KR20220125332A - Pcsk9의 표적화를 위한 조성물 및 방법 - Google Patents

Pcsk9의 표적화를 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20220125332A
KR20220125332A KR1020227027481A KR20227027481A KR20220125332A KR 20220125332 A KR20220125332 A KR 20220125332A KR 1020227027481 A KR1020227027481 A KR 1020227027481A KR 20227027481 A KR20227027481 A KR 20227027481A KR 20220125332 A KR20220125332 A KR 20220125332A
Authority
KR
South Korea
Prior art keywords
sequence
seq
gna
pcsk9
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020227027481A
Other languages
English (en)
Korean (ko)
Inventor
벤자민 오크스
션 히긴스
한나 스피너
사라 데니
브렛 티 스탈
키안 타일러
캐서린 베니
이사벨 콜린
마루프 아딜
콜 우르네스
Original Assignee
스크라이브 테라퓨틱스 인크.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 스크라이브 테라퓨틱스 인크. filed Critical 스크라이브 테라퓨틱스 인크.
Publication of KR20220125332A publication Critical patent/KR20220125332A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21111—Aqualysin 1 (3.4.21.111)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70539—MHC-molecules, e.g. HLA-molecules
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020227027481A 2020-01-10 2021-01-08 Pcsk9의 표적화를 위한 조성물 및 방법 Pending KR20220125332A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062959685P 2020-01-10 2020-01-10
US62/959,685 2020-01-10
PCT/US2021/012804 WO2021142342A1 (en) 2020-01-10 2021-01-08 Compositions and methods for the targeting of pcsk9

Publications (1)

Publication Number Publication Date
KR20220125332A true KR20220125332A (ko) 2022-09-14

Family

ID=74505368

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227027481A Pending KR20220125332A (ko) 2020-01-10 2021-01-08 Pcsk9의 표적화를 위한 조성물 및 방법

Country Status (9)

Country Link
US (1) US20230167424A1 (enExample)
EP (1) EP4087930A1 (enExample)
JP (1) JP2023510352A (enExample)
KR (1) KR20220125332A (enExample)
CN (1) CN115427570A (enExample)
AU (1) AU2021206270A1 (enExample)
CA (1) CA3163714A1 (enExample)
IL (1) IL294620A (enExample)
WO (1) WO2021142342A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
CA3257824A1 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
EP4536286A1 (en) 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Mecp2発現を低減する化合物及び方法
WO2024206555A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2024238723A1 (en) * 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
CN118086311B (zh) * 2023-05-25 2024-08-09 苏州时安生物技术有限公司 抑制PCSK9基因表达的siRNA、其缀合物和药物组合物及用途
EP4735603A2 (en) * 2023-06-30 2026-05-06 nChroma Bio, Inc. Guide rna compositions
CN116978457B (zh) * 2023-09-22 2023-12-22 成都斯马特科技有限公司 避免rna检测过程中假基因干扰的引物和探针组合及其设计方法
CN119506351B (zh) * 2024-11-20 2025-08-19 中国人民解放军陆军军医大学第二附属医院 一体化pcsk9腺嘌呤碱基编辑系统及应用

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
US11866727B2 (en) * 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY
JP2019532644A (ja) * 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Rna誘導型核酸修飾酵素及びその使用方法
KR102569848B1 (ko) * 2016-12-23 2023-08-25 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
JP7277052B2 (ja) * 2017-02-22 2023-05-18 クリスパー セラピューティクス アーゲー プロタンパク質転換酵素サブチリシン/ケキシン9型(pcsk9)関連障害の処置のための組成物および方法
US11773409B2 (en) 2017-04-21 2023-10-03 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
WO2019084148A1 (en) * 2017-10-25 2019-05-02 Monsanto Technology Llc TARGETED RNA GUIDED ENDONUCLEASE ENDONUCLEASE ACTIVITY IN EUKARYOTES
WO2019126762A2 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
SG11202113253SA (en) * 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
US12551560B2 (en) * 2019-09-09 2026-02-17 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy

Also Published As

Publication number Publication date
CN115427570A (zh) 2022-12-02
US20230167424A1 (en) 2023-06-01
CA3163714A1 (en) 2021-07-15
AU2021206270A1 (en) 2022-07-21
EP4087930A1 (en) 2022-11-16
JP2023510352A (ja) 2023-03-13
WO2021142342A1 (en) 2021-07-15
IL294620A (en) 2022-09-01

Similar Documents

Publication Publication Date Title
AU2022200903C1 (en) Engineered Cascade components and Cascade complexes
AU2018229561B2 (en) Recombinant adenoviruses and use thereof
CN111593073B (zh) 双报告基因骨架载体、四质粒假病毒包装系统、包装新冠肺炎假病毒
AU2018274655B2 (en) Viral expression construct comprising a fibroblast growth factor 21 (FGF21) coding sequence
KR101666228B1 (ko) 생물치료학적 분자를 발현시키기 위한 치료학적 유전자-스위치 작제물 및 생물반응기, 및 이의 용도
US6090393A (en) Recombinant canine adenoviruses, method for making and uses thereof
US6156567A (en) Truncated transcriptionally active cytomegalovirus promoters
KR102049161B1 (ko) 치료적 단백질을 조건부로 발현하는 벡터,상기 벡터를 포함하는 숙주 세포 및 이의 용도
KR20200074988A (ko) 아데노바이러스 벡터 및 이의 용도
KR20140113997A (ko) 부탄올 생성을 위한 유전자 스위치
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
KR20230129230A (ko) Bcl11a의 표적화를 위한 조성물 및 방법
CN115427570A (zh) 用于靶向pcsk9的组合物和方法
KR20240029020A (ko) Dna 변형을 위한 crispr-트랜스포손 시스템
KR20240037192A (ko) 게놈 통합을 위한 방법 및 조성물
TW202308669A (zh) 嵌合共刺激性受體、趨化激素受體及彼等於細胞免疫治療之用途
KR20230003477A (ko) 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도
AU2023265204A1 (en) Virus-derived construct plasmid library and construction of same
TW202605154A (zh) 大猩猩腺病毒核酸及胺基酸序列,包含彼之載體,及其用途
KR20240022571A (ko) Rna-가이드된 이펙터 동원을 위한 시스템, 방법 및 성분
CN116323955A (zh) 通过crispr/cas介导的体内末端解析拯救重组腺病毒
RU2853834C1 (ru) Последовательности нуклеиновых кислот и аминокислот аденовирусов гориллы, содержащие их векторы и их применение
RU2774631C1 (ru) Сконструированные компоненты cascade и комплексы cascade
RU2812852C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фактора viii (fviii)
RU2827658C2 (ru) Сконструированные компоненты cascade и комплексы cascade

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

P22-X000 Classification modified

St.27 status event code: A-2-2-P10-P22-nap-X000