CN116801913A - 用于靶向bcl11a的组合物和方法 - Google Patents

用于靶向bcl11a的组合物和方法 Download PDF

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Publication number
CN116801913A
CN116801913A CN202180092086.3A CN202180092086A CN116801913A CN 116801913 A CN116801913 A CN 116801913A CN 202180092086 A CN202180092086 A CN 202180092086A CN 116801913 A CN116801913 A CN 116801913A
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CN
China
Prior art keywords
sequence
seq
grna
casx
protein
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Pending
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CN202180092086.3A
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English (en)
Chinese (zh)
Inventor
B·奥克斯
S·希金斯
S·丹尼
B·T·斯特尔
I·科林
M·阿迪尔
C·乌尔内斯
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Scribe Therapy
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Scribe Therapy
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Publication of CN116801913A publication Critical patent/CN116801913A/zh
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Reproductive Health (AREA)
  • Hematology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180092086.3A 2020-12-03 2021-12-02 用于靶向bcl11a的组合物和方法 Pending CN116801913A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063120885P 2020-12-03 2020-12-03
US63/120,885 2020-12-03
PCT/US2021/061672 WO2022120094A2 (en) 2020-12-03 2021-12-02 Compositions and methods for the targeting of bcl11a

Publications (1)

Publication Number Publication Date
CN116801913A true CN116801913A (zh) 2023-09-22

Family

ID=81854906

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180092086.3A Pending CN116801913A (zh) 2020-12-03 2021-12-02 用于靶向bcl11a的组合物和方法

Country Status (15)

Country Link
US (1) US20240026386A1 (https=)
EP (1) EP4255502A4 (https=)
JP (1) JP2023552379A (https=)
KR (1) KR20230129230A (https=)
CN (1) CN116801913A (https=)
AU (1) AU2021391783A1 (https=)
BR (1) BR112023010717A2 (https=)
CA (1) CA3200815A1 (https=)
CL (2) CL2023001593A1 (https=)
CO (1) CO2023007900A2 (https=)
GB (1) GB2616795B (https=)
IL (1) IL303359A (https=)
MX (1) MX2023006565A (https=)
PE (1) PE20231177A1 (https=)
WO (1) WO2022120094A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
WO2022261149A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
US20250361525A1 (en) 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017252023B2 (en) * 2016-04-18 2024-05-02 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
MX2019003674A (es) * 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
CN110168084A (zh) * 2016-12-05 2019-08-23 爱迪塔斯医药公司 用于内源和来源DNA的单发指导RNA(ogRNA)靶向的系统和方法
US20190367924A1 (en) * 2017-02-17 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
WO2019213273A1 (en) * 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
JP2021532815A (ja) * 2018-08-07 2021-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１２ｂ酵素およびシステム

Also Published As

Publication number Publication date
CA3200815A1 (en) 2022-06-09
CO2023007900A2 (es) 2023-06-30
KR20230129230A (ko) 2023-09-07
EP4255502A2 (en) 2023-10-11
US20240026386A1 (en) 2024-01-25
EP4255502A4 (en) 2025-07-16
GB2616795A (en) 2023-09-20
WO2022120094A2 (en) 2022-06-09
PE20231177A1 (es) 2023-08-01
JP2023552379A (ja) 2023-12-15
GB2616795B (en) 2025-08-13
GB202309871D0 (en) 2023-08-16
BR112023010717A2 (pt) 2023-10-03
CL2023001593A1 (es) 2023-12-15
WO2022120094A3 (en) 2022-08-25
AU2021391783A1 (en) 2023-06-22
MX2023006565A (es) 2023-08-07
IL303359A (en) 2023-08-01
CL2024001577A1 (es) 2024-10-18

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