KR20240034746A - 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도 - Google Patents

하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도 Download PDF

Info

Publication number
KR20240034746A
KR20240034746A KR1020247000358A KR20247000358A KR20240034746A KR 20240034746 A KR20240034746 A KR 20240034746A KR 1020247000358 A KR1020247000358 A KR 1020247000358A KR 20247000358 A KR20247000358 A KR 20247000358A KR 20240034746 A KR20240034746 A KR 20240034746A
Authority
KR
South Korea
Prior art keywords
seq
sequence
nucleotides
nos
polypeptide
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
KR1020247000358A
Other languages
English (en)
Korean (ko)
Inventor
퀸튼 노먼 웨셀스
제프리 레이먼드 하스웰
티아 마리 디토마소
노아 마이클 자키모
세주티 생업타
Original Assignee
아버 바이오테크놀로지스, 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 아버 바이오테크놀로지스, 인크. filed Critical 아버 바이오테크놀로지스, 인크.
Publication of KR20240034746A publication Critical patent/KR20240034746A/ko
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015(S)-2-Hydroxy-acid oxidase (1.1.3.15)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247000358A 2021-06-04 2022-06-03 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도 Pending KR20240034746A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163197073P 2021-06-04 2021-06-04
US63/197,073 2021-06-04
US202163225046P 2021-07-23 2021-07-23
US63/225,046 2021-07-23
US202163292889P 2021-12-22 2021-12-22
US63/292,889 2021-12-22
US202263300727P 2022-01-19 2022-01-19
US63/300,727 2022-01-19
PCT/US2022/032144 WO2022256642A2 (en) 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof

Publications (1)

Publication Number Publication Date
KR20240034746A true KR20240034746A (ko) 2024-03-14

Family

ID=82694238

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247000358A Pending KR20240034746A (ko) 2021-06-04 2022-06-03 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도

Country Status (12)

Country Link
US (3) US20230032141A1 (https=)
EP (1) EP4347833A2 (https=)
JP (1) JP2024520691A (https=)
KR (1) KR20240034746A (https=)
AU (1) AU2022283922A1 (https=)
CA (1) CA3222159A1 (https=)
CO (1) CO2023018656A2 (https=)
IL (1) IL308803A (https=)
MX (1) MX2023014406A (https=)
PH (1) PH12023553297A1 (https=)
TW (1) TW202317763A (https=)
WO (1) WO2022256642A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4711451A1 (en) * 2023-05-11 2026-03-18 Yoltech Therapeutics Co., Ltd Cas protein, corresponding gene editing system thereof and use thereof
WO2025147604A1 (en) 2024-01-05 2025-07-10 Arbor Biotechnologies, Inc. Methods for treating primary hyperoxaluria via genetic editing of hydroxyacid oxidase 1

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3581654B1 (en) * 2013-12-27 2021-03-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
MX2021001070A (es) * 2018-07-31 2021-05-27 Intellia Therapeutics Inc Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1).
CN121380024A (zh) * 2020-03-31 2026-01-23 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途
WO2021257730A2 (en) * 2020-06-16 2021-12-23 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法

Also Published As

Publication number Publication date
JP2024520691A (ja) 2024-05-24
US20240254494A1 (en) 2024-08-01
AU2022283922A9 (en) 2023-12-14
CA3222159A1 (en) 2022-12-08
WO2022256642A3 (en) 2023-01-12
US20230212541A1 (en) 2023-07-06
AU2022283922A1 (en) 2023-12-07
PH12023553297A1 (en) 2024-03-18
CO2023018656A2 (es) 2024-01-25
IL308803A (en) 2024-01-01
TW202317763A (zh) 2023-05-01
WO2022256642A2 (en) 2022-12-08
EP4347833A2 (en) 2024-04-10
US20230032141A1 (en) 2023-02-02
MX2023014406A (es) 2024-02-12
US11821012B2 (en) 2023-11-21

Similar Documents

Publication Publication Date Title
KR102712656B1 (ko) Hsd17b13 변종 및 이것의 용도
AU2016206870B2 (en) Gene editing through microfluidic delivery
CN113801881A (zh) 超级增强子基因序列在促进人b2m基因表达中的用途
TW202317761A (zh) 包含靶向運甲狀腺素蛋白(ttr)的rna指導物的基因編輯系統及其用途
KR20240034746A (ko) 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
US20250127924A1 (en) Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
KR20230093328A (ko) B2m을 표적화하는 rna 가이드를 포함하는 조성물 및 이의 용도
KR20230107265A (ko) Pdcd1을 표적화하는 rna 가이드를 포함하는 조성물 및 이의 용도
CN116648505A (zh) 包含靶向b2m的rna指导物的组合物及其用途
CN113846063A (zh) 一种适用于同种异体移植的通用型人源干细胞以及构建方法
US11939607B2 (en) Gene editing systems comprising an RNA guide targeting lactate dehydrogenase a (LDHA) and uses thereof
WO2023122433A1 (en) Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha)
US20240026351A1 (en) Compositions comprising an rna guide targeting trac and uses thereof
TW202334421A (zh) 包含靶向ciita之rna引導之組合物及其用途
HK40109011A (zh) 包括靶向羟基酸氧化酶1(hao1)的rna向导的基因编辑系统和其用途
KR102958345B1 (ko) Hsd17b13 변종 및 이것의 용도
CN116670275A (zh) 包含靶向pdcd1的rna指导物的组合物及其用途
CN117897485A (zh) 包括靶向羟基酸氧化酶1(hao1)的rna向导的基因编辑系统和其用途
CN117813382A (zh) 包括靶向stathmin 2(stmn2)的rna向导的基因编辑系统和其用途
CN116867898A (zh) 包含靶向trac的rna指导物的组合物及其用途
KR20230107595A (ko) Bcl11a를 표적화하는 rna 가이드를 포함하는 조성물 및 이의 용도
HK40109670A (zh) 包括靶向stathmin 2(stmn2)的rna向导的基因编辑系统和其用途
WO2025212120A1 (en) Chemical modifications of guide rnas for crispr nucleases

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20240104

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250602

Comment text: Request for Examination of Application