EA202192806A1 - Системы редактирования генов для модифицирования гена scn9a или scn10a и способы их применения - Google Patents

Системы редактирования генов для модифицирования гена scn9a или scn10a и способы их применения

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Publication number
EA202192806A1
EA202192806A1 EA202192806A EA202192806A EA202192806A1 EA 202192806 A1 EA202192806 A1 EA 202192806A1 EA 202192806 A EA202192806 A EA 202192806A EA 202192806 A EA202192806 A EA 202192806A EA 202192806 A1 EA202192806 A1 EA 202192806A1
Authority
EA
Eurasian Patent Office
Prior art keywords
gene
editing systems
scn9a
scn10a
gene editing
Prior art date
Application number
EA202192806A
Other languages
English (en)
Russian (ru)
Inventor
Шина Саайман
Стюарт Кахалан
Анджела Йен
София Эспанола
Сара Джин Спенсер
Original Assignee
Вертекс Фармасьютикалз Инкорпорейтед
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 Вертекс Фармасьютикалз Инкорпорейтед filed Critical Вертекс Фармасьютикалз Инкорпорейтед
Publication of EA202192806A1 publication Critical patent/EA202192806A1/ru

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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/10—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
    • C12N15/63—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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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
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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
    • C12N15/1138—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 receptors or cell surface proteins
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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
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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]
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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]
    • 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
    • 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
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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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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
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    • C12N2800/90—Vectors containing a transposable element

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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Rheumatology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EA202192806A 2019-04-12 2020-04-10 Системы редактирования генов для модифицирования гена scn9a или scn10a и способы их применения EA202192806A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962833523P 2019-04-12 2019-04-12
PCT/US2020/027690 WO2020210640A1 (en) 2019-04-12 2020-04-10 Gene-editing systems for modifying a scn9a or scn10a gene and methods of use thereof

Publications (1)

Publication Number Publication Date
EA202192806A1 true EA202192806A1 (ru) 2022-02-03

Family

ID=70416602

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192806A EA202192806A1 (ru) 2019-04-12 2020-04-10 Системы редактирования генов для модифицирования гена scn9a или scn10a и способы их применения

Country Status (15)

Country Link
US (1) US20200354741A1 (enExample)
EP (1) EP3953460A1 (enExample)
JP (2) JP2022526670A (enExample)
KR (2) KR20210151110A (enExample)
CN (2) CN119286934A (enExample)
AU (1) AU2020272031B2 (enExample)
BR (1) BR112021020406A2 (enExample)
CA (1) CA3136700A1 (enExample)
CO (1) CO2021014015A2 (enExample)
EA (1) EA202192806A1 (enExample)
IL (2) IL328663A (enExample)
JO (1) JOP20210276A1 (enExample)
MX (3) MX2021012493A (enExample)
SG (1) SG11202111270SA (enExample)
WO (1) WO2020210640A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240366680A1 (en) * 2021-08-16 2024-11-07 Editas Medicine, Inc. Treatment of pain
EP4731643A2 (en) * 2023-06-26 2026-04-29 Navega Therapeutics, Inc. Compositions and methods for reducing pain and inflammation through epigenetic modulation
AU2025207849A1 (en) * 2024-01-11 2026-07-16 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to nervous system tissues
WO2025153066A1 (zh) * 2024-01-19 2025-07-24 云合智药（苏州）生物科技有限公司 用于抑制钠电压门控通道α亚基10(SCN10A)基因表达的核酸及其用途

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201418713D0 (en) * 2014-10-21 2014-12-03 Kymab Ltd Bindings Proteins
WO2016205759A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
EP3481857B1 (en) * 2016-07-06 2026-02-11 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
EP4650364A3 (en) * 2016-07-06 2026-01-21 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
WO2018165504A1 (en) * 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing

Also Published As

Publication number Publication date
MX2021012493A (es) 2022-01-06
WO2020210640A1 (en) 2020-10-15
AU2020272031B2 (en) 2026-08-06
AU2020272031A1 (en) 2021-11-18
IL328663A (en) 2026-07-01
JP2025118676A (ja) 2025-08-13
IL287149A (en) 2021-12-01
CO2021014015A2 (es) 2021-10-29
EP3953460A1 (en) 2022-02-16
SG11202111270SA (en) 2021-11-29
CN119286934A (zh) 2025-01-10
MX2026003964A (es) 2026-05-04
KR20210151110A (ko) 2021-12-13
KR20250134148A (ko) 2025-09-09
MX2026003965A (es) 2026-05-04
JP2022526670A (ja) 2022-05-25
JOP20210276A1 (ar) 2023-01-30
US20200354741A1 (en) 2020-11-12
BR112021020406A2 (pt) 2021-12-07
CN113994009B (zh) 2025-05-06
CA3136700A1 (en) 2020-10-15
CN113994009A (zh) 2022-01-28

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