KR20240023643A - 조절 핵산 서열 - Google Patents

조절 핵산 서열 Download PDF

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Publication number
KR20240023643A
KR20240023643A KR1020247002456A KR20247002456A KR20240023643A KR 20240023643 A KR20240023643 A KR 20240023643A KR 1020247002456 A KR1020247002456 A KR 1020247002456A KR 20247002456 A KR20247002456 A KR 20247002456A KR 20240023643 A KR20240023643 A KR 20240023643A
Authority
KR
South Korea
Prior art keywords
muscle
promoter
specific
synthetic
gene
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
KR1020247002456A
Other languages
English (en)
Korean (ko)
Inventor
호르헤 오마르 야네즈-쿠나
후안 마누엘 이글레시아스
싱클레어 쿠퍼
마이클 엘. 로버츠
안토니아 에브리피오티
Original Assignee
신프로믹스 엘티디
아스클레피오스 바이오파마슈티컬, 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
Priority claimed from GBGB2108997.4A external-priority patent/GB202108997D0/en
Application filed by 신프로믹스 엘티디, 아스클레피오스 바이오파마슈티컬, 인크. filed Critical 신프로믹스 엘티디
Publication of KR20240023643A publication Critical patent/KR20240023643A/ko
Pending legal-status Critical Current

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Classifications

    • 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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020247002456A 2021-06-23 2022-06-23 조절 핵산 서열 Pending KR20240023643A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2108997.4A GB202108997D0 (en) 2021-06-23 2021-06-23 Regulatory nucleic acid sequences
GB2108997.4 2021-06-23
GB2111317.0 2021-08-05
GB202111317 2021-08-05
PCT/GB2022/051611 WO2022269269A1 (en) 2021-06-23 2022-06-23 Regulatory nucleic acid sequences

Publications (1)

Publication Number Publication Date
KR20240023643A true KR20240023643A (ko) 2024-02-22

Family

ID=82655313

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247002456A Pending KR20240023643A (ko) 2021-06-23 2022-06-23 조절 핵산 서열

Country Status (8)

Country Link
US (1) US20250129381A1 (https=)
EP (1) EP4359548A1 (https=)
JP (1) JP2024524270A (https=)
KR (1) KR20240023643A (https=)
AU (1) AU2022297795A1 (https=)
CA (1) CA3221707A1 (https=)
IL (1) IL309630A (https=)
WO (1) WO2022269269A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202319008D0 (en) * 2023-12-13 2024-01-24 Ucl Business Ltd AAV-mediated gene therapy of diseases caused by impaired function of GLDC

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
PL3097197T3 (pl) * 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
TWI908746B (zh) * 2019-11-28 2025-12-21 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
EP4192580A4 (en) * 2020-08-05 2025-06-18 AskBio Inc. Methods for treating heart disease and congestive heart failure and administration of AAV vectors
CA3195177A1 (en) * 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)

Also Published As

Publication number Publication date
JP2024524270A (ja) 2024-07-05
WO2022269269A1 (en) 2022-12-29
EP4359548A1 (en) 2024-05-01
IL309630A (en) 2024-02-01
CA3221707A1 (en) 2022-12-29
AU2022297795A1 (en) 2024-01-04
US20250129381A1 (en) 2025-04-24

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Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20240122

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: 20250623

Comment text: Request for Examination of Application