g. enterotoxins
33Genes encoding viral proteins
40Proteins from RNA viruses, e.g. flaviviruses
45Paramyxoviridae, e.g. measles virus, mumps virus, Newcastle disease virus, canine distemper virus, rinderpest virus, respiratory syncytial viruses
Appl.No PCT/CN2024/136380 Applicant SHENZHEN SHENXIN BIOTECHNOLOGY CO., LTD. Inventor LI, Linxian
Provided is a metapneumovirus (MPV) vaccine. Specifically, provided is a nucleic acid comprising a polynucleotide for encoding a mutant of an MPV F protein, compared with a wild-type MPV F protein, the mutant comprises a disulfide bond mutation.
6.120112634B2M安全港位点的敲入策略
CN 06.06.2025
Int.Class C12N 9/22
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.)
16acting on ester bonds (3.1)
22Ribonucleases
Appl.No 202380074860.7 Applicant 埃门多生物公司 Inventor R·伊曼纽尔
包含向导序列部分的RNA分子及其组合物、方法和用途,所述向导序列部分具有SEQ ID NO:1‑28998中任一项所示序列中的17‑50个连续核苷酸。
7.2025516659オリゴヌクレオチド組成物及びその方法
JP 30.05.2025
Int.Class C12Q 1/6811
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
6811Selection methods for production or design of target specific oligonucleotides or binding molecules
Appl.No 2024566662 Applicant ウェイブ ライフ サイエンシズ リミテッド Inventor シヴァリラ,チック シャクティ
とりわけ、本開示は、アデノシン修飾に有用なオリゴヌクレオチド、組成物及びその方法を提供する。いくつかの実施形態では、本開示は、アデノシン修飾から利益を得る可能性がある様々な状態、障害、又は疾患を処置するための方法を提供する。
【選択図】なし
8.1020250075592CISH의 이중 대립유전자 녹아웃
KR 28.05.2025
Int.Class C12N 15/63
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
Appl.No 1020257011942 Applicant 에멘도바이오 인코포레이티드 Inventor 엠마누엘 라피
서열번호 1-6833 중 어느 하나에 제시된 서열의 뉴클레오티드를 포함하는 17-50개의 연속 뉴클레오티드를 갖는 가이드 서열 부분을 포함하는 RNA 분자를 포함하는 조성물, 및 그의 방법 및 용도.
9.1020250073155FASLG의 이중 대립유전자 녹아웃
KR 27.05.2025
Int.Class C12N 15/63
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
Appl.No 1020257011858 Applicant 에멘도바이오 인코포레이티드 Inventor 엠마누엘 라피
서열번호 1-11121 중 어느 하나에 제시된 서열의 뉴클레오티드를 포함하는 17-50개의 연속 뉴클레오티드를 갖는 가이드 서열 부분을 포함하는 RNA 분자를 포함하는 조성물, 및 그의 방법 및 용도.
10.1020250072709CTLA4의 이중 대립유전자 녹아웃
KR 26.05.2025
Int.Class C12N 15/63
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
Appl.No 1020257010505 Applicant 에멘도바이오 인코포레이티드 Inventor 엠마누엘 라피
서열번호 1-4886 중 어느 하나에 제시된 서열의 뉴클레오티드를 포함하는 17-50개의 연속 뉴클레오티드를 갖는 가이드 서열 부분을 포함하는 RNA 분자를 포함하는 조성물, 및 그의 방법 및 용도.