CA3145112A1 - Engineered nucleic acid regulatory element and methods of uses thereof - Google Patents

Engineered nucleic acid regulatory element and methods of uses thereof Download PDF

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Publication number
CA3145112A1
CA3145112A1 CA3145112A CA3145112A CA3145112A1 CA 3145112 A1 CA3145112 A1 CA 3145112A1 CA 3145112 A CA3145112 A CA 3145112A CA 3145112 A CA3145112 A CA 3145112A CA 3145112 A1 CA3145112 A1 CA 3145112A1
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Prior art keywords
aav
seq
promoter
transgene
expression
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Pending
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CA3145112A
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English (en)
French (fr)
Inventor
Chunping Qiao
Devin MCDOUGALD
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Regenxbio Inc
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Regenxbio Inc
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Publication of CA3145112A1 publication Critical patent/CA3145112A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle 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
    • C12N2830/008—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
    • 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
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region

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  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CA3145112A 2019-07-26 2020-07-24 Engineered nucleic acid regulatory element and methods of uses thereof Pending CA3145112A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962879358P 2019-07-26 2019-07-26
US62/879,358 2019-07-26
US202062967480P 2020-01-29 2020-01-29
US62/967,480 2020-01-29
US202063023152P 2020-05-11 2020-05-11
US63/023,152 2020-05-11
PCT/US2020/043578 WO2021021661A1 (en) 2019-07-26 2020-07-24 Engineered nucleic acid regulatory element and methods of uses thereof

Publications (1)

Publication Number Publication Date
CA3145112A1 true CA3145112A1 (en) 2021-02-04

Family

ID=72047119

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3145112A Pending CA3145112A1 (en) 2019-07-26 2020-07-24 Engineered nucleic acid regulatory element and methods of uses thereof

Country Status (6)

Country Link
US (1) US20230042103A1 (https=)
EP (1) EP4004214A1 (https=)
JP (1) JP2022544004A (https=)
CA (1) CA3145112A1 (https=)
IL (1) IL289922A (https=)
WO (1) WO2021021661A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113322281A (zh) * 2021-05-12 2021-08-31 成都金唯科生物科技有限公司 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
WO2024094009A1 (zh) * 2022-10-31 2024-05-10 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用

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EP4707401A2 (en) * 2021-04-09 2026-03-11 University of Washington Artificial regulatory cassettes for muscle-specific gene expression
US20250025574A1 (en) * 2021-10-28 2025-01-23 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
CN114470241A (zh) * 2022-03-03 2022-05-13 中日友好医院(中日友好临床医学研究所) 肝脏靶向性γ-干扰素表达载体在制备治疗抗肝脏肿瘤的药物中的应用
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
JP2025535114A (ja) * 2022-10-11 2025-10-22 メイラグティーエックス ユーケー アイアイ リミティド 中枢神経系における遺伝子発現のための核酸調節エレメント及び使用方法
EP4601710A2 (en) * 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024102703A2 (en) * 2022-11-07 2024-05-16 The Regents Of The University Of California Zikv-based gene delivery system
KR20260005328A (ko) * 2023-04-28 2026-01-09 청두 오리젠 바이오테크놀로지 씨오., 엘티디. 항vegf 단백질을 코딩하는 핵산, 폴리뉴클레오타이드 발현 카세트 및 재조합 아데노 관련 바이러스
WO2026028089A1 (en) * 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

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CN113322281A (zh) * 2021-05-12 2021-08-31 成都金唯科生物科技有限公司 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
CN113322281B (zh) * 2021-05-12 2024-01-05 成都金唯科生物科技有限公司 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
WO2024094009A1 (zh) * 2022-10-31 2024-05-10 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用

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Publication number Publication date
IL289922A (en) 2022-03-01
US20230042103A1 (en) 2023-02-09
WO2021021661A1 (en) 2021-02-04
JP2022544004A (ja) 2022-10-17
EP4004214A1 (en) 2022-06-01

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